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Term Definition
a4 Integrin Inhibitors Class of molecules which blocks white cell adhesion to blood vessel walls and subsequent migration of white blood cells into target tissues where they can wreak inflammatory havoc.
a4b1 Integrin A protein expressed on white blood cells that mediates adhesion and migration. Integrins are important in regulating the movements and interactions of cells.
a4b7 Integrin A protein expressed on white blood cells that mediates adhesion and migration. Integrins are important in regulating the movements and interactions of cells.
b2 agonist Shorthand for b2-adrenergic (receptor) agonist. Any of a class of compounds used to provide therapeutic bronchodilation (for asthma, COPD, and other disoders featuring constricted airways), sports performance enhancement, or weight loss.
b-amyloid Peptide (Ab) A protein fragment that has specific forms known to be constituents of senile plaques in Alzheimer disease, and implicated in the pathogenesis of the disorder.
b-secretases (BACE) These and g-secretases are enzymes that cleave amyloid precursor protein, producing b-amyloid peptide. Their inhibitors may reduce bamyloid peptide in Alzheimer disease patientsâ?? brains.
b-Thalassemia One of a group of disorders characterized by one of various beta-globin gene mutations leading to decreased or abnormal hemoglobin synthesis. Manifestations span a spectrum from none through severe, fatal anemia.
g-secretase An enzymes that cleave amyloid precursor protein, producing b-amyloid peptide. Inhibitors of g- or b-secretases may reduce bamyloid peptide in Alzheimer disease patients' brains.
11b-HSD1 11 beta-hydroxysteroid dehydrogenase type 1 is an enzyme involved in the production of glucocorticoids such as cortisol. Its overproduction in liver and fatty tissues may participate in the development of insulin resistance and certain aspects of obesity. Drugs targeting 11b-HSD1 are being developed as treatments for various conditions involving resistance to insulin’s effects such as type II diabetes, obesity, the full-blown insulin resistance syndrome, and related disorders. Furthermore, specific polymorphisms of the gene encoding 11b-HSD1 may prove useful as predictive tools both regarding individuals’ susceptibility or resistance to impaired glucose tolerance and regarding their predilection to therapeutically or adversely respond to various anti-diabetic therapies. Inhibition of the closely related enzyme 11b-HSD1 by licorice leads to an excess of steroid manufacture associated with low blood levels of potassium as well as high blood pressure. Its agonism may prove to have certain biological benefits similar to those of 11b-HSD1 antagonism.
2-Methoxyesteradiol (2-ME2) EntreMed’s formulation of a naturally occurring estrogen metabolite. Its anti-angiogenic properties, though poorly understood, have made it an investigational agent in several anti-cancer settings.
5A8 (Biogen) An experimental anti CD4 therapeutic monoclonal antibody in development against multiple sclerosis.
Abl A kinase (signaling protein that adds phosphates to proteins) involved in regulating cell division and other cellular events.
Absorption A pharmacokinetic property of a drug (or anything else entering the body) relating to its incorporation into cells, tissues, or organs, especially as it relates to the gastrointestinal, renal (kidney), or circulatory (blood vessel) system. Also called uptake.
ACE Inhibitor Any of several drugs that inhibit the biological activity of angiotensin converting enzyme (ACE). ACE activity is fundamental to the formation of the protein angiotensin II. Angiotensin II facilitates blood vessel constriction (vasoconstriction), and in so doing raises blood pressure. Thus, ACE inhibitors lower blood pressure by diminishing angiotensin II formation, thereby allowing arterial relaxation (vasodilation). Lowering peripheral blood pressure and, thus, diminishing the resistance against which a heart must pump, crucial especially for those with failing hearts, have been the main sources of clinical value for these drugs. ACE inhibitors can also slow the progression of diabetic kidney disease in type II diabetes. These drugs were originally isolated from the venom of a poisonous Brazilian snake.
Acetylcholine (Ach) The acetic ester of choline, it is a neurotransmitter that transmits nerve-to-nerve signals in the brain, causes dilation of blood vessels, inhibition of specific cardiac functions related to pumping force and beating rate, and promotion of gastrointestinal motility (peristalsis). Its depletion in Alzheimer disease is the basis for therapies that increase its availability.
Acetylcholinesterase (AchE) The enzyme responsible for breaking down acetylcholine after it is used in a nerve-to-nerve or nerve-to-muscle signal.
Acetylcholinesterase Inhibitor An agent that interferes with the enzyme, acetylcholinesterase, that breaks down acetylcholine. Used to increase the amount of acetylcholine in the brain of Alzheimer patients, which improves memory and cognition.
Activated Protein C Biologically functioning protein C, also known as APC. Protein C, when activated, functions as an anticoagulant in a system of multiple proteins (the coagulation cascade) that, in the body, maintain the consistency of blood such that people experience effects of neither clotting nor bleeding. In sepsis, APC is depleted.
Acute Of short duration; not chronic.
Acute Coronary Syndrome Aggregate term for acute events in ischemic heart disease: unstable angina, acute myocardial infarction (heart attack), and sudden cardiac death"
Acute Disseminated Encephalomyelitis (ADE) A neurodegenerative disorder characterized by inflammation of the brain and spinal cord caused by damage myelin sheath. Typically, it occurs as a complication of a bacterial or viral infection.
Acute Myelogenous Leukemia (AML) A group of cancers of myeloid-derived blood cell precursors (including those of promyelocyte, myelomonocyte, monocyte, erythrocyte precursors, and megakaryocyte origin) characterized by a rapid onset and often having readily seen, microscopic, specific, distinguishing, somatic chromosomal abnormalities.
Acute Myocardial Infarction A heart attack. Heart muscle (myocardium) tissue, due to abrupt (acute) circulating blood and oxygen deprivation, dies. Tissue death (necrosis) due to oxygen deprivation is known as ""infarction." Blood flow interruptions are typically caused by arteriosclerosis accompanied by coronary artery narrowing. Eventually, a thrombosis (clot) occludes the coronary artery, preventing the flow of blood, and therefore limiting the oxygen supply, to the myocardium.
Acute Necrotizing Hemorrhagic Encephalomyelitis A condition resembling ADE that includes bleeding and a more rapid course of a specific type of cell death.
Acyl CoA Cholesterol Aceyltransferase (ACAT) An enzyme involved in normal cholesterol production in every cell, it has also been recently shown to have a role in Ab formation in Alzheimer disease.
Adeno Associated Virus (AAV) A gene therapy vector which has been gaining popularity recently in the medical community for its excellent safety profile. Targeted Genetics and Avigen are leaders in the use of AAV.
Adenovirus A family of naturally occurring DNA viruses which can cause flu-like symptoms in people. The most popular gene delivery system until the death of an adenovirus trial participant last year. Now out of favor but still actively pursued by several companies including Onyx and Introgen.
Adhesion Molecules Cell surface molecules that mediate attachment between cells and attachment of cells to other substrates such as extracellular matrix. Adhesion molecules are like Velcro, with one adhesion molecule on the first cell or surface binding to its complementary adhesion molecule on the other cell. Adhesion molecules include ICAM, VECAM, and the family of molecules known as integrins.
Adipose Fat or fat cells.
Adjunctive Ancillary or secondary drug or treatment.
Adjuvant Contributing to the treatment of disease. Often adjuvant chemotherapy is that which follows surgery. Adjuvant chemo has the theoretical advantage of eliminating residual cancerous cells so as to improve survival. Neoadjuvant chemo is also known as primary or induction therapy, and is when chemo is given prior to surgery instead. With neoadjuvant therapy the physicians are able to monitor drug sensitivity in the intact primary tumor and determine what definitive local management is in order.
Admark Assays (Elan) Tools that identify the forms of, potentially predisposing genetic markers, ApoE, a patient has, and measure the biochemical markers, tau and the bamyloid peptide, in cerebrospinal fluid.
Adrenoleukodystrophy A rare, genetic disorder characterized by central nervous system myelin destruction and progressive dysfunction of the adrenal gland. A boy with this condition was the subject of the acclaimed movie, Lorenzo’s Oil.
Adverse Effect Profile Inventory of characteristic adverse effects, often evaluated along a spectrum from low to high corresponding with a small to large inventory. "
Adverse Event A problematic medical occurrence that may have a causal relationship with a treatment. A problematic occurrence may be an unfavorable or unintended symptom, sign, disorder, disease, or something else untoward that is temporally associated with administration of an intervention.
Aerisolize To disperse as a suspension of fine solid or liquid particles into the air or the lungs.
Affinity The strength of binding between two molecules, such as how strongly a monoclonal antibody binds to its antigen.
Affinity Maturation The natural process by which B cells producing higher affinity antibodies are selected during an immune response. This process leads to the production of high affinity antibodies.
Agonist A molecule that binds to and triggers the effect of another molecule.
AIDS (Acquired Immunodeficiency Syndrome) An epidemic disease caused by infection with Human Immunodeficiency Virus, which induces failure of the immune system.
Akt Also known as Protein Kinase B, it is an intracellular signaling enzyme central to a range of biological processes within most human cells. Akt often exists as a complex with other regulatory and signaling proteins.
Allele One version of a DNA sequence that occurs at a given chromosomal locus.
Allergic Rhinitis Hay fever, a condition characterized by nasal congestion, runny nose, sneezing, nose and eye itching, and tearing eyes. It is an immune reaction that mimics a chronic cold. Literally, “rhinitis” means “inflammation of the nose.”
Allergies A group of symptoms precipitated by an immune response to substances not typically triggering an immune response in most individuals. Specific symptoms depend on the specific allergen (inciting substance), the body part exposed, and individual variation in immune responsiveness.
Allogeneic Coming from the same species but a different individual, usually referring to transplants. Grafts can also be from one's self or one's identical twin (autologous, syngeneic) or from another species (xenogeneic).
ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig disease) A neurodegenerative disease of unknown cause which causes progressive loss of nervous input to muscles. This leads to voluntary and involuntary muscle atrophy, weakness, respiratory failure, and death. Symptoms are caused by the destruction of nerve cells in the brain and spinal cord.
Altered Peptide Ligands (APLs)

A peptide (short string of amino acids) that can bind to MHC molecules. APLs are designed to be similar to known peptide antigens that stimulate T cells. By making slight changes in the peptide sequence, the APL can change the response of the particular T cell. This can lead to the suppression of an immune response, particularly desirable in the case of autoimmune responses such as lupus or type I diabetes.

Alzheimer Disease A degenerative brain disorder that primarily effects the elderly. Symptoms include cognitive and memory decline, confusion, language disturbances, personality and behavior changes, and impaired judgment.
Amino Acid The building blocks of proteins. 20 amino acids, such as cystine, methionine, and related molecules containing amino groups are strung together as dictated by genes. The specific sequence in which they are strung together dictates the form and function of each specific protein. A child needs to ingest 9 so-called essential amino acids in order to form all the necessary body proteins, whereas adults must ingest 8. The remainder can be formed in the body. When the body breaks down or otherwise metabolizes protein, many more than 20 amino acids can be formed.
Ampakines Compounds that enhance the activity of the AMPA-type glutamate receptor, a complex of proteins that is involved in most "excitatory" communication between nerve cells in the human brain. Ampakines are under investigation for Alzheimer disease, stroke, attention-deficit hyperactivity disorder, schizophrenia, depression, anxiety, and Fragile X syndrome.
Amylin

A pancreatic islet cell hormone, secreted with insulin, that plays a role in the maintenance of glucose regulation. Amylin is predominantly manufactured in pancreatic b cells and to a lesser extent in other organs of the gastrointestinal tract and nervous system. Its physical and functional replenishment, like that of insulin, tends to modulate the effects of diabetes mellitus. The degree to which this provides therapeutic benefit is currently under intense investigation. Also under investigation are its potential as a diagnostic and prognostic marker in pancreatic cancer and its correlation to the formation of amyloid.

Amyloid Any of a group of proteins that are composed of specifically arranged aggregated fibrils. Deposition of amyloid in the brain is the defining feature of Alzheimer disease, systemic amyloidosis, and amyloidosis associated with the failure of virtually every organ/organ system, and can be a feature of multiple myeloma, monoclonal B-cell tumors, the chronic kidney failure associated with hemodialysis, medullary thyroid carcinoma, renal osteodystrophy, carpal tunnel syndrome, and other diseases.
Amyloid Plaques Protein deposits made out of bamyloid peptide that form on the brain of AD patients.
Amyloid Precursor Protein (APP) A protein most often associated with the harmful material in the brains of Alzheimer disease patients, but whose normal activity is likely to be essential for normal brain function, including a role in the repair of injuries to neurons.
Amyotrophic Lateral Sclerosis (Lou Gehrig Disease) A neurodegenerative disease of unknown cause which causes progressive loss of nervous input to muscles. This leads to voluntary and involuntary muscle atrophy, weakness, respiratory failure, and death. Symptoms are caused by the destruction of nerve cells in the brain and spinal cord.
Analgesia A condition in which the perception of pain is altered. Stimuli typically perceived as painful are no longer interpreted this way, often with sedation and without a loss of consciousness.
Analgesic A compound that produces analgesia.
Analog A molecule similar in structure to another molecule. In drug discovery, analogs of promising drug candidates are made and tested for increased efficacy and decreased side effect profiles. "
Anaphylaxis An allergic reaction. Also, the colloquial name for a severe allergic reaction that can lead to anaphylactic shock, a circumstance during which a widespread allergic reaction rages out of control. Symptoms and signs of anaphylactic shock include dizziness, loss of consciousness, labored breathing, tongue and breathing tube swelling, low blood pressure, heart failure, and death. Typical known inciting agents are bee and wasp stings, various plants, peanuts, drugs, and shellfish.
Androgen Any of the various hormones that promote development and maintenance of male sex characteristics. The primary androgen is testosterone.
Anemia Any condition characterized by a reduced number of red blood cells, amount of hemoglobin, or amount of blood.
Anesthesia

(1) Loss of sensation or the state of loss of sensation. Typically this occurs as a result of neurological disease or pharmacologic supression of nerve function. (2) Colloquial for anesthesiology.

Anesthesiologist A physician specializing in the delivery of anesthetic agents or study of anesthesiology and related areas.
Anesthesiology The study of anesthesia and related fields.
Angina Chest pain, often crushing and just behind the breastbone, due to an inadequate oxygen supply to the heart muscle. Patients tend to complain of pressure and a feeling of suffocation rather than sharp pain.
Angiogenesis Establishment of new blood vessels from preexisting ones. Also called neovascularization. Preventing angiogenesis is being investigated as a treatment for cancer.
Angioplasty A procedure during which a balloon-tipped catheter is passed through specific blood vessels to a coronary artery (or arteries) such that its inflation alleviates the artery's narrowing . Also known as Percutaneous Transluminal Coronary Angioplasty (PTCA).
Angiostatin EntreMed’s formulation of a proplasminogen cleavage product. Its anti-angiogenic properties, though poorly understood, have made it an investigational agent in several anti-cancer settings.
Angiotensinogen (AGT) Gene The AGT gene controls the production of angio-tensinogen, an enzymatic protein which is part of a regulation system that plays a part in, amongst other functions, regulating blood pressure. "
Anistreplase A purified thrombolytic (clot buster) also known as streptokinase, derived from a class of bacteria known as group C beta-hemolytic streptococci.
Ankylosing Spondylitis A chronic, debilitating disease characterized by progressive spinal fusion (ankylosis), arthritis,and physical deformity.
Antagonist A molecule that binds to and blocks the effect of another molecule. Antagonists are the opposite of agonists, which binds to and activate the target molecule.
Anthrax A serious bacterial infection caused by the bacterium Bacillus anthracis. Anthrax is the Greek word for burning coal, resembling the appearance of the infectious cutaneous (skin) lesion, a carbuncle (cluster of ulcerating boils) with a hard black center surrounded by bright red inflammation. Most human anthrax infection comes from skin contact with contaminated animal products. The pulmonary (lung) form of the disease is contracted by inhaling a large dose of spores, typically in an enclosed space protected from direct sunlight. Untreated pulmonary anthrax is usually fatal. Intestinal anthrax is caused by eating contaminated meat. "
Antibiotics A drug used to treat infection. Antibacterials are the most common sorts of antibiotics in the U.S. and western Europe, and often the two words are used interchangeably.
Antibody A protein produced by immune system B cells. Antibodies defend against invasion by foreign agents like viruses, bacteria or cancer cells. They work by binding to a marker called an antigen.
Antibody Dependent Toxicity Type of cell destruction in which specific cell surface markers of doomed cells are coated with antibody allowing, typically, natural killer cells to identify and help destroy the ill-fated cells. Antibody dependent toxicity is sometimes referred to as antibody-dependent cell-mediated cytotoxicity.
Anticholinergics Any of a group of agents designed to interfere with the activity of acetylcholine.
Anticoagulant An agent used to prevent blood clot formation
Antigen Any substance that can be recognized by the immune system. Antigens provoke the production of antibodies. Antigens include proteins and other molecules on the surface of bacteria, viruses, tumors or other foreign tissues.
Antigen Presenting Cells White blood cells, including macrophages and dendritic cells, which present bits of bacteria, viruses, and other foreign material to T cells.
Antioxidant Any substance that inhibits oxidation or reactions promoted by oxygen or peroxides (compounds in which oxygen is molecularly joined to oxygen). Cells naturally manufacture antioxidants that convert free radicals into harmless oxygen, water, and other chemicals. Oxidation can be thought of as either combining with oxygen, eliminating hydrogen, or both. Oxidation changes a compound by increasing the proportion of the electronegative part or change (an element or ion) from a lower to a higher positive valence — removing one or more electrons from an atom, ion, or molecule. Antioxidants interfere with this process, which causes interference with processes associated with specific types of inflammation and decay.
Antisense (1) An engineered oligonucleotide whose sequence is complementary to a specific mRNA, and thus has the ability to block protein production from that mRNA when introduced into cells. (2) The name of the strand of naturally occurring DNA that is subject to transcription— making mRNA. The other strand, the “sense” strand has the same sequence as the initially formed mRNA.
Aphthous Stomatitis A small, tender ulcer crater in the lining of the mouth (multiple causes). Commonly called a canker sore.
Aplastic Anemia Pancytopenia due to failure of the bone marrow to produce red blood cells, white blood cells, platelets, and other blood cells. Most often, a cause for the marrow failure is elusive. It can be caused by chemicals (benzene, toluene, insecticides, solvents), drugs (chemotherapy, gold, seizure medications, antibiotics, many others), viruses (HIV, Epstein-Barr), radiation, immune conditions (systemic lupus erythematosus, rheumatoid arthritis), pregnancy, paroxysmal nocturnal hemoglobinuria, and inherited disorders (Fanconi anemia and others). Contemporary drug therapy may include discontinuation of all bone marrow-suppressing medications and institution of androgens, marrow stimulating growth factors, and often prophylactic antibiotics. Blood transfusions may be frequent. Bone marrow transplantation may be in order plus or minus immunosuppression with antithymocyte globulin, steroids, and cyclosporine.
ApoE Apolipoprotein E, a protein that is a critical mediator of cholesterol and phospholipid transport between cells. Specific polymorphisms of its gene are associated with Alzheimer disease and coronary heart disease risk.
Apoptosis Programmed cell death through activation of an internally controlled, genetic suicide program.
Aptamers Pieces of RNA, DNA, or modified nucleic acids that can bind to specific proteins.
ARDS An acronym for the adult/acute respiratory distress syndrome, the dramatic “wet lung” manifestations of the diffuse capillary leak syndrome. This is a late-stage, often end-stage, manifestation of the sepsis spectrum during which patient's lungs fail due to fluid leaking into them accompanied by cellular damage. Patients are progressively more unable to both extract oxygen from inspired air and exhale carbon dioxide, and thus require mechanical ventilation.
Aromatase An enzyme complex involved in estrogen production. Aromatase catalyzes the conversion of testosterone (and other androgens) to estradiol (and other estrogens). Aromatase is located in estrogen-producing cells in the adrenal glands, ovaries, placenta, testicles, fat, and brain. Some breast cancers grow from estrogen stimulation. A class of anti-estrogen drugs called aromatase inhibitors can be used to treat these estrogen-dependent cancers. Aromatase inhibitors act to lower the level of the estrogen called estradiol.
Arthritis Joint inflammation. The most common, chronic forms are osteoarthritis (OA) and rheumatoid arthritis (RA). OA, or degenerative joint disease, involves mechanical erosion of cartilage between bones, whereas RA involves autoimmune cartilage destruction.
Ascites Abnormal fluid accumulation in the abdomal cavity, typically the result of severe liver disease, metastatic disease involving the abdominal cavity, or both. "
Asthma Condition characterized by inflammatory constriction or congestion of the bronchial tree, causing wheezing, coughing and difficult breathing. Also known as reactive airway disease.
Ataxia The inability to execute coordinated, voluntary movement. "
Ataxia-Telangiectasia A progressive, degenerative, multi-organ system disease that typically has its onset during the second year of life. The initial sign of the disorder is most often ataxia characterized by a wobbly walk and slurred speech. Soon after, tiny, red "spider" veins, telangiectasias, may appear in the corners of the eyes or on the surface of the ears and sun-exposed cheeks.
Atherosclerosis A process of progressive thickening and hardening of the walls of arteries owing to the deposition of, amongst other things, fat on their inner lining. Risk factors include high levels of ""bad"" cholesterol, low levels of ""good"" cholesterol, high blood pressure (hypertension), smoking, diabetes, and a family history of atherosclerotic disease especially of early onset. Atherosclerosis predisposes an individual to coronary artery disease (angina, heart attacks, and sudden death), peripheral artery disease, and strokes.
Atherosclerotic Plaque Patchy areas of atherosclerosis, typically lining blood vessel channels, that can progressively be occlusive, leading to cardiovascular disorders such as angina, unstable angina, acute myocardial infarction, sudden cardiac death, transient ischemic attack, reversible ischemic neurological deficit, stroke, peripheral vascular disease, claudication, peripheral arterial occlusion, and gangrene.
Atrial Fibrillation An abnormal, irregular heart rhythm characterized by chaotic electrical signals eminating from the atria of the heart. Also known as atrial fib or a-fib. "
Attention-Deficit Hyperactivity Disorder A condition characterized by an individual’s inability to control their own behavior or impulses. The condition has many potential manifestations, often the most prominent of which, at least in childhood, is hyperactivity. When hyperactivity is not present, the principle manifestation is often inattentiveness.
Autoimmune Disease One of many related illnesses resulting from a person's immune system attacking tissues in the person's own body (immune system reaction against self-antigens). Type I Diabetes, Rheumatoid Arthritis, Lupus, Crohn Disease, and Multiple Sclerosis are a few of many illnesses in which autoimmune reactions have been implicated. "
Autoimmune Response An immune reaction against self-antigens, which leads to damage of one’s own tissues. Autoimmune diseases include lupus, type I diabetes, and rheumatoid arthritis.
Autologous Regarding transfusions and transplants, when the donor and recipient are the same person or are genetically identical such as in the case of identical twins.
B Cell White blood cell involved in protecting the body from foreign invaders by making antibodies. Originating in bone marrow, B cells are transformed by interaction with antigens and become either a memory cell or a plasma cell. Fully mature B cells are called plasma cells and produce a single line of antibodies against a single antigen.
B Cell Lymphoma A cancer of the blood and lymph nodes derived from mutations of the B cell lymphocytes.
B Cell Receptor An antibody like molecule attached to the surface of a B cell that identifies an antigen and triggers a B cell immune response.
B Lymphocyte Stimulator (BlyS) A member of the TNF family of proteins, this small protein is secreted by macrophages and other antigen presenting cells, and stimulates B cell proliferation. BlyS was discovered and is being developed as a drug target by Human Genome Sciences.
B7 A protein present on B cells, dendritic cells, and other white blood cells. B7 binds to CD28 on T cells, which helps to trigger activiation of the specific T cells during an immune response.
Bacteria Single-celled microorganisms which can exist either as independent (free-living) organisms or as parasites (dependent upon another organism for life). The singular is "bacterium."
Basal Steady-state, often low, level essential for maintaining a fundamental vital activity of an organism. Ground state. "
Basal Ganglia Located at the base of the brain, this region is responsible for purposeful body movement and fine-tuned coordination. It is composed of 3 nerve cell clusters: the caudate nucleus, putamen, and globus pallidus. Dysfuction of this area is evident in Parkinson disease and related disorders.
Bcl-2 A protein that protects cells from apoptosis (cell suicide). Tumor cells often turn on expression of Bcl-2 to avoid cell death, and blocking activity of Bcl-2 is being explored as a cancer therapeutic. "
Bcl-X A protein similar to Bcl-2 involved in regulating apoptosis. Different forms of Bcl-X either protect cells from apoptosis or induce apoptosis.
BEMA System (Atrix) A polymer-based film technology for transmucosal delivery of drugs.
Beta-Blockers A class of drugs that block activity at badrenergic receptors, and therefore can be used against hypertension, cardiac dysrythmias, diastolic myocardial dysfunction, glaucoma, and other conditions.
bFGF Basic fibroblast growth factor, a pro-angiogenic molecule. "
Bioavailability The degree and rate at which a substance (i.e. a drug) is absorbed into a living system or is made available at the site of physiological activity.
Bioinformatics The use of computer technology to process, analyze, store, and retrieve biological data. Bioinformatics applies technology in information management and mathematics to process the avalanche of data produced in biological research, particularly in genomics and proteomics. An example of bioinformatics is the use of computers to analyze information gained from DNA microarray experiments. "
Biological License Application (BLA) Application to the Food and Drug Administration to begin marketing specific sorts of drugs known as “biologics” to the public. “Biologics,” (the FDA distinguishes from small molecule “drugs”) include (1) therapeutic DNA plasmid products; (2) therapeutic synthetic peptide products of 40 or fewer amino acids; (3) monoclonal antibody products for in vivo use; and (4) therapeutic recombinant DNA-derived products. "
Biological Pathway A cellular process serving a specific purpose such as energy production or insulin regulation. Scientists study these molecular sequences of events in order to understand problems such as tumor progression and target particular molecular processes to block with drugs.
Biopharmaceutical Company Company involved in research of new drugs as well as the manufacturing, marketing, and distribution of pharmaceutical products.
Biopsy Surgical procedure to remove all or part of a tumor for diagnostic tests. Biopsies are usually done to determine if cancer is present and, if so, what type. "
Biotechnology The industry whose companies discover, manufacture, develop, test, and market products that are made from, are degraded to, or otherwise utilize “biologic” agents. Increasingly, these agents are linked with discoveries and developments in genetics, molecular biology, biochemistry, or related fields of natural science.
Bi-Specific Monoclonal Antibody A chimeric monoclonal antibody whose two variable regions each recognize a different antigen.
Bisphosphonate One of several compounds that are analogues of pyrophosphate (P-O-P) in which oxygen is replaced with carbon (P-C-P). The first-generation products include etidronate, alendronate, and risedronate. They bind to specific crystals (hydroxyapatite) in bone, inhibiting bone resorption and stabilizing or increasing bone mineral density. These compounds have use for maintaining bone architecture in disorders characterized by bone demineralization and breakdown such as osteoporosis, hypercalcemia of malignancy, and bony disease related to multiple myeloma or solid tumor metastases.
Blast Crisis An accelerated phase of a leukemia during which a very high count of immature white blood cells, typically, are found in the bone marrow and peripheral blood. The transformation to this phase of the disease is typically characterized by multiple chromosomal abnormalities of the affected cells. The blast phase of the disease is extremely difficult to treat, and bleeding and infection may occur due to bone marrow failure. Known risk factors are exposure to ionizing radiation and benzene.
Blepharospasm Spasmodic contraction of the orbicularis occuli muscle, a muscle responsible for eyelid closure.
Blinding The concealment of treatment versus control group assignment. In a single-blind study, knowledge of group assignment is withheld from patients. For example, patients don’t know if they’re receiving the test drug or a placebo. In a double-blind study, such knowledge is withheld from not only the patients but also the researchers. When properly executed, blinding eliminates the opportunity for knowledge of assignment to influence patient response to intervention. Blinding can also eliminate the opportunity for investigator behaviors to influence outcomes.

Although straightforward, blinding is often difficult to achieve in real life. For example, if a therapeutic produces a swelling at the point of injection, physicians and sometimes patients may realize who is receiving treatment vs. placebo. Erbitux and Iressa produced a distinctive red face rash in many recipients, a mild effect which actually predicted positive therapeutic effect, but made it difficult to truly blind study subjects.

Blood Clot The coagulated phase of blood resulting from the conversion of fibrinogen to fibrin (a reaction catalyzed by the enzyme thrombin) and a resultant entrapment of red blood cells and some other cells and cell fragments.
Blood clotting factors Proteins whose role in the body is to tend to clot blood. Their function is in delicate balance with other proteins that have a tendency to prevent blood clots, destroy blood clots, or both"
Blood-CNS Barrier A network of capillaries, their distinct endothelial cell architecture, and surrounding non-neuronal cells (neuroglia), that limit the passages of certain substances between the blood and central nervous system (brain and spinal cord) tissue. Also known as the blood-brain barrier.
Bolus A large dose, whether physiologic or supplemental, such that the desired concentration of a substance is reached rapidly.
Bone Marrow The tissue located in the bones where blood is manufactured.
Bone Marrow Transplant A procedure in which a patient's bone marrow that was destroyed by chemotherapy and/or radiation therapy is replaced either with donated bone marrow or the patient's own marrow that has been collected and stored prior to chemotherapy and/or radiation therapy.
Bone Morphogenic Protein (BMP) Any of several proteins that are involved in initiating a cascade of molecular signals that lead to the production of proteins involved in bone and other specific tissue formation. Molecular signal propagation is assisted by members of a family of signal transducing proteins called the Smad proteins. The first therapeutic applications of BMPs has been in the field of orthopedic surgery, but pulmonary, vascular, and oncologic applications are virtually inevitable.
Botox Type A botulinum toxin, FDA-approved in 1989 for the treatment of strabismus and dystonia-associated blepharospasm, it has a major anti-wrinkle cosmetic role, and was approved for use in cervical dystonia in late 2000.
Botulinum Toxin (Type A / Type B) Two of seven alternative forms of the toxin, perhaps the most potent in nature, elaborated by the bacterium Clostridium botulinum. The purified toxin is the first bacterial toxin to be used as a medicine, currently used as a treatment for movement disorders such as cervical dystonia.
Bovine Derived from a cow, ox, bison, buffalo, or close relatives thereof.
BRACAnalysis Test produced by Myriad Genetics for a genetic predisposition to breast and ovarian cancer caused by mutation in the BRCA1 or BRCA2 genes.
Bradykinesia Slow movement
Brainstem The portion of the brain directly connected to the spinal cord. Its uppermost limit is a matter of debate, but its nerve cell masses coordinate specific aspects of many basic functions such as breathing, eye movement, and even aspects of consciousness itself.
Brand-Name Drugs Pharmaceuticals which are marketed using a brand name that is distinct from the scientific name of the drug. Many brand-name drugs are patented which prevents other companies from marketing copies of the specific drug.
BRCA1 and BRCA2 Genes The first breast and ovarian cancer susceptibility genes to be identified. Mutated forms of these genes are believed to be responsible for about half the cases of inherited breast cancer in women and an even greater proportion of those that occur in younger women. The BRCA1 and BRCA2 genes are tumor suppressor genes that normally act to produce proteins that suppress abnormal cell divisions. If these genes are mutated, the proteins may not function correctly, leading to uncontrolled cell divisions and cancer. Inherited mutations of BRCA1 and BRCA2 genes are responsible for about seven percent of all breast and ten percent of all ovarian cancers. While the general female population has a ten percent lifetime risk of breast cancer, women who test positive using the BRACAnalysis for BRCA1 or BRCA2 have a 56 to 86% risk lifetime risk of breast cancer and a 27 to 44% risk of ovarian cancer. "
Breakthrough Pain Pain that affects a patient between doses of a primary analgesic (pain medication).
Breast Cancer Cancer that begins in specific breast tissue. Most women have a 10% risk of developing breast cancer in their lifetime. It is the most common from of cancer in women and is the leading cause of cancer related death in the 15 to 54 age group. The American Cancer Society estimates that in the year 2000 there will be about 182,800 new cases in women in the United States and about 40,800 deaths. About 10 % of all breast cancers are associated with known genetic mutations. Women with mutations of the BRCA1 and BRCA2 genes have a 60% chance of developing breast cancer by age 70. "
Broad Spectrum Antibiotics Antibiotics with an ability to neutralize or kill an extensive array of types of microorganisms (especially relating to bacteria).
Bromotaxane A hydrophobic derivative of paclitaxel (Taxol) being developed by Liposome against ovarian and other cancers.
Bronchi Subdivisions of the trachea (windpipe) that transmit air to and from the lungs.
Bronchial Relating to the bronchi.
Bronchitis Inflammation of the mucous membranes of the bronchial tubes.
Bronchoconstriction Narrowing of airways.
Bronchodilator Any agent that opens or widens the bronchial passages, those branches of the trachea (windpipe) that eventuate into the lungs.
Buccal Inner cheek.
Bursa (1) The blood cell producing organ of birds, analogous in function to the bone marrow of mammals. (2) A closed, fluid-filled sac that provides a low-friction surface between tissues of the body.
Butorphanol A synthetically derived opioid analgesic (pain medication).
Calcium Channel Blockers Drugs that block specific calcium ion channels, and whose treatment effects include anithypertensive, anti-anginal, and anti-cardiac dysrythmias amongst others.
Cancer One of a group of disorders characterized by abnormal growth and proliferation of cells. Cancers are labeled according to the tissue in which they began to grow. All begin as a single, genetically dysregulated cell. In the United States it is estimated that 50% of men and 35% of women will develop cancer at some point in their lives. "
Capillaries The smallest blood vessels. Their walls are essentially semipermeable membranes facilitating the exchange of various substances, such as fluids, oxygen, and carbon dioxide, between the blood stream and tissues. They are the conduits for oxygenated blood distribution from arteries, via arterioles, to the tissues of the body as well as the conduit for deoxygenated blood extraction from the tissues for distribution back into, via venules, veins. They are a central component of the circulatory system, essentially between the arteries and the veins. "
Capillary Leak Syndrome A clinical situation characterized, at the cellular level, by increased permeability of the smallest blood vessels, capillaries. Such permeability leads to intermittent leakage of fluids contained in these vessels into areas not surrounded by vessels (the extravascular space). Low circulating volume (hypovolemia), an increased blood cell volume and hematocrit (hemoconcentration), weakness, swelling (edema), and organ (visceral) congestion are resulting manifestations. When the condition manifests in the lungs, it is called Acute or Adult Respiratory Distress Syndrome (ARDS). The inability to control this syndrome, often the consequence of sepsis or other causes of the systemic inflammatory response syndrome, puts the patient at risk for multi organ system failure due to ischemia and infarction, as well as death. "
Carbohydrate A molecule made up of various sugars. Carbohydrates can be attached to proteins or lipids and displayed on the cell surface, and come in a variety of different “flavors.” Carbohydrates can be antigens recognized by the immune system, and because tumor cells often produce unique carbohydrates, these antigens are potentially useful ones.
Carcinoembryonic Antigen (CEA) A protein found in many types of visceral cells and in the developing fetus. Its clinical utility to date has been as a “tumor marker,” a protein whose measurements can be followed in order to monitor disease course. Elevated CEA levels are most often associated with cancers of the colon and rectum. However, cancers of the pancreas, stomach, breast, lung, and certain types of thyroid and ovarian cancer can also elevate such levels, as can smoking, infection, inflammatory bowel disease, pancreatitis, cirrhosis of the liver, chemotherapy, and radiation therapy. Its presence on tumor cells makes it a suitable target for monoclonal antibodies.
Carcinoma Cancer originating in tissues lining or covering an organ.
Cardiarisk A test produced by Myriad Genetics for genetic susceptibility to a specific type of hypertension based on the presence of a SNP of the angiotensinogen (AGT) gene.
Cardiopulmonary Bypass A procedure, and the machine with which is performed, designed to relieve the physiologic burden of the heart and lungs during particularly demanding surgeries commonly referred to as “open heart surgery,” including CABG and heart or heart-lung transplants. Circulating blood returning to the heart though the venous circulation is diverted through a machine (pump-oxygenator, often referred to as the “heart-lung” machine) that does the work of both the heart (pump blood) and the lungs (supply the circulating red blood cells with oxygen while dissipating carbon dioxide) prior to returning the blood to the arterial circulation for tissue distribution.

As cardiopulmonary bypass removes the heart-lung unit from the patient’s circulatory system and replaces the unit’s functions, a surgical procedure necessitating that the heart either be stopped (cardioplegia) or undergo manipulation that diminishes or eliminates its functional capacity can be successfully performed. Surgical clamps are strategically placed on the venous and arterial sides of the unit in order to isolate it in a fashion that permits a surgeon perform a precise and timely operation.

Improvements in the procedure and its equipment have led to the routine successes of contemporary open heart surgery, but being “on bypass” or “on pump,” as it is referred, still has potential adversities such as neurologic changes including cognitive impairment, stroke, or seizures; kidney damage ranging from decreased urine output to complete renal failure; failure of lung re-expansion (atelectasis); systemic inflammatory response syndrome; severe anemia; and various blood clotting abnormalities.

Cardiotoxicity Toxic to the heart.
Cardiovascular Disease Disease of the heart and blood vessels. The four most common types of cardiovascular disease are high blood pressure, coronary heart disease (which includes heart attack and angina pectoris or chest pain), stroke and rheumatic fever/rheumatic heart disease. Other forms include arrhythmias (disorders of heart rhythm); diseases of the arteries, arterioles and capillaries (including arteriosclerosis and Kawasaki disease); bacterial endocarditis; cardiomyopathy; congenital cardiovascular defects; congestive heart failure; valvular heart disease; hypertensive disease; diseases of pulmonary circulation; diseases of veins and lymphatics and other diseases of the circulatory system.
Case-Control Study A retrospective, observational investigation in which researchers identify one group of patients with a specified outcome (cases) and another group without the specified outcome (controls). Researchers compare the histories of the cases with those of the controls in order to determine the extent to which each was exposed to an intervention of interest.
Caspase One of a group of enzymes that have a role in the promotion of apoptosis (genetically programmed cell death). Inhibition of such enzymes may have utility in combating cell and tissue damage in conditions such as myocardial infarction, stroke, inflammatory diseases, and neurodegenerative disease. Augmentation of such enzymes may have utility in combating proliferative conditions such as cancer.
Catalyst A substance that facilitates a chemical reaction without being used up in significant amounts. Enzymes act as catalysts in cells.
Cataract A clouding of the eye's lens. It can progress to impair normal vision. Causes include cortisone medication, trauma, diabetes, many other diseases, and aging. They will affect almost all people if they live long enough. The treatment of choice is surgical implantation of a new lens. Wearing sunglasses can help prevent cataracts.
Catecholamine One of several related molecules that act as neurotransmitters in the sympathetic nervous system such as epinephrine (adrenaline), norepinephrine, and dopa.
Catecholaminergic Nerve Cells Neurons that utilize catecholamines as neurotransmitters.
CD Markers Molecules present on the cell surface of white blood cells and platelets. The molecules are identified by interaction with monoclonal antibodies and are used to differentiate cell populations.
CD11 A group of adhesion molecules of the integrin family found on white blood cells, usually expressed with CD18.
CD11a The alpha chain of a cell surface adhesion molecule. When expressed with the beta chain, this adhesion molecule, a member of the integrin family, influences trafficking and interactions of white blood cells.
CD14 A protein on the surface of white blood cells that can be stimulated by various bacterial toxins thus initiating the inflammatory cascade responsible for sepsis spectrum.
CD147 A protein of unknown function expressed on all white blood cells, red blood cells, platelets, and endothelial (blood vessel inner lining) cells. Also known as the CBL antigen.
CD18 A group of adhesion molecules of the integrin family found on white blood cells, usually expressed with CD11 proteins.
CD20 Cell surface marker present on normal and abnormal pre B and mature B cells. CD20 is present on more than ninety percent of B cell non-Hodgkin lymphomas.
CD22 Cell surface marker present on mature B cells, it is also expressed in the cytoplasm of all B cells, and absent on the surface of T cells. It is weakly expressed on myeloid and acute lymphoblastic leukemias, and is strongly expressed on hairy cell leukemias. "
CD23 Cell surface marker present on a subpopulation of B-lymphocytes, it has been detected on cancerous cells from cases of B cell chronic lymphocytic leukemia and some cases of centroblastic/centrocytic lymphoma.
CD25 The receptor for IL-2, present on activated T and B cells as well as macrophages and other immune cells.
CD28 Cell surface marker present on a subpopulation of T cells, it is a critical mediator of activating these cells such that they participate in immune processes. Such participation requires its interaction with a molecule called B7-1 (CD80) on the surface of antigen presenting cells. "
CD3 The set of proteins associated with the T cell receptor involved with T cell signaling.
CD33 A cell surface marker present on certain white blood cells. It is enriched on certain blood stem cells and Adult Myelogenous Leukemia cells.
CD34 A protein found on the surface of blood stem cells and absent on most other blood cells, often used to purify blood stem cells.
CD4 Cell surface marker present on helper T cells.
CD40 A protein present on B cells and other immune cells which binds CD40 ligand (CD40L). On B cells, CD40 strengthens the activating signals that turn on immune responses.
CD40 Ligand A co-stimulatory molecule which binds to the CD40 cell surface marker present on B cells. The CD40 ligand / CD40 pair mediates B cell proliferation, antibody production, and immunoglobulin class switching.
CD5 Cell surface marker present on certain B cells.
CD56 A protein found on natural killer cells (a type of white blood cell) as well as some T cells. CD56 also has a role in neural cell adhesion.
CD80 Cell surface marker, also called B7-1, present on a subpopulation of antigen presenting cells. CD80 is a critical mediator of activating these cells such that they participate in immune processes. Such participation requires its interaction with CD28 on the surface of T cells.
Cell The basic unit of living organisms, it is the smallest component of a living entity capable of independent existence.
Cell division The portion of the life cycle of a cell during which physical partitioning yields cell replication. In humans, two cell division processes predominate: mitosis and meiosis. In mitosis, somatic (body) cells divide to form two daughter cells, each with the same chromosome complement as their parent cell. In meiosis, germ (sperm or egg) cell precursors divide such that functionally specialized reproductive cells have half the chromosome complement of the parent cell.
Cell Line Cells grown in the laboratory that were originally derived from a particular cell type. Cell lines, such as pancreatic, hepatic, or tumor, are used extensively to study biological processes.
Cell Membrane The semi-permeable lipid bilayer that surrounds cells. The cell membrane permits certain molecules (such as water) to enter the cell, but blocks large molecules such as proteins unless specific transporter proteins are present in the cell membrane.
Cell Surface Protein A protein located on the surface of cells which is, at least in part, exposed to the extracellular space. Such proteins are able to be targeted by monoclonal antibodies and recombinant protein therapeutics.
Cell Therapy Using cells as a therapeutic. Many cell therapies involve giving the patient their own cells which have been removed, modified, expanded, and then re-injected. Lymphocytes, monocytes, and stem cells are some of the cell types used in cell therapies.
Cellulitis Colloquially, a skin infection. Technically, a diffuse, especially subcutaneous connective tissue inflammatory process of infectious or non-infectious etiology.
Centara (Centocor) An anti-CD4 monoclonal antibody being studied for the treatment of multiple sclerosis.
Central Nervous System The brain and spinal cord
Central Pontine Myelinolysis A neurodegenerative disorder characterized by degeneration of white matter (myelin containing nerves) that primarily affects the part of the brainstem called the pons.
Cerebellum The posterior portion of the brain between the cerebrum and the brain stem, it plays a significant role in gross coordination. Dysfunction of this area is evident in Friedreich Ataxia and related disorders.
Cerebral Cortex The outermost portion of the cerebrum, it is made up of layers of nerve cells and their interconnecting nerve pathways. Much of this area of the brain is responsible for processes relating to thought, perception, and memory. Dysfuction of this area is evident in Alzheimer disease and related disorders.
Cerebrospinal Fluid Clear, watery liquid surrounding the brain and spinal cord, it protects the central nervous system from injury and cushions it from the surrounding bony structure.
Cerebrum The two halves, hemispheres, of the brain including the cerebral cortex and the basal ganglia"
Ceremides Complex lipids, they are breakdown products of cerebrosides produced by specific skin cells as they migrate towards its surface. They have good moisturizing properties. Their pathology is also implicated in events in neurodegeneration and demyelinization.
Cervical Dysplasia Typically microscopic changes from normal in cells lining the cervix. A sequence of such changes from mild to severe may herald cervical cancer. Diagnosis is made from the Papanicolau (PAP) smear. Risk factors include multiple sexual partners, early onset of sexual activity (before age 18), early childbearing (before age 16), and a history of a sexually transmitted disease (STD, such as chlamydia, genital warts gonorrhea, genital herpes, and HIV). In women with HIV, the speed of cervical dysplasia changes is accelerated. Also known as ""cervical intrapithelial neoplasia (CIN).”"
Cervical Dystonia Also known as spasmodic torticollis, it is a neurological movement disorder in which a person’s neck and shoulder muscles have contractions that force the head and neck into abnormal and sometimes painful positions making it difficult for some patients to function normally.
CHD1 Gene A gene that regulates the expression of genes involved in lipid metabolism.
Chemiluminescent Low-temperature emission of light due to a chemical reaction.
Chemogenomics Vertex's name for its structural genomics platform.
Chemokine A small protein similar to a hormone which is released by one cell to signal to another.
Chemosensitize To overcome resistance to the effects of chemotherapy. To make sensitive.
Chemotherapy (1) Treatment of disease by chemicals that have a direct effect on the disease-causing organism or disease cells. (2) Colloquial name for certain forms of cancer treatment.
Chinese Hamster Ovary Cells (CHO Cells) A laboratory cell line originally derived from hamster ovaries, used to produce recombinant proteins.
Cholesterol The most common steroid in the body, it is essential in the formation of many crucial compounds active in the body: bile acids (which aid in the digestion of fats), Vitamin D, progesterone, estrogens (estradiol, estrone, estriol), androgens (androsterone, testosterone), mineralocorticoid hormones (aldosterone, corticosterone), and glucocorticoid hormones (cortisol). It is also important in preserving normal architecture and function of the membranes that surround cells. In the bloodstream, cholesterol is carried in a particle called a “lipoprotein,” as it is encased in proteins. These aggregates vary in their densities depending upon their degree of metabolism. "
Choline (2-hydroxyethyl)trimethylammonium ion, it is most often found in the body as a neurotransmitter combined with acetate: acetylcholine, as a component of cell membranes combined with lecithin, phosphatidylcholine; in other conjugated forms; or free waiting to be made into these crucial, conjugated forms.
Cholinergic Relating to nerve cells or fibers that utilize acetylcholine as a neurotransmitter.
Cholinesterase Short for acetylcholinesterase. Often refers to the class of anti-Alzheimer drugs, ""(acetyl)cholinesterase inhibitors." More formally, an enzyme whose substrate is any ester of choline, not just one that is acetylated.
Chondrodysplasia Malformation of cartilage, typically abutting areas of bone associated with the normal progression of increased stature (""growth plates:"" epiphyses and metaphyses, as well as ""shafts:"" diaphyses, and associated with the spine). Short stature often results.
CHOP Chemotherapy A combination of cyclophosphamide, doxorubicin, vincristine, and prednisolone, a standard regimen many cancers. "
Chromosome Paired structures located in the nucleus of the vast majority of cells, each contains about 2 inches of wound-up DNA. Each normal human cell contains 46 chromosomes — 22 pairs of autosomes and two sex chromosomes. Each parent contributes one chromosome to each pair, so children get half of their chromosomes from their mothers and half from their fathers. Sperm and egg cells each contain 23 chromosomes — one of each pair and one sex chromosome. There is also a chromosome in the mitochondria, whose constituents and behavior are vastly different.
Chronic Lasting a long time; often progressing slowly but surely.
Chronic Obstructive Pulmonary Disease A disorder that persistently obstructs air flow to and from the lungs and its passages. The most common conditions of this category are chronic bronchitis and emphysema. The obstruction is generally permanent and progresses (becomes worse) over time. Tobacco smoking is a key cause. Also known as chronic obstructive lung disease (COLD).
Chronic Renal Failure (CRF) A gradual, progressive, irreversible loss of the ability of the kidneys to function: excrete wastes, concentrate urine, and conserve electrolytes. Progression may continue to end-stage renal disease (ESRD). The accumulation of fluid and waste products in the body is typically associated with azotemia (nitrogen waste products build up in the blood) and uremia (the state of renal failure-related poor health). Most organ systems are affected by chronic renal failure. The most common causes are diabetes mellitus, glomerulonephritis, polycystic kidney disease, hypertension, Alport syndrome, reflux nephropathy, obstructive uropathy, kidney stones and infection, and analgesic nephropathy. "
Chronic Renal Insufficiency (CRI) (1) Chronic renal failure not having progressed to end stage renal disease (2) That state, along a spectrum of clinical progression, thought neither to represent chronic renal failure nor normal kidney function, but leaning more so toward the former. "
Ciliary Neurotrophic Factor (CNF) Neurotrophin demonstrated to promote the differentiation and survival of various embryonic and more mature neuronal cell types. It is considered, by some, to be one of the IL-6 cytokine family because it acts through a receptor containing gp130. "
Circulatory Collapse Shock; a marked drop in a person's blood pressure and/or heart rate and/or heart pumping ability severe enough to put tissues at risk for malfunction and the individual at risk for death.
Cirrhosis A condition characterized by irreversible liver scarring. Alcohol and viral hepatitis B and C are among the myriad causes. Complications include mental confusion, coma, fluid accumulation (ascites), internal bleeding, and kidney failure. The goal of treatment is to arrest further liver damage and avoid the type of liver cancer known as hepatocellular carcinoma. "
Clinical Involving or depending upon direct observation of living patients or study subjects.
Clinical Trials Trials are designed to test effectiveness, tolerability, and safety of drugs or treatments. Clinical trials in the United States are conducted under the supervision of the Food and Drug Administration (FDA) and must be satisfactorily completed prior to the FDA granting license for the product to be brought to market. The trials seek to determine if the new drug more effective than what is currently being used, side effects, and the appropriate dosage. Randomized, double blind, controlled trials are the "gold standard" of clinical trials. Controlled testing involves comparing the outcomes associated with giving an experimental drug to one group with the outcomes associated with giving a placebo and/or another drug whose outcome profile has been well-studied to a control group. Randomized means that neither the subjects of a clinical trial nor those running the trial are allowed to choose which subjects will receive the drug being tested to prevent problems with bias. Double blind means that neither the subjects of a clinical trial nor those running the trial are allowed to know which subjects receive the drug being tested until the data has been collected. This is also to prevent problems with bias.
Clone (1) An individual developed from a single somatic (non-germ) cell from a parent, thus being an exact replica of that parent at the level of the DNA sequence. (2) A group of cells derived from a single ancestral cell such as in cancer. (3) DNA, RNA, another macromolecule, a cell, a tissue, or an organism that is a replica, at some genetic level, of one of its ancestors. "
C-myc A transcription factor involved in cell division and many other cellular processes.
Coagulation (1) The clotting of blood or the process by which blood clots. Coagulation is, essentially, initiated by blood cell fragments called platelets that produce a substance that combines with calcium to form thromboplastin. A complex cascade of biochemical reactions known as the clotting cascade use several blood clotting factors to set the stage for thromboplastin converting the protein prothrombin into thrombin. Thrombin converts the protein fibrinogen into fibrin. Fibrin, not soluble in water and therefore blood, contributes to the formation of a latticework of "fibrils," and in so doing causes the blood plasma to gel. Blood cells and plasma become enmeshed in this latticework to form a "clot." (2) To become more viscous or thickened into a mass such as with tissue coagulation by various means such as electrocoagulation, laser coagulation, or photocoagulation.
Coagulation Factor A protein in the plasma of the blood that mediates the clotting of blood
Coagulopathy Any of several known types of disturbances of blood coagulation.
Cognition The interrelated processes of knowing, thinking, learning, and judging. As a whole, also known as known as higher integrative functioning.
Colaris A test for genetic susceptibility to Hereditary Non-Polyposis Colorectal Cancer (HNPCC), a specific type of colon and endometrial cancer caused by mutations in the MSH2 and MLH1 genes.
Collagen The most important structural protein of skin, tendons, cartilage, bone, and connective tissue.
Common Variable Immunodeficiency (CVID) An immune disorder characterized by a lack of antibody producing B cells or plasma cells, low circulating levels of most or all immunoglobulin classes, and recurrent bacterial infections. Some degree of T cell dysfunction is present in up to 50% of the patients. Also known as hypogammaglobulinemia"
Comparative Genomic Hybridization A molecular analytic technique that uses any of several fluorescence in-situ hybridization (FISH) systems, or less often without fluorescence, to compare two DNA samples in terms of gene dosage or other genetic parameters. The technique has been especially useful in the study of chromosomal changes in cancer cells and cells of the products of conception (zygote, blastomere, embryo, fetus, placental membranes, etc.). The technique holds much promise to become a standard diagnostic, prognostic, and susceptibility characterizing tool.
Complement Dependent Cytotoxicity Type of cell destruction in which specific cell surface markers of doomed cells are coated with antibody allowing one of two processes to occur: 1) lysis 2) phagocytosis (engulfment by cells called macrophages, the garbage collectors of blood cells). This process requires complement, a group of proteins found in the serum.
Complement Proteins Part of the innate immune system, complement proteins are activated in a cascade of sequential protein cleavages that result in a complex of proteins that can perforate the surface of a target cell, causing that cell to die. Complement proteins, found in circulating blood, can be triggered by antibodies or other signals.
Complete Response A classical endpoint of both a clinical trial and a non-trial clinical experience, especially in oncology and rheumatology, it denotes achievement of a predetermined definition of “response (to therapy).” Definitions of response typically pertain to favorable parameters of, for instance, tumor size, tumor burden, plaque area, and other measurable clinical or biochemical markers or phenomena.
Condyloma Acuminatum Genital warts caused by specific versions of the human papillomaviruses (HPVs). It is the leading cause of abnormal Papanicolau (PAP) smears and pre-cancerous changes of the cervix.
Congestive Heart Failure The inability of the heart to pump enough blood to the meet the demands of the body’s tissues. Typically this occurs when the heart muscle is too weak and/or the resistance against the heart muscle’s pumping is too strong (as is the case in high blood pressure) and/or the valves of the heart fail to permit proper flow though its chambers or out to the body or lungs.
Conjugated Monoclonal Antibody A monoclonal antibody carrying a payload, such as radiation or chemotherapy.
Conjugation The process of chemically attaching two molecules together, such as a monoclonal antibody and another protein.
Conopeptides Small peptides isolated from the venom of predatory marine snails of the genus Conus.
Constant Region The half of the antibody molecule that interacts with the immune system but does not bind the antigen.
Conus Magus Fish-eating marine snail.
Cord blood Short for “umbilical cord blood,” it is the blood that remains in the umbilical cord and placenta following birth. Within this blood are stem cells that can be harvested and manipulated by various means in order to yield therapeutic cells, tissues, and even organs. The autologous transplantation of these cells, tissues, and organs offer the promise of significantly less rejection than many of the various cell, tissue, and organ-matching systems offered today. Cord blood is collected after the baby is born and the umbilical cord has been clamped and cut. The stem cells are immediately available for transplantation. First used in transplants in 1988, a variety of life-threatening diseases including leukemia, other cancers, and blood and immune disorders, are amenable to this sort of therapy.
Coronary Artery Bypass Grafting (CABG) A surgical procedure indicated for specific groups of patients with clinically important narrowings and blockages of the coronary arteries. Bypassing these stenoses and blockages creates new routes for increased blood flow to deliver oxygen and nutrients heart muscle (myocardium). The actual bypass graft is typically a vein from the leg (most often, the saphenous vein) or an inner chest wall artery (most often, the internal mammary artery). These grafts are the conduits for bypassing the lesions that lead to chest pain, myocardial infarction (heart attack), and sudden death.
Coronary Artery Disease Narrowing or blockage of one or more of the coronary arteries resulting in decreased blood supply to the heart (ischemia).
Corticobasal Degeneration A rare neurological disease in which portions of the cerebral cortex and basal ganglia degenerate for unknown reasons. It shares many features in common with Parkinson disease.
Corticosteroids Specific hormones of the adrenal cortex, or their bioengeneered equivalents, which have potent anti-inflammatory properties, making them pharmacologically useful agents.
Cosmetic Use Pertaining to the use of an agent for the appearance of an individual. Such use of an agent neither requires FDA-approval nor appears on a product label.
Cripto A receptor for the protein Nodal, Cripto is part of the TGFb family of proteins that signal cell division, differentiation, and activation. Cripto has been implicated in development as well as in tumor progression. It is overexpressed on some human breast, colorectal, gastric, and pancreatic carcinomas.
Crohn Disease A form of inflammatory bowel disease characterized by, amongst other things, chronic inflammation of the small and large intestine, but which can affect other parts of the digestive system as well. When inflammation is limited to the large intestine (colon) only, it is often referred to as Crohn colitis or granulomatous colitis. Involvement of the small intestine alone is Crohn enteritis. Most often the portion of the small intestine affected by this disorder is the last portion, the ileum (Crohn ileitis). Involvement of both the small and large intestine is called Crohn enterocolitis (or ileocolitis).
Crossing Over The exchange of genetic material between two paired chromosomes in meiosis. The result of crossing over is that the recombination of DNA occurs such that each individual (except for identical twins) is genetically unique. The actual event or its result is often called ""a crossover.""
Cross-Reactivity Binding of a monoclonal antibody or other protein to a second target. The second target is usually not anticipated. If an antibody is cross-reactive between two antigens, binding to the second target can cause adverse events.
Cruetzfeldt-Jakob Disease A rapidly progressive neurodegenerative disorder that is invariably fatal, it is caused by infectious, ill-formed proteins or protein fragments called prions. "
CTLA-4 A cell surface protein found on white blood cells involved in stimulating other white blood cells.
Cutaneous Relating to the skin.
Cyclin-Dependent Kinase 4 (CDK4) An enzyme involved in regulating cell growth and division, in a complex with a protein called cyclin D. CDK4 is often de-regulated in tumor cells, leading to uncontrolled growth. CDK4 is being developed as a drug target because blocking CDK4 activity may stop tumor growth. Similar anti-cancer drug targets include: (1) Natural blockers of CDK4 activity such as INK4 proteins (p15, p16, p18, and p19) and CIP/KIP proteins (p21, p27, and p57) and (2) Classical cell cycle regulators p53 and Rb.
Cyclooxygenase-2 (Cox-2) One of two well characterized forms of the enzyme cyclooxygenase, it is expressed in response to a multitude of inflammatory stimuli. Normally present at low circulating levels, COX-2 regulates prostaglandin production primarily within inflammatory cells. The inflammatory response is a natural part of healing and repairing, and is also known to produce pain. Aspirin and non-steroidal anti-inflammatory drugs (NSAIDs), by non-specific COX-1 and -2 inhibition, and subsequent inhibition of prostaglandin release, suppresses this inflammatory response. Agents such as Celebrex and Vioxx have the advantage of selectively inhibiting COX-2, thereby eliminating COX-1 inhibition-mediated adverse effects such as gastrointestinal (GI) ulcerations, perforations, or obstructions due to the loss of COX-1 mediated repair and maintenance of GI lining tissue.
Cyclophosphamide The generic name for Bristol-Myer Squibb’s Cytoxan, it is used as an anti-cancer agent in many specific circumstances.
Cyclosporine The generic name of Novartis’ Neoral and Sandimmune as well as Abbott’s Gengraf, it is indicated to suppress the immune system in order to prevent the rejection of transplanted organs in individuals receiving a kidney, liver, or heart. It is also indicated for immunosuppression in severe rheumatoid arthritis and psoriasis, and is prescribed, off-label, for various circumstances in which suppression of the immune system is desirable.
Cystic Fibrosis (CF) A single gene disorder which causes the body to produce thick, sticky mucus due to the faulty transport of salt within cells lining organs such as the lung, sinuses, and pancreas. This leads to coughing, wheezing, pneumonia, nutrient malabsorption, malnutrition, and susceptibility to infection. Most commonly found among Caucasians of Northern European descent.
Cytochrome p450 An enzyme found in the mitochondria of cells involved in energy generation and protein degradation. The primary clinical importance of the liver (hepatic) cytochrome p450 system is in its responsibility for a wide variety of drug metabolism reactions. An understanding of the system allows both prediction of drug interactions and understanding adverse effects related to too rapid or too slow a rate of drug compound breakdown by the system.
Cytokines Small proteins secreted by lymphocytes which initiate specific responses from nearby cells.
Cytoplasm The inside of cells is divided into the cytoplasm and the nucleus. The mitochondria and other oganelles are found in the cytoplasm.
Cytoplasmic Tail The portion of a cell membrane spanning protein that is inside the cell.
Cytotoxin A compound that is poisonous to cells. Many chemotherapies are cytotoxins.
Dalton A measure of molecular weight. One dalton is unofficially equivalent to 1/12 the mass of a carbon-12 atom.
D-Dimer One of a family of fibrin fragments which form and circulate in the bloodstream for several days after a thrombotic event. D-dimer fragments are released from clots by the action of the enzyme plasmin. D-dimer is normally produced as part of the wound healing process and abnormally when clots form at the wrong time and place as a result of an underlying disease. Thus, D-dimer levels become a clinical marker of the presence of undesireable thrombotic events. "
Decompensated Loss of the physiological ability to compensate for injury.
Deep Vein Thrombosis (DVT) Blood clots of the deep veins of the body, typically of the legs. Leg DVTs above the knee, especially, have a propensity to dislodge and travel (embolize) to the lung.
Dementia Mental deterioration often characterized by disorientation, memory impairment, judgment impairment, and intellectual decline.
Depression A disorder of mood often associated with extreme sadness and discouragement. Symptoms also may include disruption of sleeping and eating patterns and lack of energy.
Dermal Ulcers Eroding of the skin resulting in its concavity and depression below the level of surrounding tissue.
Dermatology The study of the skin.
Diabetes Mellitus Disorder characterized by high levels of glucose in the blood. Diabetes may be caused by a failure of the pancreas to produce sufficient insulin or, more commonly, by resistance of the body to the action of insulin.
Diabetic Neuropathy Pain, weakness, or lack of sensation in a body part that is associated with, typically poorly controlled, diabetes. Most often it affects the feet, legs, hands, or arms. Treatment can include physical therapy, medication, and, by far most importantly, improved aggressive treatment of the underlying diabetes.
Diagnostic Products Tests used by physicians to make a diagnosis by testing for particular diseases, conditions, or predispositions.
Dialysis The process of filtering the blood by passing it through a machine designed to replicate kidney filtering functions. The two most common types of dialysis are hemodialysis and peritoneal dialysis, each with advantages and disadvantages. Patients can often choose the type of long term dialysis that best matches their needs.
Differentiation 1. In developmental biology, the process by which cells mature to become progressively more specialized. Cellular specialization occurs at the expense of cellular fate potential. For example, stem cells can differentiate into liver cells, but normal liver cells tend not to mature further. 2 In oncology, relating to the degree of maturity (development) of the cancerous cells in a tumor. Differentiated tumor cells are most like normal cells, tending to grow, mature, and spread at a slower pace compared to poorly differentiated or undifferentiated tumor cells. The more undifferentiated a tumor cell, the more structurally and functionally abnormal they are, and the more likely they are to grow, develop, and metastasize uncontrollably.
Diffuse Capillary Leak Syndrome See capillary leak syndrome
Dimeric Consisting of two subunits, often referring to proteins or other biological molecules. The two subunits can be the same (homodimer) or different (heterodimer).
Direct Costs of Disease Management Costs attributable to a drug regimen, treatment of its related adverse effects, time spent by medical, paramedical, and hospital staff, procedural costs, laboratory usage costs, and other costs not considered indirect costs. Direct costs include fixed and variable costs of all resources consumed in the provision of considered interventions or their consequences.
Disease Pathway The set of proteins, and their regulators, whose activation or inactivation lead to a particular disease. By defining a disease pathway, scientists gain insight into what genes and proteins contribute to the disease and are therefore potential drug targets.
Distribution A pharmacokinetic property of a drug relating to the area(s) of the body in which it and its metabolites can be found after administration.
Diuresis An increased excretion rate of urine.
Divalproex Sodium The generic name of Abbott’s Depakote for seizure disorders; a.k.a. valproic acid.
DMARDs Acronym for disease-modifying anti-rheumatic drugs. Drugs that have anti-inflammatory properties and the capacity to slow rheumatoid arthritis disease progression.
DNA (Deoxyribonucleic Acid) A nucleic acid that constitutes the genetic material of all cellular organisms and DNA viruses. It is the main component of chromosomes. A molecule of DNA consists of two strands arranged in spiral ladder formation with side pieces composed of phosphate and deoxyribose units and the "rungs" comprised of nucleotide bases (adenine, cytosine, guanine, and thymine). The genetic data are encoded in the sequence of the nucleotides.
DNA Microarrays A platform for studying how large numbers of genes interact with each other and how a cell's regulatory network controls vast batteries of genes simultaneously. The method uses a robot to precisely apply tiny droplets containing functional DNA to glass slides. Researchers then attach fluorescent labels to DNA from the cell they are studying. The labeled probes are allowed to bind to complementary DNA strands on the slides. The slides are put into a scanning device that can measure the brightness of each fluorescent dot; brightness reveals how much of a specific DNA fragment is present, an indicator of how active it is.
DNA Sequencing Determining the exact order of the nucleotide base pairs (adenine, cytosine, guanine, or thymine) in a segment of DNA.
Dobutamine A chemical derivative of the naturally occurring amino acid derivative, dopamine, it is used in the treatment of heart failure and helps the heart pump blood more forcefully. It is marketed as Dobutrex by Eli Lilly and as a generic by Baxter.
Dolly the Sheep The first “cloned” organism. She was produced by somatic cell nuclear transfer (SCNT).
Dopamine A neurotransmitter used by particular cells to facilitate impulse transmission.
Dorsal Raphe Nucleus A group of brain cell that utilize various neurotransmitters, but is most well known for its nerves that utilize serotonin and its contribution to mechanisms mediating sleep and circadian rhythm.
Dose Limiting Toxicity Adverse effects severe enough to prevent administration of more treatment.
Dosing Frequency The rate at which an agent is administered. Optimized compliance and increased prescription writing tends to occur when agents are dosed less frequently/at greater intervals but maintain the same or better therapeutic effect.
Double Blind Implies that neither the investigator nor investigated know what intervention is being transacted in a clinical study (i.e. placebo vs. investigational compound, sham procedure vs. investigational procedure, etc.)"
Drug Delivery Relating to ways and means of delivering drugs including routes of administration (oral, intravenous, intrathecal, etc.) and facilitative modalities (pumps, polymers, pills, etc.). Optimized compliance and increased prescription writing tends to occur when agents are developed in ways to ease adverse and uncomfortable effects associated with delivery and systems thereof.
Drug Targets Molecules, parts of molecules, molecular processes, and constituents of molecular processes that can be affected by a drug. The best targets tend to have the greatest degrees of specificity.
DUREDAS (Élan) An acronym for Dual Release Drug Absorption System, it is Élan’s proprietary “bilayer” tableting technology specifically developed to provide for two different release rates or dual release of a drug from a single dosage form.
Dyskinesia Uncontrolled movements
Dyspnea Difficult or labored breathing; shortness of breath.
Dystonia A state of abnormal tone (firmness or tension) of a tissue. Most frequently used to denote abnormally increased muscle tone.
E. coli (1) Living as harmless bacteria found in the gut, E. coli has been the model bacterium of choice for over thirty years. Recombinant proteins were originally produced in E. coli, and many are still produced that way today. (2) When populating body systems other than the gut or when its genetic makeup is altered (naturally or by engineering) in specific ways, E. coli can cause numerous infectious diseases, most notably urinary tract infections, sepsis, and food poisoning .
E1A gene A multifunctional gene first isolated from adenovirus. Viruses use E1A to regulate the expression of several viral and host genes in infected cells. Expression of E1A limits the growth of tumor cells.
E2F A well-studied transcription factor that regulates many cellular processes.
EBITDA Earnings Before Interest, Taxes, Depreciation, and Amortization. EBITDA is used as a surrogate measure of a company's cash flow from operations.
Edema Tissue swelling resulting from excess water accumulation.
Efficacy Measures the power to produce, in a controlled setting such as a clinical trial, a stated effect typically attributable to a known physiologic phenomenon. It is important to derive measures of efficacy because, with appropriate statistical achievements, these data can approximate real-world effectiveness.
EFVDAS (Élan) An acronym for Effervescent Drug Absorption System, it is Élan’s proprietary effervescent drug delivery technology similar to the technology used in Alka-Seltzer.
Eldepryl Somerset’s Selegiline formulation, it is a monoamine oxidase B inhibitor which prevents the breakdown of dopamine in the brain, and is indicated, in combination with l-dopa/carbidopa, in the management of signs and symptoms of Parkinson disease.
Electroencephalogram A study the brain’s electrical activity in which scalp electrodes, attached to a machine, records the electrical impulses. Typically, the investigator is trying to determine if the brain waves represent normal activity or activity suggesting epilepsy or other disturbances of brain wave patterns. "
Electrophoresis The migration of particles in an electric field toward either of two electric poles (negative/anode or positive/cathode).
Eletriptan A later-generation triptan developed by Pfizer that has an improved treatment profile over sumitriptan, the prototypic 5-HT 1B/1D agonist, in the treatment of acute migraine.
Embolism Obstruction of a blood vessel by a, typically once mobile, blood clot or other substance. A blood clot or other substance travels through the bloodstream, lodges in a blood vessel and plugs it. Classical non blood clot substances include an air bubble, amniotic fluid, a fat globule, cholesterol crystals, a cluster of bacteria, chemicals (i.e. talc), and drugs (predominately illicit drugs). ""Embolus"" refers to the plug itself whereas ""embolism"" refers to the process by which this happens.
Embryo The organism from the time of implantation in the uterus to the end of the second month of gestation.
Endocytosis A process by which cells bring materials into themselves. The process involves pinching off a bit of the cell membrane which forms a boundary membrane of engulfed material.
Endometriosis A disorder in which ectopic endometrial tissue is found (cells of the uterine lining deposited outside of the uterus).
Endorphins A group of opioid (morphine-like), pain-suppressing chemicals produced by the body. They are considered analgesics (diminishing the perception of pain) as well as sedatives. They are manufactured in the brain, spinal cord, and many other parts of the body.
Endothelin A peptide secreted by endothelial and other cell types that has potent vasoconstricting effects. Endothelin-1, the most predominant of three known isoforms, is produced in response to shear stress, vasoconstrictors, growth factors, and other stimuli.
Endotoxin A toxin of internal origin, most notably the poisonous substance present in, especially gram negative, bacteria separable from the cell body only on its disintegration. One of the most studied initiating substances of the sepsis spectrum.
Endpoints Clinical trial measurements made in study subjects or biological samples to assess the safety, tolerability, efficacy, effectiveness, or other objectives. Endpoints are ranked in order of importance with primary being the most important and the one(s) for which the study is, especially, designed. Secondary and tertiary outcomes may also be sought.
Enema (1) Injection of material into the intestine by means of the anus. (2) Material for injection in this fashion.
Enkephalins A group of opioid (morphine-like), pain-suppressing chemicals produced by the body containing the 5 amino acid sequence, Tyrosine-Glycine-Glycine-Phenylalanine. They are considered analgesics (diminishing the perception of pain) as well as sedatives. They are manufactured in the brain, spinal cord, and many other parts of the body. "
Enkephalins A group of opioid (morphine-like), pain-suppressing chemicals produced by the body containing the 5 amino acid sequence, Tyrosine-Glycine-Glycine-Phenylalanine. They are considered analgesics (diminishing the perception of pain) as well as sedatives. They are manufactured in the brain, spinal cord, and many other parts of the body. "
Enterprise Value (EV) A measure of the market value of the operations of a company. EV is computed by taking the market value of all the outstanding shares of a company’s stock added to the value of the company’s interest bearing debt less cash and cash equivalents. Since the two external providers of capital for a company are its shareholders and debtholders, the market values of these two classes items provides the total value that the capital markets place on the company. Cash and cash equivalents are then subtracted to obtain a clearer measure of the value the market places on a company’s operations and to smooth out the effect of financing events such as IPO’s and secondary stock offerings.
Enzyme Protein that acts as a chemical catalyst within the body, increasing or decreasing the rate of a particular biochemical reaction. Most metabolic pathways in the body are controlled by a series of enzymes which each control one step in the pathway. "
Eosinophil A type of white blood cell whose tissue and blood levels often rise during an allergic reaction.
Epidemiology (1) The branch of medical science that deals with the population characteristics (e.g. incidence, prevalence), distribution, and control of disease in a population (2) The sum of the factors controlling the presence or absence of a disease, pathogen, or clinical outcome.
Epidermal Growth Factor (EGF) A skin cell growth factor known to be involved in angiogenesis.
Epidermal Growth Factor Receptors (EGFRs) Cell surface proteins that bind and are activated by EGF.
Epidural Catheter A very fine plastic tube placed through the skin into the spinal epidural space, and left in place for a defined period of time, typically less than 2 weeks, such that medication can be administered through it. Epidural means situated upon or administered outside of the dura mater, the outermost of the 3 meninges covering the spinal cord and brain. The typical medications administered in such a fashion are local anesthetics and/or narcotics for pain relief or prophylaxis. Form this comes the common colloquialism, ""getting an epidural." Epidural catheters are also used prognostically for trials of spinal medications before placement of permanent implanted ports or programmable pumps.
Epilepsy A seizure disorder characterized by episodic disturbances of brain function.
Erectile Dysfunction The consistent inability to attain and maintain an erection sufficient for sexual intercourse.
Ergotamine An alkaloid derived from ergots. An agent with great smooth muscle stimulation potency, it has significant therapeutic activity against vascular headaches including migraine.
Ergots Any of a series of alkaloids derived from rye seed residua having been transformed from fungal infection of rye grass. Specific isomers can induce uterine contraction, control bleeding, and contract blood vessels. Ergots are potent blood vessel constrictors that have been used against migraine, often in formulation with caffeine, for over a century.
ERK An intracellular signaling kinase that can promote various biological effects, including cell division. ERK proteins are related to MAP kinases.
Erythema Nodosum Leprosum (ENL) Tender, inflamed, subcutaneous nodules associated with leprosy, it is often accompanied by fever, swollen lymph nodes, and joint aches.
Erythropoetin (EPO) A hormone produced by the kidney, and to a much lesser extent, the liver, that promotes red blood cell development in the bone marrow.
Ester Any of a class of often fragrant compounds represented by the formula RCOOR´ and typically formed by the reaction between an acid and an alcohol with elimination of water.
Estradiol A hormone which is the phenolic alcohol C18H24O2 primarily secreted by the ovaries. It is the most potent of the naturally occurring estrogens, and can be administered in its natural or semi-synthetic esterified form especially to treat menopausal symptoms "
Estrogen A substance, natural or synthetic, that exerts biological effects characteristic of estradiol, estriol, or its naturally occurring relatives. Thought of as female sex hormones, they are made in the ovary, fetoplacental unit, testes, and the adrenal cortex. They are responsible for the development of the female secondary sex characteristics, and during the menstrual cycle they act on the female genitalia to produce an environment suitable for the fertilization, implantation, and nutrition of the early embryo.
European Agency for the Evaluation of Medicinal Products (EMEA) The government agency responsible for approving and regulating medicines for commercial distribution in the European Union. Analogous to the U.S.’s FDA.
Evidence Based Medicine (EBM) As defined by Sackett in 1996, EBM is the “conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients.” Evidence based health care occurs when decisions affecting patient care are considered in the context of the appraisal of all valid, relevant information. It has grown to incorporate, as “evidence-based (medical) practice,” the integration of clinical expertise with optimal clinical evidence from systematic research. EBM is currently evolving into an approach to health care practice and delivery in which the clinician (for individual patients) and administrative decision makers (for populations) are acutely aware of not only the research evidence in support of clinical practice, clinical trial, regulatory, epidemiologic, and other medical decisions, but also the strength of that evidence.
Ex Vivo Out of the body. Often refers to manipulations done to a patient’s cells after they’ve been removed. For example giving gene therapy to harvested blood cells before re-injection is ex vivo therapy.
Excretion A pharmacokinetic property of a drug relating to its elimination from the body. Also known as elimination.
Expanded Label Use The use of a drug in a way both approved by the FDA and permitted to be put on its label and advertised as its intended purpose. Indications added to the label identified as expanded label uses imply (1) that they were not on the label prior to (2) acceptance of and approval for a supplemental biologic license application (sBLA).
Expressed Sequence Tag (EST) A short DNA sequence, typically around 200 base pairs in length, derived from mRNA produced by a defined cell. mRNA corresponds to transcribed (expressed) sequences of DNA, so the EST sequence is a fragment of a gene. Efficient methods of generating and sequencing ESTs led to EST databases containing 60,000 or more ESTs in the 1990s, generated by public research efforts, Incyte, and Human Genome Sciences, and others. Besides corresponding to fragments of genes and containing information about the cell of origin, ESTs have no inherent information about the function of the corresponding gene.
Extracellular Matrix The substance(s) between cells of a given tissue or tissues.
Extracellular Protein A protein found outside of cells, either on the cell surface or secreted into the surrounding fluid.
Fabry Disease An X-linked single gene disorder resulting in deficiency of the lysosomal enzyme a-galactosidase A, involved in the biodegradation of lipids, thus causing lysosomal storage defects. "
Factor Xa An enzyme involved thrombin generation, factor Xa is necessary for clot formation from induced by both intrinsic and extrinsic signals. Activated factor Xa becomes an essential component of the prothrombinase complex (along with factor Va, prothrombin, phospholipid, and calcium). Since prothrombinase complex assembly is the penultimate step in thrombin generation, factor Xa affects both the amount of active thrombin generated and the amount of fibrin (clot) formed. Factor Xa inhibitors have helped to rejuvenate the anticoagulation marketplace.
Familial Mutation A mutation that is associated with a specific disease or trait in a family, the inheritance of which substantially increases the risk of specific disease or trait development.
FAST Rating System The Btech Investor system to rate the quality of drug targets as Fundamental, Ancillary, Superficial, or Trivial.
Fast Track Status A designation given by the FDA to drugs for which the FDA wishes to expedite the review process. Typically, this designation is given to treatments for serious or life-threatening conditions and that have the demonstrated potential to address an unmet medical need.
Fastmelt Proprietary, convenience-based drug delivery formulation designed to release a drug rapidly within the mouth where it dissolves into a drug solution and is then swallowed.
Febrile Feverish; having a fever.
Female Sexual Arousal Disorder Persistent or recurrent inability to attain, or maintain until sexual activity completion, an adequate lubrication-swelling response associated with sexual desire and excitement. Response includes vaginal lubrication and expansion as well as swelling of the clitoris and other external genitalia. This scenario must be accompanied by distress and/or interpersonal difficulty.
Fen-Phen Any of several combination drug regimens featuring the compounds fenfluramine and phentermine, or derivatives thereof. The most popular branded versions were Redux (dexfenfluramine) and Pondimin (fenfluramine) from Wyeth. Fenfluramine and dexfenfluramine were withdrawn from the U.S. market in 1997 after their off-label use in obesity management was associated with the development of heart valve abnormalities. Herbal fen-phen is typically a combination of extracts of Saint-John’s wort and ephedra derivatives such as ma huang.
Fentanyl A synthetic opiate with agonist activity at kappa and mu receptors, it is a mainstay in the treatment of chronic and/or intractable pain in those people who have experienced opiates and have stable pain, and are typically unable to take oral agents or are intolerant of other available opiates.
Ferritin A blood protein whose measurement can help determine the amount of iron stored in the body. Its concentration is most often measured in the serum, the component of blood most like water (as compared to [1] ""plasma"" which also contains red and white cells and platelets and [2] ""whole blood"" which, on top of plasma, also contains clotting and anti clotting factors and other proteins and substances).
Fetoplacental Barrier The physically elusive border of the circulations of a pregnant mother and the fetus she carries, who lies within a temporary organ joining them called the placenta. Drugs that remain on the maternal side of this barrier are usually safe for the fetus.
Fibrinolysis The breakdown of fibrin, the protein generated by thrombin's enzymatic conversion of fibrinogen.
Fibrinolytic Relating to the breakdown of fibrin, enzymatic in nature and by drugs in acute clotting syndromes such as myocardial infarction, stroke, or pulmonary embolism.
Florescent Protein A laboratory construction of a protein coupled to a florescent dye, used to track the location of the protein in cells.
FLT-3 A ligand-receptor system that plays a role in hematopoietic stem cell development.
Fluorescence in-situ hybridization (FISH) A molecular technique used to study chromosomes. Under the microscope, fluorescent tags glow under ultraviolet light to reveal hybridization of molecular probes to specific regions of the DNA that make up chromosomes. This technique is being increasingly leveraged in the molecular diagnostic lab. The most classic example is 3 copies of chromosome 21 being readily detected in a cell of a fetus with Down Syndrome. Increasingly more common is the detection of multiple copies of the Her-2 gene in a breast cancer cell, thus signifying the potential utility of Herceptin therapy.
Food and Drug Administration (FDA) The government agency responsible for approving and regulating food and drugs for commercial distribution in the United States.
Fragile X Syndrome The most common inherited cause of developmental delay / mental retardation, it affects approximately 1 in 2,000 males and 1 in 4,000 females worldwide. It is second only to Down syndrome as a cause of mental retardation. It is one of several neuropsychiatric conditions caused by an expanded set of repeated elements along the length of a chromosome (trinucleotide repeats). Trinucleotide repeat expansion in Fragile X is along a specific stretch of the X-chromosome.
Framingham Study (The Framingham Heart Study) A landmark, longitudinal, epidemiologic study begun in 1948. Approximately 12,000 Framingham, Massachusetts residents who had not yet developed overt symptoms of cardiovascular disease or suffered a heart attack or stroke were enrolled in the initial investigations designed to collect medical data. At that time, the cardiovascular disease death rates had been increasing steadily since the beginning of the 20th century and had reached epidemic proportion. However, little was known about the causes of cardiovascular disease. The study's objective was, and still is, to identify the common factors or characteristics that contribute to cardiovascular disease. The study is responsible for the identification of the major cardiovascular disease risk factors: high blood pressure, high blood cholesterol, smoking, obesity, diabetes, and physical inactivity as well as the related factors such as blood triglyceride and HDL cholesterol levels, age, gender, and psychosocial issues. More recently, a significant proportion of initial study subjects and their family members have also provided DNA samples. More than 1,000 published manuscripts have resulted from the study.
Free Cash Flow (FCF) A company’s cash flow available for payment to the providers of capital (stock and debt holders) and to be reinvested in the company.
Free Radicals A molecule or subset of a molecule that has at least one unpaired electron. Because they have a free electron, free radicals are typically short-lived, highly reactive molecular fragments. These fragments are often formed by the splitting of a molecular bond, and they are capable of initiating or mediating a wide variety of chemical reactions. They have been associated with many events mediated by oxidation such as adverse inflammation and aging.
Friedriech Ataxia The most common inherited type of ataxia, it is a degenerative CNS condition that affects balance, coordination, movement, and sensation.
Fully Human Antibody A monoclonal antibody derived from human genes. These antibodies avoid the immune reaction against antibodies derived from mouse genes.
Functional Genomics Applying genomic information to determine gene function, commonly using microarrays and model organisms. An important part of target validation.
Functional Proteomics Applying proteomic information to determine protein function
Fusion Protein A protein created in the laboratory by splicing together two or more gene segments. Fusion proteins often combine the functions of two proteins into one.
G Protein Coupled Receptor (GPCR) A large family of related cell surface proteins that associate with internal signaling molecules (G proteins). GPCRs are often pursued as drug targets, for example the beta adrenergic receptor is a GPCR target for beta blockers.
GABA Gamma-aminobutyric acid. It is the most important inhibitory neurotransmitter in the central nervous system (CNS). It and its receptors facilitate chloride entry into the interior of nerve cells thus inhibiting specific neurotransmitter release in the presence of positive voltage polarization pulses. Such inhibition is extremely common as GABA receptors can be found at 60 - 80% of CNS neurons. Subtypes of GABA receptors can be activated by specific mushroom toxins (GABA-A) as well as the antispasmodic amino acid drug baclofen (GABA-B). These drugs directly mimic the action of GABA at the receptor. Drugs that allosterically facilitate GABA receptors at several distinct sites can be used as sedatives and anxiolytics. These drugs bend the receptor open to indirectly facilitate GABA binding. Prime examples are benzodiazapines and alcohol.
Gadolinium The element, Gd , atomic number 64 and atomic weight 157.25 (3) g, that is used as an intravenous contrast agent, or “dye,” that, when imaged with magnetic resonance imaging (MRI), improves the definition of certain, especially pathologic, structures.
Gallium A rare metal whose principal form has an atomic weight of 69. Isotopes such as gallium 68 can be produced by cyclotrons and emits gamma rays. Citrated gallium 68 is used as a radiotracer to identify sites of inflammation and tumor tissue within the body.
Gangrene Death of tissue due to the loss of its blood supply often allowing bacteria to invade it and accelerate its decay. Gas gangrene is an acute emergency involving invasion of a deep penetrating wound by anaerobic bacteria (bacteria that survive with little or no oxygen) that generate gas and pus. Dry gangrene is a chronic consequence of the death of tissue due to vascular (blood vessel) insufficiency. It is unaccompanied by bacterial invasion, and over time tissue simply dries up and shrivels.
Gastrointestinal Related to the stomach, intestines, mouth, and other organs involved in food digestion.
Gaucher disease A single gene disorder in which glucocerebroside accumulates in the lysosomes of various cells. Often leads to enlarged spleen, anemia, bone fractures, and can lead to death from bone marrow failure.
Gene The functional and physical unit of heredity passed from parent to offspring. Genes are segments of DNA and most genes contain the information for making a specific protein.
Gene Chip Affymetrix brand name for its DNA microarrays.
Gene Delivery Transport of therapeutic genes to patients.
Gene Discovery The identification of genes.
Gene Expression A gene is expressed when its sequence is transcribed into RNA. Most frequently, that RNA is an mRNA which is later translated into protein. Although all genes of an organism are present in each cell, not all genes are active in each. Some genes are activated only in certain tissues; others are activated only in response to certain environmental stressors. Studies of gene expression patterns are a source of information about gene function.
Gene Expression Data Data generated by microarrays which measure the amount of RNA being made from thousands of individual genes in a particular sample.
Gene Splicing The technique of manipulating DNA sequences in the laboratory. Scientists can synthesize and manipulate DNA to create new genes, usually from splicing different parts of natural genes together in new ways. Many biotechnology processes involve gene splicing techniques, for example the development of recombinant proteins and humanized monoclonal antibodies.
Gene Therapy Treatment of a disease or condition by adding to or replacing a patient’s DNA.
Genealogical Databases Databases which contain family histories, often including medical histories and pedigree analyses. These databases can be used in combination with patient DNA samples to identify genes whose mutations cause genetic predisposition to disease. "
Generic Drugs Pharmaceuticals which are bioequivalent copies of marketed brand-named drugs. Generic drugs typically are introduced by companies which specialize in generic drugs after the patents protecting brand-name drugs expire. Generic drugs are usually significantly less expensive than their branded equivalents.
Genetic Counseling An educational counseling process for individuals and families who have a genetic disease or who are at risk for such a disease. Genetic counseling provides patients with information about their condition and its risks, helping them make informed decisions.
Genetic Counselor A master’s degree-level (at least) health care provider whose profession it is to provide genetic counseling.
Genetic Database A database containing DNA sequence information derived from any of several sources.
Genetic Engineering Using recombinant DNA technology.
Genetic Marker A polymorphic sequence of DNA with an identifiable physical location on a chromosome and whose inheritance can be followed. A marker can be a gene, or it can be some section of DNA with no known function. Because DNA segments that lie near each other on a chromosome tend to be inherited together, markers are often used as indirect ways of tracking the inheritance pattern of a gene that has not yet been identified, but whose approximate location is known. Markers can also be exploited for mapping.
Genetic Screening Testing a population group to identify a subset of individuals at high risk for having or transmitting a specific genetic disorder.
Genetic Variation Differences in genetic composition at any given level: total genome, sub-genomic, chromosomal, genic/allelic, or nucleotide. Most often, the biotechnology industry uses differences at the level of the DNA sequence, specifically at the level of the allele or single nucleotide. Variation at these levels gives rise to polymorphisms, DNA spellings along analogous stretches (loci) of DNA, the differences of which may be associated with disease, freedom from disease, drug response, lack of drug response, adverse effects, and other human traits. The origins of genetic variation are direct consequences of sexual reproduction: (1) mutations (altering DNA by changing base pairs), (2) segregation (random distribution of one of each chromosome pair into sperm or egg), and (3) recombination (an event in sperm and egg cell development [meiosis] during which specific DNA is “shuffled”). These processes make each sexually-produced individual unique and provide the basis of genetic variation.
Genetics The science of biological inheritance.
Genome All the DNA contained in an organism or a cell, which includes both the chromosomes within the nucleus and the DNA in mitochondria.
Genomics Study of the genome, the complete complement of DNA in an organism.
Germ-Line Mutation A change in the DNA of the sperm or egg that therefore has a chance to be passed on to offspring.
Glaucoma Eye condition in which fluid pressure inside the eyes is increased due to impaired fluid drainage from the eye. Untreated, the optic nerve and other parts of the eye are at risk of impairment and loss of vision or even blindness can ensue. Common varieties include open-angle (the common adult-onset type of glaucoma) and acute angle-closure (less common, but type that can rapidly impair vision). Treatment may include medication, surgery, or laser surgery.
Glial Cell A type of support cell for neurons found in the brain.
Glial Derived Neurotrophic Factor (GDNF) A member of the transforming growth factor-beta protein superfamily, it is a potent neurotrophic factor that enhances survival of midbrain neurons that manufacture dopamine. Since its write-up in Science in 1993, GDNF has been widely explored in preclinical investigation relating to the management of Parkinson disease. In Parkinson disease, dopamine-producing neurons fail to provide dopamine in adequate amounts needed to preserve motor accuracy.
Glial Growth Factor A small hormone-like protein which induces growth of glial cells, a type of support cell for neurons found in the brain.
Glioma A general category of brain cancers that includes astrocytomas, oligodendrogliomas, and ependymomas.
Glucagon-Like Peptide 1 (GLP-1) Secreted from the L-cells of the gastrointestinal tract, GLP-1 has potent effects on glucose-dependent insulin secretion, insulin gene expression, and pancreatic islet cell formation. GLP-1 augments insulin secretion; decelerates gastric emptying; promotes satiety; suppresses glucagon secretion from pancreatic alpha-cells; facilitates insulin sensitivity in peripheral tissues; upregulates expression of pancreatic beta-cell genes GLUT2, glucokinase, insulin, and PDX-1; and promotes pancreatic beta-cell integrity. Recombinant versions of the peptide have pharmacologic properties that may make them suitable for energy-related metabolic disorders as well as disorders affecting a variety of organ systems.
Glucose A simple sugar that is a main energy source for metabolism. Blood glucose levels are tightly regulated by the hormones insulin and glucagon.
Glucose Tolerance The ability of an organism to properly uptake, utilize, and metabolise glucose. At the cellular level, the hormones insulin and glucagon are the principle mediators of these related processes. At the clinical level, impaired glucose tolerance is manifest by and is defined by high blood glucose levels after a standard test. At lower levels, impaired glucose tolerance is a precursor to diabetes mellitus. At higher levels, it is a characteristic of diabetes mellitus.
Glutamate A salt or ester of glutamic acid (the amino acid HOOC-CH2-CH2-CH(NH2)-COOH), it is an excitatory neurotransmitter. Overactivity in Alzheimer disease is the basis for therapies that modify its activity.
Glutathione S-Transferase Any of several related DNA protective enzymes of particular importance in specific biochemical detoxification pathways including those related to free radical scavenging systems. Without the activity of glutathione S-transferase, toxic products can promote mutagenesis and tumor formation. As variations of the protein can exist for cancerous, precancerous, or unaffected cells, modulating its effects in different systems can have varying results.
Glycosaminoglycan Any of a group of polysaccharides containing amino sugars (e.g. glucosamine), they include hyaluronic acid, keratan sulfate, chondroitin sulfate, dermatan sulfate, and heparin sulfate. Their three-dimensional structures enable them to trap water, which forms a gel giving glycosaminoglycans (GAGs) elastic properties. These elastic properties are most notable in the GAG-containing buffering tissues of the body such as cartilage. GAGs are also known as mucopolysaccharides (MPSs), and their accumulation in the lysosomes of the cells of various tissues is a feature of the single gene disordered inborn errors of metabolism known as mucopolysaccharidoses.
Glycosylation Relating to the state of having attached carbohydrates or the process of attaching them.
gpIIb/IIIa Receptor Short for "glycoprotein IIb/IIIa receptor complex," it is the platelet's cell surface receptor complex that interacts with the blood clotting protein, fibrinogen, in order to facilitate platelet aggregation and blood clotting/coagulation. Its hereditary absence leads to the bleeding disorder "Glanzmann thrombasthenia," and interference with its biological function, by drugs, can be used for therapeutic anticoagulation.
Grade A tumor classification scheme based upon the microscopic (histologic) appearance of tumor cells. The spectrum extends from low grade in which tumor cells resemble normal cells of the same type to the highest grade where tumor cells bear virtually no resemblance at all to their normal counterparts. Grades in between are typically numbered or lettered in ascending order of severity, and pathologists set nomenclature with regard to correlating the degree to which the cells differ from normal. "
Graft vs. Host Disease (GVHD) Condition that occurs after bone marrow or other blood cell related transplants when the donor’s immune cells manufacture antibodies against host tissues. Patients try to receive closely genetically matched bone marrow to reduce the risk of serious GVHD.
Gram Negative Bacteria that lose the crystal violet stain (thus appearing the color of the red counterstain) in J.M.C. Gram's staining method. The organisms that stain this way, gram-negative bacteria, have a cell wall composed of a thin layer of a material called peptidoglycan. Since the early 1900s Gram's staining has aided in distinguishing between different types of bacteria. A specific antibiotic has a tendency to work better for either gram-negative or gram-positive bacteria or subsets of either, rarely for both. Gram-negative bacteria include most of the bacteria normally found in the gastrointestinal tract as well as gonococci (veneral disease) and meningococci (bacterial meningitis). The organisms responsible for cholera and bubonic plague are also gram-negative bacteria.
Gram Positive Bacteria that retain the color of the crystal violet stain in in J.M.C. Gram's staining method. The organisms that stain this way, gram-positive bacteria, have a cell wall composed of a thick layer of a material called peptidoglycan. Since the early 1900s Gram's staining, has aided in distinguishing between different types of bacteria. A specific antibiotic has a tendency to work better for either gram-negative or gram-positive bacteria or subsets of either, rarely for both. Gram-positive bacteria include staphylococci ("staph"), streptococci ("strep"), and pneumococci, as well as the bacteria responsible for anthrax and diptheria.
Granulocyte Colony Stimulating Factor (GCSF) (1) A growth factor (small hormone like signaling protein) secreted by certain white blood cells which stimulates growth of other white blood cells. (2) Generic name of Amgen’s Filgrastim formulation, Neupogen, indicated in many specific circumstances in which a patient has a low white blood cell count.
Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) A growth factor (small hormone like signaling protein) secreted by certain white blood cells which stimulates growth of other white blood cells.
Growth Factor Any of an array of molecules that are known to mediate cell growth, often also associated with cell differentiation, locomotion, and contractility. Many are involved in essential developmental processes, fundamental cell and tissue repair processes, and wound healing. Growth factors are peptides, similar in structure to hormones.
Growth Hormone Made in the pituitary gland, it stimulates release of a liver hormone called somatomedin which, by several mechanisms, stimulates growth. Its many roles include stimulating protein synthesis, stimulating mobilization of free fatty acids, and inhibiting glucose utilization. Also known as somatotropin and somatropin.
Hairy Cell Leukemia A rare version of chronic leukemia in which abnormal white blood cells, under the microscope, appear to be covered with tiny hairs. "
Haplotype (1) A set of genes that code for different proteins but are in close enough physical proximity along a chromosome that they tend to be inherited as a unit. An individual’s gene sequence with respect to one member of a pair of allelic gene sequences; two people are of the same haplotype (but different genotype) if alike with respect to one allele of a pair but different with respect to the other allele of a pair. (2) A series of DNA sequences that are in close enough physical proximity along a chromosome that they tend to be inherited as a unit (3) The phenotype(s) determined by (1) or (2). Derived from haploid genotype.
HbA1c Short for hemoglobin A1c, it is a form of hemoglobin that has glucose attached to it. Because the glucose tends to remain attached for the life of the cell, approximately 4 months, measurement of HbA1c in the blood tends to reflect a person's average blood glucose level for that period of time. Thus, it is the classical test measurement used to follow the effectiveness of diabetes mellitus control. The recommended frequency of measurement is 3 to 6 months. Normal HbA1c is less than 7%; diabetics rarely achieve this target. Above 9% is considered poor control, and above 12% is extremely poor control.
HDL Short for “high density lipoprotein,” HDL cholesterol is the “good” cholesterol, as elevated HDL levels are associated with a decreased risk of coronary heart disease. See “cholesterol” entry.
Head and Neck Cancer (1) Colloquialism for squamous cell carcinomas arising from structures of the head and/or neck (2) Any malignant neoplasm arising in the head and/or neck
Health-Related Quality of Life (HRQOL) The degree to which aspects of optimal quality of life (QOL) are related to optimal health (typically mental, perceived physical, social, sexual, and role functioning). As a goal of much of health care is stated to be improved QOL, a significant challenge becomes its measurement such that (1) improvement can be identified and (2) cost-benefit and cost-utility analyses can be conducted. On an individual level, HRQOL is determined from standardized, questionnaire-derived measurements of an individual’s cognitive and affective response to their health status and, among other things, positively correlates with increased compliance with prescribed drug regimens. Such compliance is known to increase both prescription filling and sales. On a population level HRQOL, often measured in part by quality adjusted life years (QALYs) is often used in analyses of cost-benefit or cost-utility. A QALY is a year of life adjusted for its quality or value. A year of perfect health is equal to 1.0 QALY. The value of a year in ill health is discounted depending upon its severity.
Hedgehog A family of proteins that signals cells, best characterized in the nervous system, to progress down specific developmental pathways. Members of the hedgehog family include sonic hedgehog, patched, smoothened, and the cubitus interruptus / Gli family of transcription factors. Sonic hedgehog was originally isolated as a vertebrate homologue to the fruit fly protein hedgehog. Hedgehog proteins are hyperactive in certain proliferative disorders of both the central nervous system (CNS) (such as medulloblastoma) and periphery (such as various tumors) and are underactive in certain neurodegenerative disorders or CNS developmental anomalies such as holoprosencephaly (the complete or partial absence of a forebrain and related structures). Interfering with hedgehog activity has therapeutic potential in proliferative disorders whereas augmenting its activity has therapeutic potential in degenerative and developmental disorders.
Hematology The study of the blood and the organs that form its components.
Hemodynamics (1) The branch of physiology that deals with blood circulation. (2) The forces or mechanisms involved in blood circulation.
Hemoglobin The oxygen-carrying pigment and primary protein of red blood cells.
Hemophilia (1) An X-linked single gene bleeding disorder. Blood does not clot efficiently and abnormal bleeding occurs. Hemophilia A is caused by lack of blood clotting factor VIII while hemophilia B is caused by lack of factor IX. (2) Acquired hemophilia is due to a molecular inhibitor of either of these factors.
Hemorrhage Bleeding.
Heparin A type of anticoagulant (anti-clotting) medication. The natural mucopolysaccharide found most prominently in the liver.
Hepatectomy Surgical removal of the liver.
Hepatitis B A specific type of infectious inflammation of the liver caused by the Hepatitis B Virus.
Hepatitis C Liver inflammation caused by the Hepatitis C Virus (HCV).
Hepatocellular carcinoma The most common sort of liver cancer.
HER2 Gene A gene overexpressed in approximately 30% of metastatic breast cancers. Its overexpression is associated with poor clinical outcomes such as metastasis, resistance to chemotherapy, and short survival. This gene also tends to be overexpressed in a substantial proportion of patients with poor-outcome ovarian cancer and non small cell lung cancer. Its expression in normal, healthy adult tissue is low. The gene encodes a specific receptor in the epidermal growth factor receptor (EGFR) family, a cadre of receptors known to be overexpressed in cancers of the head and neck, kidney, bladder, prostate, and colon.
Herceptin Genentech’s monoclonal antibody formulation directed against the HER 2 gene, for the treatment of breast cancer.
Herzyme (Élan / Ribosome) Developmental ribozyme formulation for the treatment of metastatic breast cancers where the gene HER2 is overexpressed. The ribozyme recognizes, binds, and can enzymatically digest specific mRNA sequences.
Hippocampus A deep forebrain structure that helps regulate emotion and memory.
Histamine A substance with a fundamental role in the biochemistry of allergy. It dilates blood vessels and makes blood vessel walls leaky. Pharmacologically antagonizing its effects with "antihistamines" tends to provide relief from many allergic symptoms.
Histones A group of proteins found in the nucleus of every cell. DNA wraps around histones, which allows compression of the DNA into the small space available in the nucleus. Histones have several other functions such as regulating gene expression.
HIV (Human Immunodeficiency Virus) The retrovirus that causes AIDS (Acquired Immunodeficiency Syndrome).
HLA-DR Antigen A major histocompatibility complex (MHC) protein. MHC proteins present antigens to T cells. Their sequence varies widely between individuals, making them key factors in graft rejection.
HMG CoA Reductase Abbreviated name of 3-Hydroxy-3-methylgluatryl coenzyme A (CoA) reductase (HMGR). This enzyme functions in cholesterol metabolism to catalyze the conversion of HMG-CoA into CoA and mevalonate, the rate limiting step of cholesterol synthesis. Drugs that inhibit this chemical reaction, “statins,” have been effective in lowering blood cholesterol levels to provide cardiovascular event risk reduction.
Homocysteine A type of amino acid typically formed in the body as a byproduct of meat consumption. Homocysteine can damage blood vessels by injuring their lining cells and by stimulating the growth of smooth muscle cells. Elevated blood levels of homocysteine have been associated with elevated risks of coardiovascular disease; cardiovascular events including heart attacks, strokes, and peripheral vascular disease; venous thromboembolism; Alzheimer disease; and poor obstetrical outcomes. The most studied ways to decrease blood levels of homocysteine are eating less meat and taking folate, B6, and B12 vitamin supplements. Though homocysteine was found to be a cardiovascular risk factor prior to cholesterol being found to be one, scientific and clinical attention has only recently focused on this crucial amino acid.
Hormone A substance secreted in the body and carried through the bloodstream to various tissues, where it serves a regulatory function.
Hormone Replacement Therapy (HRT) (1) Combination therapy of estrogen plus a progestogen. Estrogen replacement therapy (ERT) treats menopause by minimizing its short-term changes such as hot flashes, disturbed sleep, and vaginal dryness. ERT can also prevent osteoporosis when it is associated with low, circulating estrogen levels, as well as reduce the risk of heart attacks or sudden death by up to 50%. Vaginal ERT can alleviate vaginal dryness, more severe vaginal changes, and bladder effects. When ERT is unopposed (used without a progetogen), it is associated with an increased risk of endometrial cancer (cancer of the lining of the uterus). Progestogen significantly reduces this risk. (2) Replacing any hormone of low circulating levels.
HPC2 Gene A gene whose mutations contribute to a susceptibility to familial prostate cancer.
H-ras A small signaling protein in the G protein family involved in intracellular signal transduction. Overactive H-ras activity is associated with tumor formation.
HuMab Mouse The proprietary mouse strain from Medarex that produces fully human antibodies.
Human Anti-Mouse Antibodies (HAMA) Antibodies produced in humans during an immune response against transplanted mouse proteins. HAMA responses are often seen when mouse monoclonals are used as therapeutics, and can lead to the immune rejection of the mouse monoclonal.
Human Genome Project Begun as the Human Genome Initiative, it is the collective name for several projects begun in 1986 by The United States Department of Energy (DOE). The goals of the Initiative were to (1) create an ordered set of DNA segments from known chromosomal locations, (2) develop new computational methods for analyzing genetic map and DNA sequence data, and (3) develop new techniques and instruments for detecting and analyzing DNA. The DOE initiative is presently known as the Human Genome Program, and the national combined effort, led by DOE and the National Institutes of Health (NIH), is known as the Human Genome Project. The United States Human Genome Project officially began October 1, 1990 as part of an international effort to determine the location and structure of the entire set of human genes. It continues in earnest, having a completed, published first draft in Feb., 2001, and with intermediate and final sequence milestones to be completed by June 2001, and no later than the year 2003, respectively.
Human Papilloma Virus A group of more than 60 viruses responsible for causing warts. Most produce warts on the hands, fingers, and face. However, the notorious, sexually-transmitted versions are confined primarily to the moist skin of the genitals, producing genital warts and increasing the risk for cervical cancer. "
Humanized A technology that assembles monoclonal antibodies by attaching several small pieces of the antibody derived from a mouse to the basic framework of a human antibody. The goal is to decrease the chance of an adverse immune response.
Huntington Disease A progressive neurological disorder caused by a mutation in a gene on chromosome 4 and characterized by abnormal body movements and early onset dementia. It is one of several neuropsychiatric conditions caused by an expanded set of repeated elements along the length of a chromosome (trinucleotide repeats). Trinucleotide repeat expansion in Huntington disease is along a specific stretch of chromosome 4.
Hybridoma An immortal cell with unique properties made in the laboratory from the fusion of a tumor cell and a normal cell.
Hyperglycemic (1) Relating to a state of hyperglycemia — excess of sugar in the blood. (2) An agent designed to raise blood sugar.
Hyperglycosylation Related to having abnormally abundant attached carbohydrates.
Hyperparathyroidism Excessive parathyroid hormone (PTH) production by the parathyroid glands. PTH regulates calcium and phosphorus balance in the body. The disorder can be of a primary parathyroid gland etiology or secondary to other primary pathologies. A tertiary form also exists.

Primary hyperparathyroidism involves excessive PTH production due to enlargement of one or more of the 4 tiny parathyroid glands located at the front and base of the neck. Effects of increased calcium manifest in several body systems including the skeletal, gastrointestinal, renal (kidney), muscular, cardiovascular, and central nervous system. Most commonly, primary hyperparathyroidism is seen in people over 60 years of age with women more likely to be affected than men. Radiation to the head and neck increases risk as does the rare parathyroid carcinoma.

Secondary hyperparathyroidism, from disorders associated with hypocalcemia (low levels of calcium in the blood), can arise in many settings and can also involve significant disorders of phosphate balance. Contributors can be disorders of vitamin D (such as osteomalacia [rickets], vitamin D deficiency, vitamin D malabsorption, and abnormal vitamin D metabolism induced by drugs), disorders of phosphate metabolism (such as kidney disease, malnutrition, malabsorption, aluminum toxicity, cancers, and phosphate depletion), calcium deficiency (not enough in the diet, too much lost in the urine), and especially chronic renal failure.

Chronic renal failure-induced secondary hyperparathyroidism is especially important and complex. It is a disorder in which phosphate clearance is impaired, phosphate is released from bone, vitamin D production is diminished or absent, intestinal calcium absorption is low, and blood levels of calcium are diminished. A feedback loop accentuates increased bone resorption (bone breaks down in an attempt to regulate abnormal levels of calcium, phosphate, and other entities) and hyperphosphatemia (high levels of phosphates in the blood) ensues which causes accelerated secondary hyperparathyroidism. Various therapies can contribute to high levels of calcium and phosphorous in the blood that can have deleterious effects on multiple organ systems.

Hypertension Persistently high arterial blood pressure, from one of a number of different mechanisms. Hypertension is a risk factor for the development of heart disease, peripheral vascular disease, stroke, and kidney disease.
Hypoglycemic (1) Relating to a state of hypoglycemia — deficiency of sugar in the blood. (2) An agent designed to lower blood sugar.
Hypoperfusion Underperfusion. Not enough blood, and therefore oxygen, getting to tissues or organs.
Hypotension Low blood pressure.
Idiopathic Thrombocytopenia Purpura (ITP) An autoimmune disorder in which an individual forms antibodies that destroy their own platelets. Its cause is unknown. Tiny red dots on the skin, called petechiae are often the initial bleeding manifestation. Progression to persistent nosebleeds and even gastrointestinal bleeding may occur. ""Idiopathic"" means that its cause is unknown (sometimes “immune” is substituted as this system plays an obvious role in its pathogenesis). ""Thrombocytopenic"" means that there are not enough circulating platelets. ""Purpura"" means excessive bruising.
IL-1 A cytokine (small signaling molecule) produced by blood cells that signals other cells to perform cellular functions. IL-1 can be cleaved into a peptide involved in cell death (apoptosis). IL stands for interleukin.
IL-12 A cytokine produced by immune cells which has various effects on many cell types.
IL-1b The beta chain of the IL-1 cytokine. IL-1b and IL-1a combine to form a cytokine produced by immune cells.
IL-2 A cytokine (small signaling molecule) produced by helper T cells that signals other cells to divide or perform other cellular functions. IL stands for interleukin.
IL-2R The cell surface protein that binds and is activated by IL-2.
IL-4 A cytokine (small signaling molecule) produced by white blood cells that signals other cells to divide or perform other cellular functions. IL-4 promotes allergic responses via production of IgE. IL stands for interleukin.
IL-6 A cytokine produced by immune cells which has various effects on many cell types, including inducing differentiation and activation.
IL-8 A cytokine (small signaling molecule) produced by white blood cells that signals other cells to divide or perform other cellular functions. IL stands for interleukin.
Imaging The action or process of producing an image especially by means other than visible light. In diagnostic medicine, common methods for imaging include x-ray, computed tomography (CT), magnetic resonance imaging (MRI), positron emmission tomography (PET), ultrasound, and various angiographic modalities. "
Immortal Cell A cell that can divide in culture indefinitely. Most mammalian cells die after a limited time out of the body.
Immune Mediated Thrombocytopenia Low platelet count in the peripheral circulation due to destruction of platelets and/or their precursors (such as megakaryocytes) by the immune system.
Immune System The network of white blood cells, lymph nodes, spleen, thymus, and related organs that distinguish between ""self"" and “not self,"" protecting the body from invasion from pathogens or foreign bodies. The immune system also defends against some cancer cells.
Immunogenicity The degree to which an antigen is recognized by the immune system. Because immunogenicity varies, it is easier to make monoclonal antibodies against some antigens than others. Immunogenicity also influences the effectiveness of vaccines and other therapies.
Immunoglobulin (Ig) Antibody.
Immunoglobulin E (IgE) Class of antibodies typically involved in allergies and asthma, among other processes.
In Vitro In the test tube. Often refers to biological experiments in the lab which later need to be verified in vivo.
In Vivo In the body. Often refers to experiments or treatments given directly to live subjects.
Incidence A term used in epidemiology indicating the number of new cases that occur in a given group of people (population) each year (or other specified period of time). Most often expressed as number of new cases per 100,000 populations per year. "
Incubator A laboratory device used to keep cells and tissues at a specific temperature and humidity condusive to growth.
INDAS (Élan) Acronym for Insoluble Drug Absorption System, it is proprietary technology designed to improve the solubility and absorption characteristics of poorly water soluble drugs.
Indirect Costs of Disease Management Costs attributable to work time lost and travel expenses incurred by patients and/or their friends and family in obtaining patient care.
Indium (In) The metallic element of atomic number 49 and atomic weight 114.82.
Individualized Medicine The goal of an individual care plan based upon knowledge of their genetic profile primarily derived from gene expression analyses and genotyping, and including both disease susceptibility analyses and response-to-treatment/medication profiling. "
Infarction The development of an infarct, a region of tissue death due to a local lack of oxygen.
Infection The growth and development of an organism that lives on or in another organism and draws its nourishment from its "host." When a human is infected by another organism, the organism derives its nourishment from the person. The presence of an organism on or in another is not sufficient for the determination of infection. For example, the normal growth and development of mouth, throat, intestinal, or vaginal organisms is not considered an infection, and even unusual organisms can coexist in or on humans "colonizing" them without infecting them. True infections are most often accompanied by signs of inflammation in people with intact immune systems.
Inflammation One of several fundamental pathologic processes, it is characterized by complex cellular and subcellular reactions occurring in affected blood vessels and affected tissues in response to abnormal stimulation including injury and trauma. Such stimulation can be from physical, chemical, or biologic agents and causes local reactions and structural changes, destruction or removal of the harmful agent, and responses that repair and heal. The so-called “cardinal signs” of inflammation are: rubor (redness), calor (heat), tumor (swelling), and dolor (pain).
Inflammatory Bowel Disease A group of chronic illnesses characterized by inflammation of the bowel (the small or large intestine). They can be limited to the intestine or associated with disease of the eyes, liver, gallbladder, skin, joints, and other organs. Crohn disease and Ulcerative colitis are the most common of these disorders.
Inflammatory Cascade The series of biochemical events that occurs when severe inflammation is left, whether intentionally or not, untreated. Many of these events are mediated by cytokines and clotting factors in the blood"
Inflammatory Disease Disease in which inflammation underlies the pathology.
Inositol Hexaphosphate (IHP) Also known as Phytate, Phytic Acid, or IP-6, it is a naturally occurring component of plant fiber for which evidence suggests its being a potent antioxidant. It is present in a wide variety of plant foods, especially wheat bran, whole grains, and legumes.
Inotrope Colloquial term for a substance or apparatus that increases the force of, especially cardiac, muscular contractions. Technically, these are "positive inotropes," whereas a substance or apparatus that decreases the force of muscular contractions is a "negative inotrope."
Insomnia Inability to sleep
Insulin A hormone made by the pancreas or taken by injection that regulates the amount of sugar (glucose) in the bloodstream.
Integrin A family of transmembrane proteins that serve as receptors for various components of the extracellular matrix.
Intensive Care Unit A hospital unit physically and identifiably separate from general routine patient care areas. These areas have a high density of special physiologic monitoring equipment and skilled personnel for the care of critically ill patients requiring immediate and concentrated continuous care, observation, and attention. There are several types of intensive care units, also known as ICUs. Coronary Care Units (CCUs) are ICUs staffed with nursing and supportive personnel specially trained in the care of patients with known and suspected coronary artery events. Medical ICUs (MICUs) are ICUs staffed with nursing and supportive personnel specially trained in the care of patients with general internal medical disease that requires intensive, supportive care. Surgical ICUs, respiratory ICUs, pediatric ICUs, neonatal ICUs, high-risk obstetrics units, and burn units are likewise staffed for the care of patients relevant to their disciplines.
Intensivist A physician specializing in the care of patients admitted to the ICU including neonatologists and high-risk obstetricians.
Intercellular Adhesion Molecule-1 (ICAM-1) One of a family of molecules, the cellular adhesion molecules (CAMs), that exist on the surface of virtually every cell in the body. It not only mediates the interaction between specific cell types but also transmits signals into the cells when activated. "
Interferons A family of cytokines (small hormone like proteins) secreted by white blood cells that influence growth and other functions of target cells.
Interleukins see Cytokines.
Intervention The act or process of intervening (coming in-between). In medical practice, interventions are the administration of a drug, the performance of a surgical procedure, counseling, or other modalities designed to relieve suffering. Drugs, surgeries (and the devices with which they are performed), and counseling themselves are said to be interventions.
Intracellular Protein A protein found inside the cell, either in the nucleus or the cytoplasm. Drugs targeting intracellular proteins must pass through the cell membrane.
Intracranial neoplasm A tumor within the confines of the skull.
Intractable Refractory; resistant to treatment
Intraocular Pressure Pressure created by the continual renewal of fluids within the eye. It is increased in glaucoma. In acute angle-closure glaucoma, it rises because the canal into which the fluid in the front part of the eye normally drains is suddenly blocked, whereas in chronic glaucoma, a gradual imbalance between production and removal (resorption) of the fluid in the back part of the eye (with supply exceeding demand) occurs.
Intravenous Within a vein or veins
Intravenous Immunoglobulin (IVIg) Complex mixtures of antibodies purified from healthy people and given as therapeutics to patients for a variety of conditions.
Invasive With regard to tumors, suggests movement into the bloodsteam, lymphatic system, or impingement upon crucial structures in the body.
Investigational New Drug Application (IND) Application to the Food and Drug Administration for permission to begin human clinical trials.
Ion Channel A protein found in the membrane of many cell types, particularly neurons in the brain, that allow sodium, potassium, and other ions to flow into and out of cells in a regulated manner. Ion channels are important for many cellular processes, including signaling in the brain and the immune system.
IPDAS (Élan) Acronym for Intestinal Protective Drug Absorption System, it is Élan’s proprietary multiparticulate tablet technology designed to enhance the gastrointestinal tolerability of potentially irritant or ulcer-causing drugs.
Ischemia Localized tissue anemia and oxygen debt due to obstruction of the inflow of arterial blood.
Isoenzyme Any of two or more functionally similar yet chemically distinct enzymes.
Isolex Cell Selection System (Nexell) A magnetic bead separator which is used to isolate and purify stem cells in the clinic.
Isotope Variants of elements by virtue of different mass numbers, typically due to a different number of neutrons in their nuclei.
Junk DNA DNA which does not code for proteins. So-called 'junk' DNA makes up over 95% of our total DNA content. Although lack of understanding of the function of this DNA led to its name, junk DNA is now known to be critical for the regulation of genes, for providing templates for evolutionary processes, for regulating chromosome replication, and many other processes. The vast majority of genetic variation occurs in regions of DNA that does not code for proteins, which include intervening sequences (introns), regulatory regions (such as promoters and enhancers), and many other sorts of non-protein coding DNA.
Kallikrein A family of proteins best known for involvement in blood vessel responses to various stimuli. Kallikrein initiates a cascade of molecular events leading to increased vascular permeability, smooth muscle contraction, vasodilation, and pain. C1 esterase inhibitor (C1-INH) is a naturally occurring kallikrein inhibitor.
Kaposi Sarcoma A skin malignancy that tends to afflict elderly people or those with an abnormal immune system as in AIDS. The tumor has many blood vessels, and is characterized by soft purplish plaques and raised skin lesions that form nodules. In AIDS patients, the tumors can also arise internally and cause severe internal bleeding. "
Kennedy Syndrome The most common form of adult-onset spinal muscular atrophy syndrome.
Keratinocyte Skin cell that makes keratin, a protein found in the upper layer of the skin, hair, nails, and animal horns.
Keratinocyte Growth Factor 2 (KGF2) A growth factor (small hormone like signaling protein) secreted by certain cells which stimulates growth of skin cells (keratinocytes).
Kinase A class of enzymes which add phosphate to proteins and other molecules inside of cells. Different kinases have specificity for certain targets, and represent an important class of intracellular signaling molecules relevant to many disease processes.
Kit A kinase (signaling protein that adds phosphates to proteins) involved in regulating cell division and other cellular events. Related to the kinase Abl.
Knockout Mouse A strain of mouse engineered to lack the expression of a specific gene or genes.
Krabbe Disease A lethal, demyelinating condition caused by a deficiency of the activity of the enzyme galactosylceramidase. It is more appropriately called Globoid Cell Leukodystrophy. The group of disorders known as leukodystrophies affect growth of the myelin sheath of nerve fibers in the central nervous system.
LDL Short for “low density lipoprotein,” LDL cholesterol is the “bad” cholesterol, as elevated LDL levels are associated with an increased risk of coronary heart disease. See “cholesterol” entry.
L-Dopa 3,4 dihydroxyphenylalanine, the biologically active form of dopa, it is a product of the amino acids tyrosine and phenylalanine and a synthetic precursor to other catecholamines (norepinephrine) and epinephrine as well as the skin pigment, melanin. Being a precursor to the central nervous system neurotransmitter dopamine, it can be used in a specific formulation to enhance depleted dopamine stores in Parkinson disease, as dopamine itself is unable to penetrate the blood-CNS barrier. Also known as levodopa.
Leprosy Symptomatic infection with Mycobacterium leprae, it may involve the skin, peripheral nerves, anterior eye, upper respiratory tract, and other organ systems. Leprosy can slowly and progressively lead to deforming disability with most affected people dying with it rather than of it. Leprosy is also called Hansen disease.
Leptin A hormone involved in body fat regulation. Originally it was thought to trigger weight loss, but now it is thought to signal the brain as to the quantity of fat there is on the body. The interactions of leptin with other chemicals in the body remain poorly understood, and thus it has thus far made a poor pharmaceutical target.
Leukaphoresis (1) The process of separating the white blood cells, or any of their subsets, from the remainder of blood components and collecting them. (2) A machine which aids in doing this.
Leukemia One of many types of blood cell cancer cells, most often of white blood cells or their precursors. Leukemia cells usually look quite different from their normal counterparts, and do not function properly.
Leukotriene Along with prostaglandins, one of the most well-studied chemical mediators of allergic and other inflammatory reactions. Released from mast cells and basophils, leukotrienes were originally known as the slow reacting substances of anaphylaxis. Their actions are somewhat similar to those of histamine, but Leukotriene D4, for instance, is 10 times more potent than histamine. Leukotrienes have additional key roles in the allergic inflammatory response. Researchers hope that antileukotrienes will prove more effective than antihistamines in combating allergy. Leukotriene modifiers have been used to a limited extent in the treatment of asthma.
Lifetime Risk Estimated risk of getting a condition in a lifetime, typically expressed as a ratio of 1:X. "
Ligand A molecule that interacts with a receptor.
Lipase An enzyme that breaks down specific fats/glycerides.
Lipid The biochemical term for fat. Lipids play important roles in cells, especially as an ingredient of cell membranes. For gene therapy, lipids can be formed into balls (liposomes; like soap bubbles) which can carry the therapeutic genes and fuse with cell membranes in the body.
Lipid Metabolism The process by which the body processes lipids (fats).
Liposome A hollow sphere made out of lipid which can carry payload such as gene therapy.
Lithium A positively charged element similar to sodium and potassium. Its similarity to these other elements allows it to interfere with their activities inside cells and on cell surfaces. Lithium also interferes with the biological activities of other positively charged atoms such as calcium and magnesium. Pharmacologically, lithium interferes with the activity of certain neurotransmitters. It also influences tryptophan and serotonin concentrations in the brain, and increases bone marrow production of white blood cells. Lithium has been used for the treatment of mania, including that associated with bipolar disorder, since the 1950s.
Locus Ceruleus The major group of norepinephrine-utilizing neurons in the brain, it plays a role in mediating specific functions relating to memory, emotion, arousal, and attention.
L-selectin A protein expressed on white blood cells that mediates adhesion and trafficking.
Lumen A channel or cavity through which a fluid flows.
Lym-1 A monoclonal antibody that binds a class II major histocompatability complex (MHC) protein. Lym-1 is being investigated for the treatment of B cell lymphomas.
Lymph Fluid containing a high concentration of white blood cells, found inside lymphatic vessels and lymph nodes.
Lymphatic System The organ system including the spleen, lymph nodes, and lymphatic ducts that, along with blood vessels, are responsible for the circulation of white blood cells through the body. Immune reactions take place in the lymphatic system organs.
Lymphocytes White blood cells such as B cells and T cells.
Lymphocytic Leukemia A group of malignant diseases of the white blood cells known as lymphocytes, predominantly B and T cells and their precursors.
Lymphography Visualization of the lymphatic system by x-rays (or the like) following injection and distribution of a contrast agent (dye).
Lymphoma A general term for cancer originating in the lymphatic system and involving cancerous growth of lymphocytes. There are two main categories of lymphoma: Hodgkin Disease and non-Hodgkin Lymphomas.
lymphotoxin Members of the tumor necrosis factor (TNF) family, lymphotoxin a and lymphotoxin b have similar biological roles as TNFa. These cytokines have diverse functions in immune development, immune function, and other processes.
Lysis Dissolution, explosion, tearing, as in a mechanism of cell killing.
Lysosomal Storage Disease One of several single gene disorders, each characterized by a specific defective enzyme leading to the accumulation of specific metabolites. As the enzyme is present in the intracellular digestive organelle called the lysosome, the accumulation tends to occur there.
Lysosome Intracellular digestive organelle.
M Protein A substance in the blood that physicians use to determine the severity of multiple myeloma.
Macaque Monkeys One of twelve short-tailed species of monkeys of the genus Macaca that can be both arboreal and ground dwelling. Includes Rhesus monkey.
Macrolide An antibiotic class that targets the 50S ribosomal subunit of bacteria, blocking the ability to synthesize proteins. Macrolides are derived from Streptomyces (spore forming bacteria that grow slowly in soil or water and somewhat appear like fungi) and have a complex chemical (meacrocyclic) structure. Zithromax (azithromycin), Biaxin (clarithromycin), and Erythromycin, in all of its forms, are macrolides.
Macrophage A white blood cell which destroys invading bacteria and cells by engulfing (eating) them, a process called phagocytosis.
Macular Degeneration A progressive disorder affecting the central part of the retina causing gradual loss of central vision.
Magnetic Targeted Carriers Technology (FeRx) A novel, experimental, proprietary delivery system designed to decrease drug toxicity currently in clinical trials for delivery of chemotherapy agents.
Magnitude of Treatment Effect The degree to which an intervention produces or contributes to an outcome. The most basic measurements for quantifying its extent are the relative and absolute benefit increases, risk reductions, and risk increases. The statistics describing relationships between two or more sets of numbers are the statistics that really matter in research. Qualitative terms like trivial, small, moderate, and large, are also frequently used to discuss results in terms of comparative magnitudes.
Major Histocompatibility Complex (MHC) A family of proteins that present antigens to T cells. Class I MHC molecules, found on nearly all cells in the body, present antigens to CD8+ cytotoxic T cells. Class II MHC molecules, found on “professional” antigen presenting cells like macrophages and dendritic cells, present antigens to CD4+ helper T cells.

MHC molecules are the most polymorphic proteins coded by the human genome, with hundreds of alleles in the population. Every person (except for identical twins) has a distinct set of MHC proteins. Differences in MHC proteins are largely responsible for tissue and organ graft rejection.

Malaria Contracted from the bite of specific mosquitoes infected with one of many species of Plasmodium parasites, it is the most common infectious disease in the world, affecting approximately 40% of the Earth’s population. Acute infection can be life-threatening and chronic infection range can be debilitating and degenerative.
Malignant (1) Regarding abnormal growths, it implies potential or actual spread to other areas of the body (potential or actual metastasis), and implies the existence of cancer. (2) Resistant to treatment or severe, as in malignant hypertension.
Mammalian Cell Culture The technique for growing mammalian (usually mouse or human derived) cells in the laboratory.
MAP Kinases A family of intracellular signaling proteins involved in many biological processes, including cell division. MAP stands for mitogen activated protein.
Market Capitalization A company’s common stock price multiplied by the number of shares outstanding.
Marketing Authorisation Application (MAA) Application to the European Agency for the Evaluation of Medicinal Products (EMEA) to begin marketing drugs and biologics to the public. Analogous to a BLA or an NDA in the U.S.
Mass Spectrometry One of several techniques in which some sort of instrument is used in order to produce ions from atoms or molecules which are then separated according to their charge-to-mass ratios and detected.
Mast Cells White blood cells that release histamine and other mediators of allergy and asthma.
Matrix Metalloproteinases A class of pro-angiogenic proteases that specifically degrades extracellular matrix.
MEDIPAD (Élan) A drug delivery system that can deliver a particular amount of a drug for up to 48 hours from a patch on the chest. This drug delivery system combines the simplicity of a patch with the delivery capabilities of an infusion pump.
Megakaryocyte A huge bone marrow cell that is an ancestor of platelets.
Melanocortin Any of a group of pituitary peptide hormones that include adrenocorticotropin (ACTH) and the a, b, and g-melanocyte-stimulating hormones. Melanocortins bind to G-protein coupled receptors. These ligands and their receptors participate in the control of many well-characterized endocrine, autonomic, and central nervous system activities. The most well studied biological phenomena related to these proteins are inflammation, appetite regulation, body weight regulation, insulin regulation, and skin pigmentary regulation.
Melanoma A pigmented tumor of the skin and, in rare instances, of the mucous membranes. A malignant melanoma tumor may be invasive and spread to lymph nodes and other sites more frequently than other skin cancers.
Meningitis Inflammation of the meninges - the 3-layered covering of the central nervous system (the brain and spinal cord).
Mesenchymal Cells The stem cells that give rise to connective tissues including bone marrow stroma, bone, cartilage, ligament, tendon, muscle, and fat.
Messenger RNA (mRNA) An RNA molecule transcribed from DNA and, typically, translated into the amino acid sequence of a polypeptide/protein.
Meta-Analysis An analysis of analyses. The statistical analysis of a collection of analyses for the purpose of integrating the findings. A strong meta-analysis collects trials of similar methodology in order to enhance the statistical power to detect outcomes.
Metabolic Disorders (1) disorders characterized by the presence of the body chemistry’s inability to either convert small molecules into larger ones (anabolic disorders) or large molecules into smaller ones (catabolic disorders) (2) generic term for “inborn errors of metabolism,” devastating, often childhood, diseases caused by an absent or malfunctioning enzymatic protein (3) euphemistic term in drug discovery press releases and public discourse for obesity and/or type 2 diabetes mellitus.
Metabolism The universe of chemical changes occurring in a tissue, this consists of creating large molecules from smaller ones (anabolic changes) and small molecules from larger ones (catabolic changes).
Metabolite A product of metabolism; usually referring to a product of catabolism or a waste product.
Metachromatic Leukodystrophy A genetic disorder caused by a deficiency of the enzyme arylsulfatase A. The group of disorders known as leukodystrophies affect growth of the myelin sheath of nerve fibers in the central nervous system.
Metastasis Cells that have spread from an original location. The metastatic cells, typically cancerous, are of the type of the original source. So, if a metastatic cancer arose in the colon and metastasized to the liver, the cells growing in the liver are colon cancer cells.
Metastatic The spread of tumor cells from an original location in the body to a secondary location via the lymph system or blood circulation. To metastasize effectively, tumor cells must detach from their original location, invade a blood or lymphatic vessel travel in the circulation and establish a new cellular colony.
Metastatin An anti-angiogenic formulation in discovery/pre-clinical testing at EntreMed.
Microbe An organism of microscopic or ultramicroscopic size.
Microbeads Plastic spheres, the size of mammalian cells or smaller, capable of being used as binding surfaces for proteins and other biological reagents.
Microorganism An organism of microscopic or ultramicroscopic size.
Micropor Technology (Altea Genomics) Convenience-based proprietary drug delivery technology allowing controllable, transdermal delivery of macromolecular (large molecule) drugs through microscopic pores in the outermost dead layer of skin cells. The technology has delivered physiologically relevant amounts of peptides, proteins, and DNA in pilot clinical studies.
Microtubules Elements in the cytoplasm of cells that participate in chromosome movement during phases of the cell cycle leading to cell division.
Migraine Headaches Any of several sorts of vascular headaches some associated with a symptom complex including any or all of the following: vertigo, nausea and vomiting, photophobia (light hypersensitivity), and scintillating appearances of light (scintillating scotomata).
Mild Cognitive Impairment A evolving diagnosis that represents some transition zone between mild memory impairment and a more severe cognitive decline such as that seen in Alzheimer disease (AD), vascular dementia, and other dementing illnesses. It is a useful concept when studying the prevention of AD because it identifies high-risk patients. It is a detrimental concept when used loosely as it may encourage failures to detect other types of cognitive disturbances. Great variability in diagnostic criteria and neuropsychologic testing exists such that the precise, global criteria for this diagnosis remains uncertain. Also known as “incipient dementia” and “isolated memory impairment,” amongst many other non-specific terms.
Mimetic Relating to, characterized by, or exhibiting mimicry. Pharmacologic compounds can mimic specific biologic and pharmacologic functions, typically in the context of a receptor interaction. Often, they physically resemble a functionally relevant portion of the molecule they mimic. Their administration is often preferred to that of the mimicked compound due to more appealing phamacokinetics and/or a more acceptable safety and tolerability profile, often due to the lack of a discarded portion of the parent compound.
Mini-Mental Status Examination (MMSE) A five-minute screening test designed to assess basic cognitive function in a number of different areas such as orientation, short term memory, recall, writing, calculation, and higher integrative functioning. It provides a rapid way to determine if more sophisticated testing is necessary. Also known as mental status exam and mini-mental state exam.
Mitochondrion The principle energy source of a cell, it is a cytoplasmic organelle in which is located enzymes specifically designed to catalyze reactions that, in total, produce energy. It is also within this organelle that our non-nuclear genetic material (mitochondrial DNA) is housed, virtually all of which is transmitted to subsequent generations by a motherâ??s ovum (egg). "Mitochondria" is the plural form.
MLH1 Gene A gene whose mutations play a key role in the susceptibility to HNPCC-hereditary colon and endometrial cancer.
MMAC1 Gene A gene whose mutations have been found to play a role in the most common and most lethal brain cancer, glioblastoma, in addition to advanced cancers of the prostate, breast and skin, and involved in other cancers such as those of the blood, bladder, lung and testis. "
Modalities Treatment methods
MODAS (Élan) An acronym for Multiporous Oral Drug Absorption System, it is a proprietary drug delivery technology which formulates a tablet in such a way as to facilitate more predictable particle release rates and absorption.
Model Organism An organism used for research purposes, commonly E. coli bacteria, yeast, fruit flies, and mice.
Monoamine Oxidase B (MAO-B) An enzyme involved in the catabolism of amines such as dopamine and phenylethylamine. MAO-B’s activity has been implicated in oxidative stress that underlies the acceleration of neurodegenerative processes. Thus, its inhibition tends to have effects that retard this process. Selegiline is the prototypical inhibitor of this enzyme.
Monoclonal Antibody A laboratory-engineered antibody, derived from a single cell, that recognizes a specific antigen.
Monocytes White blood cells involved in interacting with T cells, often with phagocytic (cell eating) properties.
Monotherapy A single therapeutic agent.
Morbidity Illness short of death.
Morphelan An oral form of morphine designed to have both rapid onset and long duration.
Morphine The prototypic opiate analgesic (pain medication) with agonist activity at kappa and mu receptors.
Movement Disorders The broad category of neurological diseases featuring impaired involuntary movement, including Parkinson disease, essential tremor, other types of tremors, dyskinesias, and dystonia.
MSH2 Gene A gene whose mutations play a key role in the susceptibility to HNPCC-hereditary colon and endometrial cancer.
MTC-DOX (Élan/FeRx) Magnetic Targeted Carriers (MTC) technology combined with the chemotherapeutic doxorubicin. MTC is a novel, experimental, proprietary delivery system designed to decrease drug toxicity from targets other than the tumor being exposed to a chemotherapeutic agent.
Mucopolysaccharidosis I (MPS-1) Hurler syndrome, Scheie syndrome, and Hurler-Scheie syndrome, all characterized by the deposition of mucopolysaccharide, a specific type of carbohydrate, in tissues due to varying degrees of deficiency of the enzyme alpha-L-iduronidase. "
Multiple In reference to company stock market valuation, multiple is used to describe ratios of some measure of the market value of a company´s stock over some measure of a company´s financial performance. The most commonly used multiples are price to earnings (P/E) and enterprise value to EBITDA or (EV/EBITDA).
Multiple Myeloma A malignancy of antibody-producing plasma cells often characterized by anemia, hemorrhages, recurrent infections, and weakness.
Multiple Sclerosis A neurodegenerative disease caused by damage to the brain and spinal cord, particularly to the myelin sheath which surrounds nerves.
Multipotent Stem Cells Class of stem cells that can give rise to a specific cell population, for instance, blood stem cells give rise to white and red blood cells but not neurons.
Murine Derived from mice or rats.
Mutation A heritable alteration in a DNA sequence or chromosome.
Myelin A fatty substance that insulates nerves, it serves as an electrical conduit by ensuring that signals racing down nerves remain intact during transit.
Myeloablative Chemotherapy Chemotherapy that wipe outs the cellular components of the bone marrow.
Myeloid Progenitor Inhibitory Factor-1 (MPIF-1) A naturally occurring human chemokine that has been demonstrated to have the capacity to inhibit stem cell growth. Being a potent suppressor of bone marrow, Human Genome Sciences is somewhat ironically exploring the potential of a recombinant version of this compound as a way to arrest marrow cells at a given developmental state during chemotherapy. The thinking is that marrow cell counts can be preserved through treatment such that cell stimulation after the marrow-suppressive effects of chemotherapy have worn off might provide normal, not suppressed, counts of circulating red cells, white cells, and platelets.
Myelosuppression Diminished bone marrow function, often induced by chemotherapy or radiation.
Myocardial Infarction A heart attack. Heart muscle (myocardium) tissue, due to abrupt (acute) circulating blood and oxygen deprivation, dies. Tissue death (necrosis) due to oxygen deprivation is known as ""infarction." Blood flow interruptions are typically caused by arteriosclerosis accompanied by coronary artery narrowing. Eventually, a thrombosis (clot) occludes the coronary artery, preventing the flow of blood, and therefore limiting the oxygen supply, to the myocardium.
Naked DNA The simplest form of gene therapy in which the gene and control regions are injected without a vector into the patient.
NanoCrystal Technology (Élan) A proprietary drug delivery technology that helps deliver poorly water soluble drugs by delivering the drug as very small crystals stabilized by polymers.
Naratriptan The generic name of Glaxo Wellcome’s Amerge, a 5-HT 1B/1D agonist indicated for the treatment of acute migraine.
Natriuresis An increased excretion rate of sodium.
Natriuretic Peptide One of a group of protein-like substances whose principal physiologic role is to increase the rate of sodium excretion via urination.
Necrosis A type of pathological cell death.
Negative Predictive Value Considering people being tested for a given condition by a particular test, it is the proportion of persons who test negative that truly do not have the disorder of concern.
Neoplasia 1. The formation of tumors. 2. A condition characterized by tumors.
Neotrofin (NeoTherapeutics) An investigational agent with properties that play a role in amyloid plaque interruption, and that shows ample evidence of actual regeneration of nerve cells in vitro. Neotrofin is being investigated for treatment of Alzheimer disease.
Nerve Growth Factor A polypeptide growth factor member of the neurotrophin family which also includes Brain Derived Neurotrophic Factor (BDNF) and Neurotrophin-3 (NT3). It controls the survival and development of certain populations of neurons and has been reported to stimulate growth of other cell types.
Neural Nerve; pertaining to nerve cells; of the brain.
Neuraminidase An enzyme from influenza that helps the virus leave cells in order to infect other cells.
Neurodegeneration Progressive, central nervous system (CNS) cell death.
Neurodegenerative Disorders Disorders characterized by the death of neurons and/or related cells. Alzheimer disease, Parkinson disease, Friedreich Ataxia, Multiple Sclerosis, and Amyotrophic Lateral Sclerosis are the prototypical neurodegenerative disorders.
Neuroimmunophilin Ligand Investigational agents known to have the ability to, essentially, grow neurons in vitro, being investigated for the treatment of Alzheimer and other neurodegenerative disorders.
Neurological Disorders Disorders of the nervous system including neurodegenerative disorders, stroke, seizure disorders, peripheral neuropathy, traumatic injury to the brain and spinal cord, and many others.
Neurology The study of the nervous system and its disorders.
Neuromuscular Disorders Disorders of the nervous and musculoskelatal system.
Neuron Nerve cell; cells transmitting electrical signals that make up the brain, spinal cord, and peripheral nerves.
Neurotransmitter A chemical that carries a chemical signal between neurons and muscles and from neuron to neuron. Neurotransmitters are released across the synapse - the gap between two neurons or a neuron and a muscle. Different types of neurons release different neurotransmitters.
Neurotrophins Structurally related molecules that support the survival of different classes of embryonic neurons. They include nerve growth factor (NGF), brain-derived neurotrophic factor (BDNF),neurotrophin-3 (NT-3), GDGF, and ciliary neurotrophic factor.
Neutropenia Below normal circulating concentrations of neutrophils, a type of white blood cell filled with microscopic granules containing enzymes that digest microorganisms. Neutrophils are a typeof granulocyte. Granulocytes are part of the immune system that has general, non-specific activity; unlike B and T lymphocytes, granulocytes do not just respond exclusively to specific antigens.
Neutrophil A type of white blood cell. They contain microscopic granules (and are, thus, classified as granulocytes), little sacs containing enzymes that digest microorganisms such as bacteria.
New Drug Application (NDA) Application to the Food and Drug Administration to begin marketing a drug to the public.
NFkB A transcription factor that activates genes involved in immune responses as well as other classes of genes.
NFkB Antisense Oligonucleotide A small piece of DNA complementary to the NFkB gene which has the potential to neutralize production of the NFkB protein. NFkB is a transcription factor, meaning it is a protein that turns on and off a specific programs of genes.
Niacin Nicotinic acid or nicotinamide, forms of Vitamin B3 used by the body to form the vital coenzymes called nicotinamide adenine dinucleotide (NAD) and nicotinamide adenine dinucleotide phospate (NADP). Niacin plays a preventive role in the prophylaxis of cancer, diabetes, cardiovascular disease, skin breakdown, and dementia. Niacin can play a therapeutic role in dyslipidemia and cardiovascular disease.
Nifedipine Extended Release Tablets Formulations of calcium channel blocking agent, used for treatment of hypertension, that have a longer duration of effect. Examples are Pfizer’s Procardia XL, Bayer’s Adalat CC, and Mylan’s generic. "
Nitric Oxide (NO) A colorless gas formed by oxidation of nitrogen or ammonia, it is a substance that tends to relax blood vessels in the body. NO is also a fundamental mediator of inflammation. Excess NO production has been implicated in multiple pathological processes including sepsis and other processes characterized by hyperactive inflammatory processes. "
Nitroglycerine Of chemical formula C3H5NO3(3), it is formed by the action of sulfuric and nitric acids upon glycerine and is (1) A powerful vessel opening (vasodilating) agent used in the treatment of heart muscle injury, ischemia, and infarction. Many generic formulations by various manufacturers are marketed. (2) A potent component of dynamite.
Non-Hodgkin Lymphoma (NHL) One of two main categories of lymphoma involving cancerous growth of B or T cells. About 85% of NHL are of B cell origin compared to about 15% of T cell origin.
Non-Steroidal Anti-Inflammatory (NSAID) Drugs A class of anti-inflammatory analgesics (pain medications) that do not entail a steroid component.
Notch One of a family of cell surface proteins involved in cell fate decisions in the immune system and other organs.
Nucleic Acids DNA and RNA.
Nucleotides One of the structural components, or building blocks, of DNA and RNA. A nucleotide consists of a base (one of four chemicals: adenine, thymidine, guanine, and cytosine) plus a molecule of sugar and one of phosphoric acid.
Nucleus An organelle (compartment) found within most cells that contains most of the genetic material (DNA). Bacteria and red blood cells are examples of cells that do not contain nuclei. In mammalian cells, the small proportion of non-nuclear DNA is found in the mitochondria.
Obesity Abnormal body mass index, usually defined as more than 20 percent above average weight for age, height and bone structure.
Off-label Use The use of a drug in a way neither approved by the FDA nor permitted to be put on its label and advertised as its intended purpose. Once approved, physicians are free to prescribe a drug for any indication they see fit.
Oligonucleotides Short sequences of DNA, usually 8 to 30 nucleotides long, synthesized in the laboratory for research or therapeutic purposes.
Oligos Lab slang for oligonucleotides.
Oligosaccharide A chain of sugars attached through O- or N-linked chemical bonds. Oligosacchariades are often attached to cell surface proteins or lipids. Specific oligosaccharide structure can vary between cells, and may be a marker for tumor cells. "
Omega-Conotoxin (OCT) Conotoxins are small peptide neurotoxins found in the venoms of Conus snails. Those of the omega family typically have 25-28 amino acid residues and fold into three-dimensional structures stabilized by three disulfides. They act by binding to and inhibiting presynaptic calcium channels, thereby preventing neurotransmitter release. This feature has been harnessed to provide clinical analgesia (pain relief).
Oncogene A gene that, when mutated, has the potential to contribute to the transformation of a normal cell into a tumor cell. When unaltered, these genes play a normal role in cell growth. Oncogenes are potential targets for drugs and diagnostic tests.
Oncology The study of cancer.
Oncoprotein A protein encoded by an oncogene.
Opiate Containing or derived from opium, such as morphine. Opiates are naturally occurring basic (alkaline) molecules with a complex fused ring structure used for pain management. "
Oral Mucositis Inflammation of the mucous membranes of the oral cavity.
Organ Rejection One of several sorts of immunologic response to incompatibility in a transplanted organ.
Orphan Disease An FDA category of disease defined as one that affects fewer than 200,000 people in the United States. There are more than six thousand different rare disorders.
Orphan Drug A drug targeted to treat an Orphan Disease, which is a condition affecting a population less than 200,000. The Orphan Drug Act offers tax credits, protocol assistance, research grants and a seven-year monopoly on drug sales as incentives to pharmaceutical companies to develop and manufacture Orphan Drug.
Orthopedic Surgery The branch of surgery concerned with the skeletal system.
Osteolytic Relating to bone breakdown.
Osteopetrosis A condition characterized by abnormally thick, dense bones due to an inherited defect in bone resorption, the process by which old bone is broken down and removed to make room for new bone to be added to the skeleton. Although bones are unusually dense, they are also quite fragile and easily broken. Bone marrow disease can also be a prominent feature of the disease, leading to anemia and/or neutropenia and/or thrombocytopenia. Blindness, deafness, and strokes may occur when the skeleton is so dense that it interferes with the normal course of blood vessels or nerves.
Osteoporosis Loss of calcium and other substances from bones, causing bones to become weak and prone to fractures.
Outcome Consequence or result, the most important in late-stage clinical trials and medical practice being those that involve or depend upon direct observation of living patients or study subjects. Outcomes not meeting these criteria are said to be surrogate or secondary outcomes.
Outpatient A patient treated without being admitted for a stay in the hospital.
Overall Response Complete response plus partial response. Some studies will additionally include “minor responses,” variably defined but always less than a partial response.
Oxidative Stress A condition caused by the presence of too many free radicals in a cell. In response, cells naturally manufacture antioxidants that convert free radicals into harmless oxygen, water, and other chemicals.
P Value The probability that an observed difference between the intervention group and control group is due to chance alone if the null hypothesis is true. The null hypothesis proposes that an intervention has no effect, and that there are no true differences in outcomes between an intervention group and a control group. When P is less than the prospectively determined a-level (typically 0.01 or 0.05), then the null hypothesis is rejected, and statistical significance is said to be achieved for the relevant finding. The a-level is the probability of a false-positive (type I) error. For instance, setting a at the traditionally accepted medical hypothesis testing level of 0.05 implies that investigators, and the medical community, accept that there is a 5% chance of concluding incorrectly that an intervention is effective when it is ineffective in reality (you'll see p< 0.05 or p=a value less than 0.05 in statistically significant findings). Occasionally you'll see an a-level set at 0.01 (tight) or 0.10 (lax).
P/E Price-earnings multiple. Multiple of a company’s current common stock price divided by its diluted annual earnings per share. While a company’s P/E multiple is usually computed using the most recently available stock price, the earnings per share from different annual time period can be used. The most frequently earnings per share that are used in computing P/E are trailing and forward 12 months and current and next fiscal year.
p10 Gene A tumor suppressor gene in the p16 family that is a potential anti-cancer drug target.
p16 Gene A tumor suppressor gene whose protein product is involved in regulating cell growth and division, in the same biological pathway as p53 and Rb. p16 is being explored as potential anti-cancer drug target. Germ-line mutations of p16 are involved in the susceptibility to some familial melanoma and pancreatic cancer.
p38 p38 MAP kinase has a broad range of biological effects in many cell types. In immune system cells, p38 regulates production of the fundamental proinflammatory cytokines, TNF-alpha and IL-1. Inhibiting the molecular activities of p38 MAP kinase has the potential to be of value in a wide variety of inflammatory conditions. Preclinical efforts have mostly focused on rheumatoid arthritis, systemic inflammatory response syndrome, adult respiratory distress syndrome, restenosis, allergy, asthma, reperfusion injury, and neuropathic pain. The inflammatory component of Alzheimer disease and certain cancers has also been addressed by anti-p38 MAP kinase inhibition in preclinical investigation.
p53 Gene A tumor suppressor gene which encodes a protein that regulates cell growth and, when working properly, is able to cause cells to destroy themselves. p53 is frequently found to be inactivated or mutated in many tumor cells. For example, p53 is often found to be mutated to an inactive form in the lung cells of smokers with lung cancer by a compound found in tobacco smoke.
Paclitaxel The generic name of Bristol-Myers Squibb’s Taxol and Liposome/Elan’s experimental Bromotaxane, the former being indicated in specific scenarios in ovarian carcinoma, non small-cell lung cancer, and Kaposi sarcoma.
Paget Disease Also known as osteitis deformans, Paget disease is a metabolic bone disease characterized by bone destruction and regrowth that results in deformity. Excessive bone tissue breakdown followed by abnormal bone formation results in structurally enlarged but weakened new bone that is filled with an abnormally large quantity of new blood vessels that are also often malformed. Its cause is unknown, but several lines of evidence point toward early viral infection plus genetic predisposition.
Pain Management Clinical intervention designed to alleviate pain or make it, at least, more bearable.
Paliate To moderate the intensity of a disease, disorder, or the symptoms thereof. Often, the term is used in the care of seriously ill people to indicate that the opportunities to inhibit disease progression have been lost, but the opportunities to provide pain and discomfort relief remain.
Pancreas An elongated, lobulated salivary gland of the abdomen, its endocrine portion produces glucose-regulating hormones insulin and glucagon and its exocrine portion produces “juice” containing enzymes useful in digestion.
Pancreatic Islet Cells The cells in the pancreas which secrete the glucose-regulating hormones insulin and glucagon. Specific islet cells are destroyed in patients with type I diabetes.
Pancytopenia A clinically relevant shortage of red (anemia) and white (neutropenia, leukopenia) blood cells as well as platelets (thrombocytopenia).
Parathyroid Hormone Hormone manufactured by the parathyroid gland (behind the thyroid gland in the neck), it is a fundamental regulator of calcium and phosphorus balance. "
Parenteral By means other than the gastrointestinal tract. Not enteral. From para: alongside of, beyond, or aside from and enteric: of or relating to the intestines. Main routes of parenteral administration are intravenous, subcutaneous, and intramuscular, with much development in the areas of transdermal, intramedullary, and pulmonary/inhaled. Main routes of enteral administration are oral (by mouth) and rectal with tube feeding capacity for various bowel segments reserved for those unable to take nutrition by mouth.
Parkinson Disease A neurological disorder characterized by shaking and difficulty with walking, movement, and coordination. The disease is associated with damage to specific parts of the brain that control muscle movement.
Parkinsonism The constellation of movement disorder symptoms prompted by pathology of the basal ganglia and extrapyramidal areas of the brain. Parkinsonism can be caused by Parkinson disease, specific drugs or toxins, and conditions related to Parkinson disease.
Partial Response Often used in cancer investigations, a decrease in the amount of detectable cancer of 50% or higher measured using predetermined standards. More precisely, a true partial response in cancer is a decrease in the total cross sectional area of all measurable tumors over 50%. Research in non-cancerous proliferative diseases, rheumatologic diseases, neurodegenerative diseases, and others have analogous partial response scales.
PASI Score A standard measurement of the extent of disease in patients with psoriasis. PASI is an acronym for "psoriasis area and severity index." The scoring system can be used to measure a patient's response to marketed or developmental anti-psoriasis therapies. The index's elements include assessing the extent of psoriasis over the various body regions as percent of body surface area, the extent of body region affected, and the extent of psoriatic changes. Results are often issued as comparisons of the proportion of patients in a given treatment group experiencing a given percent change in PASI scores rather than comparisons of the mean or median PASI score for a treatment group.
Pathogen An agent such as a virus or bacteria that causes pathology or disease.
Pathology The study of the fundamental nature, causes, and development of abnormal conditions and the structural and functional changes that result.
Pathophysiology A contraction of the words ""pathology"" and ""physiology,"" and combining their meanings. It is the study of disease associated malfunctions of individuals, organs, tissues, and cells. Pathophysiologic alterations also imply functional changes rather than structural defects. "
Payload A substance attached to a monoclonal antibody to improve its effectiveness. Payloads can be radiation, chemotherapeutics, enzymes, or other molecules.
Pegylation Containing polyethylene glycol (PEG) or having it added. Pegylation is a common chemical strategy for extending the duration of action of compounds.
Penicillin An antibiotic class that targets penicillin-binding proteins in the cell walls of bacteria, inhibiting production of the cell wall. The original antibiotic of the class, penicillin itself, was named for the fungal mold Penicillium notatum from which it is derived. Now, there are several generations of semi-synthetic and synthetic penicillins.
Peptide A chain of amino acids, such as a hormone or a growth factor. Proteins are peptides, although peptides is usually used to refer to short chains of less than 10 amino acids.
Perforin A protein released by cytotoxic T cells which literally pokes holes in the membranes of target cells.
Performance Status A measure of a study subject's degree of disability or symptom severity. Disease-specific, numeric scales are used for these sort of assessments. These measures are often confused with prognostic measurements. A patient’s performance status reflects their functional capacity rather than prognosis.
Perfusion Blood flow, therefore oxygen flow, to tissues and cells.
Peripheral Nerves Nerves located outside of the brain and spinal cord that control muscle movement and other functions. Any nerves not in the central nervous system.
Peripheral Neuropathy A classification of disorders involving pathology of nerves, but not including nerves of the brain or spinal cord.
Peripheral Vascular Disease Occlusive, typically atherosclerotic, disease of the vessels of the extremities (more often legs and feet compared to arms and hands). Manifestations can include diminished blood flow and pain while walking or standing.
Peroxidation A molecular reaction that joins oxygen to oxygen; its increased frequency has been implicated in adverse inflammation and aging.
Peroxisome proliferator-activated receptors (PPARs) Members of the nuclear hormone receptor subfamily of transcription factors. PPARs unite with retinoid X receptors (RXRs) to regulate the transcription of various genes. PPARs exist as PPARa, PPARd, and PPARg. PPARg is most well known for its involvement in fat cell (adipocyte) differentiation and regulation of insulin sensitivity. The drug class known as thiazolidinediones normalize elevated plasma glucose levels by activating PPARg, and are used for the management of noninsulin-dependent diabetes mellitus. Many companies are trying to improve on the risk-benefit profile of this class of marketed drugs by developing their own PPARg agnonists with different physical chemistries.
P-glycoprotein A protein "pump" found on the cell surface that expels chemotherapeutics from tumor cells. The activities of multiple drugs are diminished by interactions with this protein.
Phage Display A laboratory technique used to screen for particular proteins produced by viruses. Phage display can be used to identify fully human antibodies that bind to a particular target antigen.
Phagocytosis The process of one cell engulfing and digesting another.
Pharmacodynamics A compound’s pharmacologic effect on patients, it includes the study of uptake, movement, binding, and interactions of agents at their tissue and cellular site(s) of action.
Pharmacogenetics The study of genetic contributions to, and causes of, individual variations in drug response. Often, as a colloquialism, referred to as pharmacogenomics.
Pharmacogenomics Genome-wide analysis of the genetic determinants of drug efficacy, toxicity, pharmacokinetics, and pharmacodynamics in order to correlate genetic variation with drug response.
Pharmacokinetics Patients’ pharmacologic effects on compounds. Typically these effects are described with the classical properties relating to the movement of drugs within biologic systems: absorption (or uptake), distribution, metabolism, and excretion (or elimination), with toxicology often included (ADME-T). Binding and biotransformation are also considered pharmacokinetic properties.
Pharmacoproteomics High-throughput analysis of proteins correlated with drug efficacy, toxicity, pharmacokinetics, and pharmacodynamics in order to correlate protein expression with drug response.
Pharmacotherapeutics (1) Drugs (2) Chemotherapeutics or chemotherapies (3) Treatment of disease by pharmacologically active agents such as (1) or (2).
Phase I Trial The initial phase of clinical trials, performed in order to determine the safety of an investigational drug. For every 100,000 drugs screened, approximately 85 make it to this phase. Approximately 23% of drugs reaching phase I make it to market. This phase typically lasts approximately 1-3 years.
Phase II Trial The second phase of clinical trials, performed in order to determine the clinical effectiveness of an investigational drug. For every 100,000 drugs screened, approximately 70 make it to this phase. Approximately 28% of drugs reaching phase II make it to market. This phase typically lasts approximately 2 years.
Phase III Trial The final pre-FDA approval phase of clinical trials, performed in order to determine, with statistical rigor, the clinical efficacy and longer-term safety of an investigational drug. For every 100,000 drugs screened, approximately 33 make it to this phase. Approximately 60% of drugs reaching phase III make it to market. This phase typically lasts approximately 3-4 years.
Phase IV Trial A post-FDA approval phase of clinical trials, performed in order to determine additional indications for a drug.
Philadelphia Chromosome The genetic rearrangement between chromosomes 9 and 22 found in chronic myeloid leukemia patients, which leads to the abnormal activation of the protein kinase Abl.
Phosphatase An enzyme that removes phosphate groups from proteins and other target molecules.
Phosphodiesterase An enzyme that is a phosphatase that works on diesters in order to hydrolyze only one of the two ester groups. These enzymes play different roles in many cellular processes. Medically important diesters include cyclic nucleotides such as cyclic guanosine monophosphate (cGMP) and cyclic adenosine monophosphate (cAMP). Medically important inhibition of phosphodiesterase activity has occurred in the treatment of congestive heart failure, cardiac dysrhythmias, asthma, headaches, erectile dysfunction, and female sexual arousal disorder. Intensive research is ongoing in anti-inflammation, immune modulation, diabetes mellitus, memory, chronic obstructive pulmonary disease, depression, and blood clotting disorders, amongst others. Caffeine is a popular phosphodiesterase inhibitor.
Photodynamic Therapy Treatment that destroys proliferative cells with lasers and drugs that become active upon exposure to light. Applications have primarily been in proliferative diseases of the eye and cancer.
Photodynamic therapy Treatment that destroys proliferative cells with lasers and drugs that become active upon exposure to light. Applications have primarily been in proliferative diseases of the eye and cancer.
Physician's Desk Reference (PDR) A compendium, published annually by Medical Economics Company, that provides a primary guide to the use of all prescription drugs available in the United States. The PDR is the most fundamental pharmaceutical resource for physicians, and includes more than 3000 pages of detailed, FDA-approved, prescribing information. Its Companion Guide headlines the PDR resources that augment the main guidebook.
Pick Disease A form of dementia similar, in many ways to Alzheimer, but with a different pattern of brain atrophy.
Placebo A medically inert substance identical in appearance to a substance being studied, administered in order to distinguish between drug effects and suggestive effects of the material under study.
Plaques Patchy area on a body surface or cut surface of an organ, often described by the constituents of the area. Plaque formation on neurons is associated with neurodegenerative disease.
Plasma The liquid portion of the blood and lymphatic fluid.
Plasmid A laboratory vector used to transfer recombinant DNA to a new cell.
Plasminogen The inactive precursor of the fibrinolytic protein plasmin. When circulating plasminogen is converted to plasmin by tissue plasminogen activator, it begins a relentless attack on the fibrinogen fibers that entangle the blood cells caught in a blood clot. Plasmin thus dissolves clots, a process is called fibrinolysis.
Platelet Activating Factor (PAF) A powerful proinflammatory molecule, it is an acetyl glycerol ether phosphocholine made by many kinds of stimulated cells (e.g., basophils, neutrophils, monocytes, macrophages, endothelial cells). Interference with its activities during severe inflammation may prove therapeutic.
Platelet Derived Growth Factor (PDGF) A family of several closely related secreted proteins produced by a variety of cells, but most well known for their release from specific granules in platelets. Some of their roles include stimulation of migration and proliferation of fibroblasts, smooth muscle cells, and monocytes in addition to a legion of other pro-inflammatory activities.
Platelets Blood cell fragments that assist in blood clotting.
Pluripotent Stem Cells Stem cells that can develop into most cell types but lack the ability to form an entire organism.
Pneumonia Lung infection.
Polyclonal Antisera A mixture of antibodies with the ability to bind millions of different antigens, but enriched for certain antigens. Polyclonal antisera, a technological precursor to monoclonal antibodies, are made by injecting an animal with a certain antigen, and later purifying antibodies from the blood of that animal.
Polyethylene Glycol (PEG) A condensation polymer of ethylene glycol and water, they are wax like solids available in a variety of molecular weights, and when attached to some proteins lengthens their plasma half-life, reduces antigenicity and immunogenicity, and reduces sensitivity to proteolytic enzymes. "
Polyglutamate Many glutamates strung together or "polymerized."
Polymerase Chain Reaction (PCR) A fundamental process of molecular genetics that allows analysis of any short sequence of DNA (or RNA) without having to clone it. One may amplify selected DNA sections without having to employ the aid of bacteria and their replication, a process that takes weeks. With PCR such amplification takes only a few hours. Its utilities are myriad: diagnostic, DNA fingerprinting, find bacteria and viruses, study evolution, amplify DNA of individuals from ancient times, etc.
Polymorphism The existence of more than one form (“spelling”) of a DNA sequence, with each form being too frequent in the population to be due to new mutation alone. Examples of polymorphic DNA sequences are (1) genes for sickle cell and thalassemia believed to have prospered because they protect against malaria, (2) nucleotides, SNPs, within the angiotensinogen gene used by Myriad for their CardiaRisk diagnostic, (3) differences in trinucleotide repeat length detected by PCR-based diagnostic regimens in neurodegenerative conditions such as Huntington Disease. Polymorphisms are used as a tool in diagnostics, drug discovery, and for anticipating drug responses.
Polyp A tissue mass, often having a curiously swollen appearance, that develops on and projects from a surface such as the inside wall of a hollow organ or the skin.
Pompe Disease Acid maltase deficiency which leads to buildup of glycogen in all organs, especially the heart. Skeletal muscles can also be affected. This lysosomal storage disease causes progressive muscle weakness and muscle degeneration.
Positive Predictive Value Considering people being tested for a given condition by a particular test, it is the proportion of persons who test positive who truly have the disorder of concern.
Positron Emission Tomography (PET) An imaging technique that employs short-lived radioactive substances. The three-dimensional colored images provide information about, especially, metabolic processes unattainable with standard diagnostic imaging. A patient lies on a table within a scanner. Short-lived radiopharmaceuticals discharge positrons from the subject’s body. When positrons encounter electrons within the body, gamma rays are produced. Within the scanner are coils of detectors made up of specific crystals that generate light when struck by a gamma ray. The scanner records these gamma rays, mapping an image of the region of the radiopharmaceutical. As this radiopharmaceutical contains a common body chemical, PET permits the physician to see the location of the metabolic process of concern. A classic example is glucose (the form of sugar the body prefers for energy production) combined with a radioisotope shows where glucose is being utilized in the brain, heart muscle, or a tumor.
Postprandial After a meal.
Potency (1) Pharmacologic: the degree to which a compound exerts a given biochemical effect, series of effects, or aggregate effect. Potency should not be equated with efficacy, as clinical effects of both increasing and decreasing a compound's potency can be therapeutic, adverse, or neutral (2) Clinical: the degree to which erectile function is preserved"
Pouchitis Postoperative inflammation arising in an ileal pouch, a small bowel remnant (post-removal of the colon and rectum) attached to anal musculature in efforts to avoid ostomy voiding.
Preclinical The phase of product development before clinical trials usually involving animal models.
Pre-Diabetes A recently categorized condition of typcially overweight or obese people with impaired glucose tolerance at high risk for the development of type II diabetes mellitus. Many medical organizations feel that this term should become standard diagnostic nomenclature, as these individuals not yet diabetic but with impaired glucose tolerance need to be aggressively targeted for interventions aimed at diabetes prevention.
Preeclampsia Formerly known as toxemia and sometimes referred to as pregnancy-induced hypertension, preeclampsia is a condition of pregnancy, typically in the last trimester. The condition is most often characterized by the abrupt onset of hypertension (high blood pressure), leakage of large amounts of the protein albumin into the urine (albuminuria), and edema (swelling) of the hands, feet, and face. It is the most common pregnancy complication, affecting approximately 5-10% of all pregnancies, most commonly in first pregnancies. Risk factors include diabetes, twin gestation, age over 20, and family history. Preeclampsia may be a sign of placental detachment from the uterus or eclampsia, a clinical syndrome featuring seizures and coma in which both the life of mother and fetus are at risk. Current treatment consists of bed rest and sometimes supportive medication not specifically directed at the most fundamental biological processes underlying the disorder. When this treatment is ineffective, the definitive therapy is induction of labor and delivery. Preeclampsia typically resolves within 24 hours after the baby's birth.
Pre-Market Approval Application to the Food and Drug Administration to begin marketing a medical device to the public.
Preprandial Before a meal.
Presenilin 1 A gene on chromosome 14 whose germ-line mutations can predispose a carrier to Alzheimer disease. Its protein product is involved in normal amyloid precursor protein processing, and dysfunction of this gene leads to impaired amyloid precursor protein processing.
Presenilin 2 A gene on chromosome 1 whose germ-line mutations can predispose a carrier to Alzheimer disease. Its protein product is involved in normal amyloid precursor protein processing, and dysfunction of this gene leads to impaired amyloid precursor protein processing.
Pressors Short name for vasopressors.
Prevalence A term used in epidemiology indicating the number of existing cases that occur in a given group of people (population) each year (or other specified period of time). Most often expressed as number of existing cases per 100,000 populations per year. This reflects both the incidence of a condition and its survival rate, a more accurate reflection of the burden of a condition on the health care system.
Preventive Medicine A proactive approach in the practice of medicine with the goal of avoiding diseases or disease complications by methods that include early identification of risk factors for a disease.
Primary Care Physician The physician directing and overseeing an individual’s overall care plan, typically an internist (internal medicine specialist), pediatrician, or family practice physician. "
Primatized An IDEC Pharmaceuticals proprietary technology applied to monoclonal antibodies. Primatized technology uses the Macaque monkey in the manufacture of antibodies, resulting in antibodies that are so close to human structure that they are less likely to provoke an adverse immune response.
PRODAS (Élan) An acronym for Élan’s proprietary Programmable Oral Drug Absorption System, a multiparticulate drug delivery technology based on encapsulation of controlled release tablets in the diameter range of 1.5 to 4mm.
Prodrug A compound whose pharmacologic action depends upon its being converted from an inactive to an active form via metabolic processes within the body. Such conversion is called biotransformation.
Progression-free Survival The time a study subject survives from a predetermined start point, often from diagnosis or first treatment without the disease, without evidence of disease progression. Evidence of disease progression can come in the form of biochemical, imaging, or clinical markers. Any or all these forms of evidence may be required, depending on the trial.
Progressive Supranuclear Palsy A rare, degenerative brain disorder related to Parkinson disease, with early symptoms include falling, difficulty walking, imbalance, and slow movement. The actor Dudley Moore is affected with this disorder. "
ProNet Myriad's name for its proprietary system for high throughput identification of protein-protein interactions.
Prophylactic Preventive measure.
ProSpec Myriad's name for its proprietary mass spectrometry system. Mass spectrometry, an important tool for the future of proteomics, is used to determine extracellular protein interactions and structural information.
Prospective Relating to or effective in the future. A prospective study sets parameters, objectives, and outcomes to be assessed prior to initiation of the study. This ensures that study subjects are as similar as possible, and are treated as similarly as possible, in order to reduce the likelihood of confounding variables leading to erroneous cause and effect conclusions. Properly executed, the prospective, randomized, controlled trial offers the strongest evidence of clinical efficacy or effectiveness of preventive and therapeutic procedures in the clinical setting.
Prostaglandin Any of a variety of hormone-like substances derived from arachadonic acid that function in a wide range of body functions such as contraction and relaxation of smooth muscle, dilation and constriction of blood vessels, blood pressure control, and inflammation.
Prostate A chestnut-shaped gland that surrounds the beginning of the male’s urethra, it secretes a milky liquid into the prostatic urethra as semen is emitted from the seminal vesicle.
Prostate Specific Antigen (PSA) A glycoprotein (a protein with sugars attached) found in the epithelial cells of the prostate. Some can be detected at low levels in the blood of all adult men, but increased levels may be seen in men with disorders of the prostate including benign prostatic hypertrophy and prostate cancer.
Prostate-specific Membrane Antigen (PSMA) A prostate specific antigen produced by the membrane of prostate cancer cells. Its role in the normal prostate epithelium is poorly characterized.
Protease An enzyme that degrades proteins and peptides.
Protease Inhibitor (1) One of a class of drugs that mimic a specific protein chain of the human immunodeficiency virus (HIV) that protease normally degrades. This prevents HIV protease from cutting this protein into the shorter pieces that HIV needs to make new copies of itself. Often, although new copies of HIV are still made and make their way through the walls of the infected cells, these new copies are defective and unable to infect other cells. In this manner, this class of agents significantly reduces the quantity of new, infectious HIV copies manufactured inside cells (the viral load). (2) Any drug or naturally occurring substance that inhibits a protease. The most studied naturally occurring class of protease inhibitors are the so-called serpins, or serine protease inhibitors.
Proteasome A large protein complex found inside cells that degrades other proteins.
Protein One of a group of large, complex molecules consisting of chains of amino acids linked together, containing carbon, hydrogen, oxygen, nitrogen, and (often) sulfur.
Protein Kinase A (PKA) A protein involved in intracellular signal transduction in most human cells.
Protein Kinase C (PKC) A protein involved in intracellular signal transduction in most human cells.
Protein Therapeutic A protein produced either by recombinant technology or purified from a human or animal used as a drug. Also includes monoclonal antibodies.
Proteinuria Protein in the urine.
Proteomics Molecular characterization of the proteins found in an organism, with an emphasis on unraveling the systematic interactions of the proteins within the living cell. The technology involves large scale (""high-throughput"") protein separation and identification. Proteomics will provide the link between genomics and drug discovery by elucidating the function and interaction of proteins in vivo and identifying potential therapeutic targets. Proteomic research will depend in part on the development of sophisticated database and information technology tools.
Proto-oncogene A normal (wild-type) gene that plays a role in cell division or proliferation which has the potential to cause tumor formation when mutated.
ProTrap Myriad’s technology designed to screen large numbers of potential drugs for their ability to modulate a given protein’s function.
Provax Antigen Formulation IDEC’s proprietary technology applied to antigen formulation. The Provax antigen is a combination of chemical entities designed to induce cellular immunity in animals that are immunized with protein antigens. Cellular immunity is mediated in part by cytotoxic T-Lymphocytes, which are responsible for direct destruction of virally infected cells and cancer cells. This technology may prove effective in inducing both cellular and antibody immunity. "
Psoriasis A group of disorders all characterized by eruptions of specific types of skin lesions often characterized by dry, flaky skin.
Psoriatic Arthritis Joint inflammation associated with psoriasis. It affects approximately 10% of individuals with psoriasis.
Pulmonary Relating to the lungs, pulmonary arterial circulation, or the orifice leading from the right ventricle of the heart into the pulmonary artery.
Pulmonary Embolus (PE) A blood clot that has made its way into and occluded the lung's pulmonary artery or one of its branches. Most often, the source of embolus is a deep vein thrombosis (DVT) in the leg --typically, above the knee.
Pulseless Electrical Activity One of several forms of cardiac arrest, charcterized by the presence of an electrical signal on a cardiac monitor without a corresponding pulse generated by contractions of the heart's ventricles. PEA is often associated with a specific and reversible clinical condition.
Pure Red-Cell Aplasia Any of a group of conditions characterized buy the shut down of the manufacture of the precursors of red blood cells in the bone marrow. The marrow continues to manufacture precursors to white blood cells and platelets.
Pyrimidines Thymine (T), cytosine (C), and uracil (U), constituents of nucleic acids (DNA and RNA). DNA contains T and C whereas RNA contains U and C. Nucleic acids are made up of purines and pyrimidines.
Quality Adjusted Life Year (QALY) A year of life adjusted for its quality or value. A year of perfect health is equal to 1 QALY. The value of a year in ill health is discounted depending upon its severity.
Quinolone An antibiotic class that targets the bacterial enzyme DNA gyrase in order to inhibit the coiling of bacterial DNA thus interfering with bacterial replication. Quninolones include Cipro (ciprofloxacin), Floxin (ofloxacin), Levaquin (levofloxacin), Tequin (gatifloxacin), and Trovan (trovafloxacin).
Radiation A stream of particles or electromagnetic waves emitted by the atoms and molecules of a radioactive substance as a result of nuclear decay. Radiation treatment interferes with growth and replication of cancer cells by changing the structure of molecules that make up the cell's DNA.
Radioimmunotherapeutic A drug carrying radiation that hones in on its target using activities derived from our knowledge of immunology. Often involves a monoclonal antibody, with attached radiation, honing in on a specific cell surface marker.
Radio-iodine A radioactive isotope of the naturally occurring element, iodine. "
Radioisotope An isotope with an unstable nucleus that spontaneously emits radiation. The radiation emitted includes alpha particles, nucleons, electrons, and gamma rays. Radioisotopes can be used in radiation therapy to treat cancer or as tracers as in nuclear medicine scans for diagnostics.
Raf A protein involved in intracellular signal transduction that is activated by another signaling protein called ras.
Randomized A technique, based on chance distribution, of assigning patients to treatment and control groups. Refers to when neither subjects of a clinical trial nor those running the trial are allowed to choose which subjects will receive the intervention(s) being tested. Properly executed, this strategy effectively neutralizes patient prognostic factors by spreading them evenly among treatment and control groups.
Ras An intracellular signaling protein that transmits signals from outside the cell to the nucleus, resulting in cellular responses like mitosis or activation. Ras is a member of the small G protein family of proteins.
Rater-blinded A study in which those assigning clinical performance scores (such as scalar scoring systems used for the measurements of cognition, functional status, or other extents of disease involvement) are unaware of which alternative treatment is being given to each trial participant.
Rb Gene A tumor suppressor gene similar in function to p53. Rb encodes a protein that regulates cell growth and division. Rb is inactivated or mutated in many tumor cell types, including retinoblastoma, for which it is named.
Reagents Materials used for biological experiments or procedures.
Receptor (1) A protein that selectively interacts with a specific substance (ligand) in order to accomplish a specific biological task or initiate a specific series of biological activities. (2) A sensory nerve terminal that receives and responds to stimuli.
Receptor Tyrosine Kinase A type of protein found in the cell membrane that contains a ligand binding domain as well as a kinase domain. When the ligand - often a growth factor - binds, the kinase becomes active, adding phosphates to the amino acid tyrosine on target proteins.
Recombinant DNA DNA that results from the insertion of a (nucleotide) sequence not originally present.
Recombinant Proteins Proteins made from recombinant DNA technology wherein engineered DNA coding for a specific therapeutic protein of choice facilitates the protein’s mass production. Recombinant proteins such as Humulin (human insulin) and Epogen (erythropoetin) offer a relatively safe and inexpensive alternative to animal-based proteins with, often, a better adverse effect profile. These proteins led to the birth of the biotechnology industry.
Red Blood Cells The blood cells responsible for carrying oxygen, via hemoglobin, to tissues. a.k.a. erythrocytes.
Refractory Resistant to treatment.
Regulatory Region The DNA adjacent to genes which regulates when and where the gene is active (transcribed into mRNA and translated into a protein). Regulatory DNA includes promoters, enhancers, silencers, and other elements.
Rehabilitation Specialist (1) Physicians with a specialty in physical medicine and rehabilitation, a.k.a. physiatrists. (2) Non-physician specialists in physical medicine and rehabilitation, physical therapy, occupational therapy, or speech and language therapy.
Rejection The refusal by the body's immune system to accept transplanted cells, tissues or organs. For example, a kidney transplanted may be rejected.
Relapse noun: A recurrence of disease symptoms after a period of improvement. intransitive verb: To return to a former worse state.
Renal Cell Carcinoma Cancer that develops in the lining cells of the renal tubules. The tubules are responsible for blood filtration and urine production.
Reperfusion Injury Cell and tissue damage that occurs in certain instances when blood flow is restored to cells that have been previously deprived of blood flow. This paradoxical occurrence often proceeds at an accelerated pace compared to the original injuries. Tissues sustain a loss of cells in addition to those that were irreversibly damaged at the end of the initial deprivation. The goal of medical therapy is to decrease the fraction of cells that may otherwise be destined to die in the area at risk.
Resection The surgical removal of a portion of a tissue or organ, often with accompanying pathology such as tumor or walled-off infection (abcess).
Respiration A fundamental life process in which oxygen oxidizes organic fuel molecules to provide energy, carbon dioxide, and water.
Respiratory Disease Disorders of gas (primarily oxygen and carbon dioxide) exchange and disorders of the organs, tissues, cells, and airway passages that participate gas exchange. "
Restenosis Narrowed again. Most often used to describe the re-narrowing of the lumen (channel) of a blood vessel after a procedure had been successful at widening it. Coronary artery stenting is a common procedure after which restenosis might ensue.
Restriction enzymes "Molecular scissors," they are enzymes that "recognize" and "cut" specific DNA sequences. Knowledge of the cutting sites for such enzymes allows one to reliably cut DNA sequences at specific sites for either manipulation such as occurs in recombinant DNA activities or discovery of the sequences of DNA such as occurs in gene discovery or clinical diagnostics.
Retention Enema A rectal injection of a therapeutic or diagnostic substance introduced at a low pressure and allowed to dwell for several hours before expelling.
Retinoic Acid An acidic form of vitamin A with many functions in the body, especially in regulating development.
Retrospective Of, relating to, or given to retrospection, the act or process or an instance of surveying the past. Retrospective studies are any of several sorts of observational (not experimental) investigations that provide weak empiric evidence because of the potential for large confounding biases to be present when there is an unknown association between a factor and an outcome. The greatest value of observational, retrospective study is that they provide preliminary evidence that can be used as the basis for hypotheses in stronger experimental, prospective, controlled trials.
Retrovirus A family of viruses that carries RNA and is able to transcribe DNA from its RNA using an enzyme called reverse transcriptase. The human immunodeficiency virus (HIV) is a retrovirus.
Retrovirus A family of viruses that carries RNA and is able to transcribe DNA from its RNA using an enzyme called reverse transcriptase. The human immunodeficiency virus (HIV) is a retrovirus.
Revascularize To restore patency to occluded blood vessels, especially by an invasive procedure. Examples include coronary artery bypass grafting (CABG) and percutaneous coronary interventions (PCIs) for coronary arteries, carotid endarterectomy and cerebrovascular interventions for arteries leading to the brain, and other bypass surgeries and percutaneous interventions for peripheral arteries.
rGelonin A recombinant form of gelonin attached as a payload to monoclonal antibodies. Gelonin is a cytotoxin, and thus improves the ability of the monoclonal to kill target cells. rGelonin was developed by Xoma.
Rheumatoid Arthritis A chronic disease characterized by progressive joint cartilage inflammation.
Riboflavin Vitamin B2.
Ribonucleotide Reductase An enzyme that makes DNA from RNA by removing an oxygen molecule.
Ribosome A cell's protein factory, it is a structure located in the cytoplasm and is composed of two subunits, one larger than the other (30S,50S). Molecular constituents include both ribosomal RNA (rRNA) and protein. The active site, made of rRNA, resides on the large subunit, and is site of the chemical reaction that converts genetic information from mRNA into the protein building blocks known as amino acids. "
Ribozyme A molecule of RNA with enzyme like properties. Ribozymes were probably the precursors to protein enzymes during the development of life on earth. Ribozymes that can cleave specific RNA sequences are being investigated as potential therapeutics.
Ringer's Lactate A solution of electrolytes and lactate used to increase circulating volume so that enough oxygen-carrying blood reaches vital organs.
Risk Factor Any factor that is statistically related to an increased or decreased risk of a person's getting a disease. Different diseases have different risk factors.
Rizatriptan Generic name of Merck’s Maxalt formulations, the 5-HT 1B/1D agonist is indicated in acute migraine.
RNA (Ribonucleic Acid) A nucleic acid found in cells that have nuclei. RNA serves as a messenger between different structures in the cell and plays a key role in the synthesis of proteins based on the "instructions" received from DNA. In some viruses, RNA serves as the genome.
RNA Interference A technique that uses double stranded RNA to inhibit translation of specific mRNA into protein. The technique has been successful in model organisms, and is potential superior to antisense technology.
RNAse An enzyme that degrades RNA.
Saline A solution of varying concentrations of sodium chloride (NaCl) in water. “Normal Saline,” sufficient in most therapeutic situations, is of 0.9% NaCl, virtually the same salinity found in most mammalian cells and in the blood.
Schizophrenia The most chronic and disabling of the major mental illnesses, its most prominent manifestation is an inability to distinguish real from unreal experiences. It affects approximately 1% of most populations including that of the U.S.
Sclerosis Localized hardening of a tissue, most often due to post-inflammatory scarring.
Secreted Protein A protein secreted by cells that enters the extracellular space. Secreted proteins, such as hormones and growth factors, are often excellent drug targets, especially for recombinant proteins and monoclonal antibodies.
Seizure Disorder One of several conditions all characterized by symptoms referable to pathologic electrical brain activity. Common symptoms include: uncontrollable motor activity (a.k.a. convulsion, epileptic attack), unusual sensations, and clouding or loss of consciousness.
Selected Lymphocyte Antibody Method (SLAM) The proprietary technique acquired from ImmGenics by Abgenix that allows efficient production of monoclonal antibodies from blood cells, an alternative to hybridoma generation.
Selectin A family of adhesion molecules found on white blood cells and other cells. Selectins include p-selectin, l-selectin, etc.
Selegiline The generic name of Somerset’s Eldepryl formulation, it is a monoamine oxidase B inhibitor which prevents the breakdown of dopamine in the brain, and is indicated, in combination with l-dopa/carbidopa, in the management of signs and symptoms of Parkinson disease.
Sensitivity A characteristic of a diagnostic test that describes the degree to which the test detects a disorder when it is truly present. It is the proportion of all disordered patients for whom a positive test is obtained.
Sepsis Systemic Inflammatory Response Syndrome due to infection.
Sepsis Spectrum The aggregate of the conditions known as the systemic inflammatory response syndrome, sepsis, severe sepsis, septic shock, multi organ-system failure, the diffuse capillary leak syndrome, and the adult/acute respiratory distress syndrome. This series of conditions can occur as a result of the over stimulation of various immunologic and blood coagulation and fibrinolytic pathways. "
Septic Shock Severe sepsis with hypotension (systolic BP < 90mmHg) despite adequate fluid resuscitation or with the requirement for vasopressors (agents that constrict blood vessels)/inotropes (agents that increase the pumping power of the heart) to maintain blood pressure.
Serious Adverse Event An adverse event that results in death, is life threatening, requires inpatient hospitalization, prolongs existing hospitalization, or results in persistent or significant disability or a congenital anomaly (birth defect).
Serotonin 5-hydroxytryptamine, produced by platelets in the blood, it serves to constrict blood vessels, inhibit specific stomach (gastric) secretion, and stimulate smooth muscle. Its highest levels, normally, are in specific central nervous system regions (basal ganglia and hypothalamus). Modulation of serotonin and/or its receptors have been used in the treatment of depression, anxiety, schizophrenia, eating disorders, obsessive compulsive disorder, migraine, and panic disorder.
Serum The acellular, clear liquid that can be separated from clotted blood.
Severe Chronic Neutropenia A group of rare conditions, characterized by low, circulating levels of neutrophils, that can be either the result of a single gene defect or acquired for unknown reasons.
Severe Combined Immunodeficiency (SCID) A congenital immune deficiency typically characterized by a severe defect in both the T cell and B cell systems that leads to one or more serious infections within the first few months of life. The infections may even be life threatening.
Severe Sepsis Sepsis with evidence of organ hypoperfusion (not enough blood, and therefore oxygen, getting to organs).
Shingles Herpes Zoster infection. Typically, it is an acute, localized infection with the virus Herpes varicellae (a.k.a. Varicella zoster) that causes painful blistering eruptions. The infectious agent is the same virus that causes chickenpox. Shingles, though, occurs because the virus had become inactive after chickenpox and re-emerges many years later in this manner.
Short Stature Height at least two standard deviations below the mean height-for-age-and-gender given one’s ethnic background.
Shy-Drager Syndrome A progressive, degenerative disorder of the central and autonomic nervous systems, it is characterized by an excessive drop in blood pressure causing lightheadedness or momentary blackouts upon standing or sitting up, and may include features of Parkinson disease.
Single Gene Disorder A disorder whose genetic contribution is from a defect in only one gene. These disorders, many rare, but in aggregate quite common, help clinical investigators explore the ‘proof-of-principal’ necessary to move gene therapy forward"
Single Nucleotide Polymorphism (SNP) Variations in a single nucleotide base that occur in DNA. In humans, these variations occur at a frequency of one every 1,000 bases. These variations can be used to track inheritance in families. SNP is commonly pronounced ""snip." SNP discovery refers to the identification of a specific location within a DNA sequence where there is variation in a single nucleotide base across a population. SNP scoring refers to the measurement of the presence or absence of a particular SNP in the DNA sequence of a particular individual. Used in pharmacogenomics as a tool for diagnostics, drug discovery, clinical trials, and for anticipating drug responses.
Sinusitis Sinus inflammation, often with an infectious component.
Smad Proteins Any of several transcription factors and related molecules that assist with the intracellular signal transduction of cellular interactions initiated by transforming growth factor (TGF)-b and bone morphogenic protein. Developmental products that target Smad proteins might have applications in wound healing, tissue repair, pulmonary, vascular, and oncologic scenarios.
Small Inhibitory RNA (siRNA) A short double stranded RNA molecule, usually less than 30 nucleotides long, that is an intermediate in RNA interference. siRNAs can inhibit specific mRNA translation in mammalian cells.
Small Molecule Library An institution’s reservoir of prepared small molecule compounds. Such libraries are maintained in order to facilitate compound screening and target validation. Small molecules interacting favorably with a target may become drug compound candidates whilst the target may, thus, become validated.
SODAS (Élan) An acronym for Spheroidal Oral Drug Absorption System, it is the proprietary, controlled release, bead-based, delivery technology on which Élan was founded. "
Sodium Channels Proteins found on the surface of brain and spinal cord nerves, peripheral nerves, heart, kidney, intestine, smooth and skeletal muscle, and other tissues and cells, they are involved in transmitting sodium-mediated electrical signals.
Solid Tumor A tumor classification typically used to describe a tumor that does not have its origin in a blood or lymph (hematopoetic) cell.
Solubility The degree to which a solid can dissolve.
Somatic Cell All cells other than the germ cells or gametes in an organism.
Somatic Cell Nuclear Transfer (SCNT) “Cloning” technique which involves the transfer of the nucleus (containing the vast majority of the cell’s DNA) from an adult cell into a de-nucleated pluripotent cell.
Sonic Hedgehog A protein that signals cells, especially those in the nervous system, to progress down specific developmental pathways. Sonic hedgehog was originally isolated as a vertebrate homologue to the fruit fly protein hedgehog, which is also involved in development.
Spasticity A state of increased muscle tone and exaggerated deep tendon reflexes causes by pathology involving “upper motor neurons,” those neurons that originate in the brain and travel down specific paths to the spinal cord where they meet and communicate with a specific (“lower motor”) neuron that then extends to a specific target on a muscle. "
Specificity A characteristic of a diagnostic test that describes the degree to which the test does not detect the presence of a disorder when it is truly not present. Specificity is the proportion of non-disordered patients for whom a negative test is obtained.
Speculoscopy The direct visualization of the cervix via a speculum attached to a chemiluminescent source.
Speculum A device used to widen an opening to look within a passage or a cavity. Vaginal speculums are employed so that the cervix is more easily visible, a nasal speculum aids visualization into the nostrils, and an ear speculum helps one to look within the ear canal at the eardrum.
Sphingosines The principal long chain base found in sphingolipids, those lipids that are important constituents of nerve tissue. Examples of sphingolipids include: ceramides, cerebrosides, gangliosides, and sphingomyelins. "
Spinal Cord The column of nervous tissue extending from the medulla of the brain stem to the 3rd lumbar vertebra in the spinal canal. All nerves to the trunk and limbs emanate from this tissue, and it is the center of reflex action in which are the conducting neural paths to and from the brain. "
Spinal Fusion A surgical procedure in which two or more spinal vertebrae are joined to one another by bone. The most prevalent indication for the procedure is for low back vertebral instability (when there is abnormal motion between the vertebrae) such as occurs in spondylolisthesis ("slipped disk") and after certain surgical procedures of the area. Other indications may be scoliosis or pain syndromes due to disk disease.
Spinal Muscular Atrophy Syndromes A group of neuromuscular disorders all characterized by a disease of the “anterior horn cell,” a cell leading from the spinal cord to all various skeletal muscles of the trunk and extremities. The degeneration of these cells eventually disengages nervous input from muscles such that the muscles wither away, atrophy, and death ensues from respiratory failure from lack of nervous input to the diaphragm and related musculature. "
Spinocerebellar Ataxia A group of degenerative disorders of the cerebellum and spinal cord characterized by progressively worsening wobbly walking, muddled speech, and dysfunction of cranial nerves.
Stable Disease Variably defined circumstances characterized by neither response to therapy nor progression of disease.
Standard of Care Medically usual attention and intervention as defined by a given reference from global, international, national, or regional medical societies, governmental concerns, or other communities. Legally, the degree of attention and intervention a reasonable person would take to prevent an injury, or other harm, to another.
Statin Any of several drugs known to inhibit the activity of the cholesterol synthetic enzyme, 3-hydroxy-3-methylglutaryl-Coenzyme A (HMG CoA) reductase. This colloquial designation derives from the fact that branded compounds of this class have their names end in “-statin.” The cholesterol lowering properties of the agents are great enough to confer a decreased risk of cardiovascular events on consistent users. Recent evidence suggests that not only do these drugs target HMG CoA reductase, but also various proteins involved in the maintenance of blood vessel architecture.
Stem Cell A relatively undifferentiated cell that has the ability to develop into many, if not all, cell types in the body.
Stenting The deployment of a prosthetic tube into a vessel or passageway to keep it open. The prosthesis is called a ""stent,"" and they are primarily used in narrowed coronary arteries in order to help keep them open after balloon angioplasty. Once deployed, the stent permits the normal flow of blood and oxygen to the heart. Stenting narrowed carotid arteries (the vessels in the front of the neck that supply blood to the brain) may prove useful in treating patients at elevated risk for stroke. In the esophagus, stents treat certain types of constriction, and they can be deployed in the ureters to maintain the drainage of urine from the kidneys, and in the bile duct to keep it open. "
Stereotactic Implies precise localization of a target in three-dimensional space, typically for purposes related to biopsies, surgeries or radiation therapy.
Steroids (1) A group name for compounds that contain a cyclopenta-a-phenanthrene ring system. Some of the substances included in this group are progesterone, adrenocortical hormones, the gonadal hormones, cardiac aglycones, bile acids, sterols (such as cholesterol), toad poisons, saponins, many other hormones, and some of the carcinogenic hydrocarbons. (2) Colloquialism for natural and synthetic compounds having biological actions similar to those of steroid hormones, especially those of the adrenal cortex — so-called “corticosteroids” — that have potent anti-inflammatory properties and are pharmacologically useful agents for this reason.
Streptokinase A purified thrombolytic (clot buster) also known as anistreplase, derived from a class of bacteria known as group C beta-hemolytic streptococci.
Striatonigral Relating to the striatum and the substantia nigra.
Striatum A group of brain cells involved in motor (movement) and cognitive planning, many of its connections are to the basal ganglia.
Stroke An injury of the brain due to bleeding or to an interruption of the blood supply.
Structural Genomics The generation of three dimensional structural information about proteins and the use of that information, using advanced computational methods, to predict compounds capable of interacting with the proteins and effecting their functions. Structural genomics techniques are potentially useful in identifying new drug compounds for particular protein targets.
Subcutaneous Beneath the skin.
Substantia Nigra Part of the basal ganglia, this region of cells utilizes many neurotransmitters, but is known most of all for its dopamine using neurons, destroyed in the development of Parkinson syndrome. It plays a specific role in the coordination of movement.
Sulfonylurea Any of several hypoglycemic compounds related to the sulfonamides and used in the oral treatment of diabetes mellitus.
Sumatriptan The prototypic triptan (5-HT 1B/1D agonist), it is the generic name of Glaxo Wellcome’s Imitrex, and is indicated in acute migraine (injection, nasal spray, and tablets) and cluster headaches (injection).
Supplemental Biological License Application (sBLA) A BLA submitted to the FDA applying for a drug’s approval for additional indications. Implies a desire to expand the drug label.
Surrogate Marker A measurement of a compound’s biological activity, or effects on sub-clinical levels such as radiographically / imaging or blood test results, that substitutes for a true clinical endpoint such as pain relief, functional and quality of life improvements, disability, or death.
Synapse The connection between two neurons or a neuron and a muscle cell, gland, or other cell. "
Synaptic Cleft Gap between two neurons where neurotransmitters are released, allowing the neurons to communicate.
Synaptic Transmission The transmission of information from one neuron to another or from a neuron to a muscle cell.
Synergy When combination of two stimuli are greater the sum of their individual effects.
Synovial Fluid Fluid that lubricates joints and provides nutrients to cartilage. Also known as the synovia.
Systemic Throughout the body; not localized.
Systemic Inflammatory Response Syndrome (SIRS) A serious medical condition typically manifest by at least two of the following: high or low core body temperature, rapid heart rate (tachycardia), rapid respiratory rate (tachypnea), and an elevated white blood cell count. Approximately 15% to 30% of all patients admitted to an intensive care unit (ICU) have this condition. Cytokines, dysfunctional immune cells, and the inability of tissues to obtain and utilize oxygen interact in a complex manner that leads to a severe dysregulation of immune and metabolic response of the patient. Persistent SIRS leads to multi organ-system failure, which accounts for approximately 70% of deaths in the ICU. Blood-borne infection, trauma, burns, pancreatitis, inborn errors of metabolism (inherited enzyme defects) and anaphylaxis are the most common causes of this syndrome.
Systemic Lupus Erythematosus (SLE) An autoimmune connective tissue disease with an amazing array of possible signs and symptoms. Antibodies against nucleic acids typically underlies its pathology.
T Cell A white blood cell derived from the thymus that plays a key role in the immune response against viruses or against "foreign cells" including transplanted organs and tissues and cancer cells.
Tachycardia Rapid heart rate.
Tachyphylaxis The rapid onset of a progressive decline in clinical response following repetitive administration of a pharmacologically or physiologically active agent.
Tachypnea Rapid respiratory rate.
Tangles Short for "neurofibrillary tangles," the pathological hallmark of degenerate brain nerve cells affected by Alzheimer disease. They are made up of a renegade version of a protein called tau.
Target Molecules, parts of molecules, molecular processes, and constituents of molecular processes that can be affected by a drug. The best targets tend to have the greatest degrees of specificity.
Target Validation The process of demonstrating, by a variety of techniques, that a drug interacting with a given target could have a therapeutic effect.
Tau A poorly-understood, microtubule-associated protein, it is the predominant protein component of the paired helical filaments and neurofibrillary tangles characteristic of specific pathological lesions of the Alzheimer disease brain. One of its normal roles involves assistance in the formation of microtubules, elements in cytoplasm that participate in chromosome movement during phases of the cell cycle leading to cell division.
Telomerase The enzyme required for replication of the ends of chromosomes, known as telomeres. Dividing cells must express telomerase to avoid having chromosomes shrink with each cell division, which eventually results in apoptosis. Cancer cells often activate telomerase expression.
Telomere The DNA located at the end of each chromosome. The enzyme telomerase is required to properly replicate telomeres.
Teratogen An drug or other agent that causes abnormal embryonic or fetal development (birth defects).
Testosterone Male sex hormone (androgen) secreted by the interstitial cells of the testis and responsible for triggering the development of sperm and of many secondary sexual characteristics.
Tetracycline An antibiotic class that targets the 30S ribosomal subunit of bacteria, blocking the ability to synthesize proteins. "
TGFb A cytokine (small signaling molecule) produced by certain white blood cells that signals other cells to divide or perform other cellular functions. Originally identified as a T cell growth factor.
TGFb Superfamily The family of related cytokines (small hormone like signaling proteins) that effect the growth of white blood cells and other cell types. Originally isolated as a tumor growth factor.
Thalidomide N-(2,6)-dioxo-3-(piperidyl)phthalimide, originally indicated as a sedative-hypnotic, it is an agent that has been discovered to have anti-angiogenic and immunomodulatory effects. The generic of Celgene’s Thalomid, it is indicated for the treatment of the skin condition, erythema nodosum leprosum, in leprosy and is being investigated in several anti-cancer settings.
Therapeutic Used to treat a disease or condition, as opposed to diagnostic or for research.
Thrombin The fundamental clotting enzyme that converts fibrinogen to fibrin after a long series of molecular events initiates blood clot formation. Interference with its action has an anticoagulant effect.
Thrombocytopenia A reduction in circulating platelets, which are also known as thrombocytes. Thrombocytopenia may be due to increased destruction or decreased production. Chemotherapy, radiation, and HIV infection are common causes for decreased production, while the administration of the anticoagulation agent heparin, and various immunologically mediated mechanisms, including those contributing to idiopathic thrombocytopenic purpura (ITP) are common causes for increased destruction. The preferred treatment is to correct its underlying cause. However, when that is not possible, and the patient is at a high risk for hemorrhage, a platelet transfusion may be used. Pharmacologic intervention may, infrequently, be used to reduce the need for transfusions.
Thrombolytic An agent that, by virtue of interfering with specific biochemical pathways, effectively dissolves clots (thrombi), thereby re-opening vessels. Streptokinase, tPA, Urokinase, Alteplase, and Reteplase are examples.
Thrombus Blood clot.
Thyroid Located in the lower portion of the neck, it is a butterfly-shaped gland, an organ that produces hormones essential for the function of virtually every cell in the body. The thyroid hormones are involved in the regulation of metabolic biochemical reactions as well as growth and development. The thyroid manufactures and stores hormones that are most well-known for the regulation of heart rate, blood pressure, body temperature, and the rate at which food is converted into energy.
Tissue Factor A protein that, along with Factor VIIa and others, is responsible for triggering the blood clotting system in normal hemostasis (bleeding arrest) and the majority of cases of abnormal hemostasis resulting in thrombotic (clotting) diseases. Interruption of its activities may be therapeutic when abnormal hemostasis is implicated in a disease process.
Tissue Plasminogen Activator (tPA) One of a group of related proteins whose most prominent biological role is in breaking down blood clots. Recombinant analogs are the thrombolytics, clot-busters, Activase and TNKase from Genentech and Retevase from Johnson & Johnson.
Topical Relating to a surface area. Applying a topical agent to a given area on the skin, for instance, is intended to affect only that area. Whether the agent's effects prove limited to that area generally depends upon whether it stays there or is absorbed into the bloodstream or transferred to another area (such as the eye).
Totipotent Stem Cell A stem cell that can develop into any cell type (i.e. the cell formed by the fusion of the egg and sperm).
Toxicology The science that deals with poisons and their effects (pharmacologic, clinical, industrial, or legal). The pharmacologic and clinical aspects are considered in basic pharmacokinetic studies.
Toxin A compound that is poisonous.
Transchromosomal Mouse A transgenic mouse that contains an extra, artificial chromosome.
Transcription Factor A protein that binds to DNA in order to guide and activate a different molecule, called RNA polymerase, whose function it is to synthesize RNA from DNA. Once formed, the RNA is most often translated into a protein.
Transdermal Relating to, being, or delivering a medication in a manner for absorption through skin into the subcutaneous tissue and/or bloodstream.
Transfusion The transfer of blood or blood products (components such as cells or plasma) from the bloodstream of a donor into that of a recipient. Transfusion of one's own blood (autologous) is the safest method but requires planning ahead and not all patients are eligible. Directed donor blood permits the recipient to receive blood from known donors. Typically, however, volunteer donor blood is most readily available and, when properly tested is associated with a low incidence of adverse events. "
Triglycerides A lipid form whose levels correlate with very low density lipoprotein (VLDL) and chylomicron levels. Very low density lipoproteins tend toward being a “bad” cholesterol element, as elevated levels of VLDL can predispose to coronary artery disease and elevated levels of chylomicrons can predispose to acute pancreatitis and other conditions. See “cholesterol” entry.
Triple Blind Bias-minimizing study technique relying upon analytic statisticians' lack of knowledge of which study participants are in which comparison group(s) in addition to the double-blind bias-reduction technique of both the participants and the investigators kept unaware of which participants are in which group(s).
Triptans Class of anti-migraine drugs that are 5-HT 1B/1D agonists, and act on serotonin receptors in the brain and cause blood vessels to narrow (constrict) by virtue of an actual or mimicked elevation in serotonin.
Tuberculosis A disease caused by infection with Mycobacterium tuberculosis. Although it can affect any tissue or organ, the most common site of pathologic infection is the lungs"
Tubulin A protein that polymerizes to form microtubules, a part of the cytoskeleton within cells. Microtubules are important for cell stability and various cell-associated movements, such as cell division and the movements of motile cells such as sperm or cilia.
Tumor A growth characterized by abnormal rate of growth and abnormal structure. Tumors can be benign (not cancer) or malignant (cancer).
Tumor Classification Categorizing tumors. This ranges from simple categories such as site of origin and whether the tumor is benign of malignant to more complex categories that include distance of metastatic spread and the degree to which the tumor cells resemble normal cells of the same tissue. Molecular, genomic, and proteomic technologies increase the ability to sub-categorize tumors in novel ways with the hope that such precision will translate into better drug targets and other means of enhanced ability to make medical intervention. "
Tumor Necrosis Factor a (TNFa) A cytokine (small signaling molecule) produced by macrophages and other white blood cells that signals other cells to divide or perform other cellular functions. Along with IL-1, TNFa is a principal mediator of inflammation.
Tumor Suppressor Gene A gene whose encoded protein normally limits the growth and division of cells. When a tumor suppressor gene is mutated, it may fail to keep a cancer from growing. BRCA1 and p53 are well-known tumor suppressor genes.
Turner Syndrome A genetic disorder affecting females who have either only one X chromosome in some or all cells or has two Xs, but one is damaged. Signs include short stature, delayed skeletal growth, short fourth and fifth fingers, broad chest, and sometimes cardiovascular abnormalities. Women are typically infertile due to ovarian failure. Treatment may include human growth hormone and estrogen replacement therapy. "
Type I Error In hypothesis testing, the conclusion that a treatment or intervention has a desired effect when it really does not. Also known as a false positive or alpha error.
Type II Error In hypothesis testing, the conclusion that a treatment or intervention does not have a desired effect when it really does. Also known as a false negative or beta error.
Ulcer Erosion of either mucous membrane or skin resulting in its concavity and depression below the level of surrounding tissue.
Ulcerative Colitis A form of inflammatory bowel disease characterized by, amongst other things, chronic inflammation of the colon and ulcerations of its lining.
Uveitis Inflammation of the uvea. Collectively, the uvea refers to the iris, the choroid, and the ciliary body. The iris is the colored portion of the eye that immediately surrounds the pupil. The choroid is the thin middle layer of the eye between the sclera (the white of the eye) and the retina (the nerve layer that lines the back of the eye, senses light, and creates impulses that travel through the optic nerve to the brain). Many blood vessels run through the choroid. The ciliary body is tissue that connects the iris with the choroid and includes muscles that act on the lens to change its shape.
Vaccine Historically, a preparation of modified microorganisms or molecules derived from them used in prophylaxis against various infectious diseases. Novel vaccines use a modified immune system stimulant, implicated in a given disorder, in order to generate an immune response to that stimulant such that its pathological development, or related pathological developments, undergo immune surveillance and are prevented. "
Vaccinia (1) A localized skin infection caused by inoculation with the vaccinia virus that can move throughout the body in patients with inadequate immune systems. (2) A virus thought possibly to be a hybrid of two others called variola and cowpox, it is the agent used to induce immunity to smallpox (variola being the causative agent in smallpox).
Validated Target A drug target, usually a protein, that has been demonstrated by any of a variety of methods to have therapeutic potential.
Valproate Valproic acid or its sodium salt, it is an anticonvulsant used in the management of seizure disorders.
Variable Region The half of the antibody molecule that binds to its antigen.
Vascular Endothelial Growth Factor (VEGF) A growth factor known to be necessary for angiogenesis.
Vasculature The blood vessel network of a tissue, an organ, an organ system, or the body as a whole.
Vasopressor A substance that elevates arterial blood pressure by constricting blood vessels. These agents are frequently used in intensive care units when patients have refractory hypotension.
Vector In molecular biology, a vector is the carrier for DNA or RNA that allows the nucleic acid to be delivered and integrated into the target cell. A vector is itself usually made of DNA, containing sequences that instruct the target cell to maintain and replicate the vector and the DNA it is carrying.
VEGF Vascular endothelial growth factor, a pro-angiogenic molecule.
Venous Insufficiency Failure of the veins to perform their physiologic roles. The most important dysfunction in this condition is their decreased effectiveness in carrying blood, from which oxygen has been extracted by tissues, back to the lungs to pick up oxygen.
Venous Thromboembolism (VTE) Aggregate of deep vein thrombosis and pulmonary embolism
Venous Ulcers Localized loss of a varying depths of skin and subcutaneous tissue typically occurring over or near the ankles, and possibly occurring at any area drained by incompetent or obstructed veins. Also known as venous stasis ulcers.
Viral Phage A virus that infects bacteria. Can be used in the laboratory to carry recombinant DNA.
Virus A non-cellular microbial entity that consists of a core of DNA or RNA enclosed in a coat of protein. The virus is not a cell, and can live and reproduce only by invading susceptible cells and commandeering the reproductive capabilities of that cell, causing it to reproduce viral progeny. Viruses can infect bacteria, plant and animal cells. Viruses can be modified to carry therapeutic genes for gene therapy.
Vitamin B2 Riboflavin
Waldenstrom Macroglobulimemeia A condition in which a proliferation of lymphocyte-like or plasma cell-like cells are associated with the development of huge circulating proteins in people that may come to attention because of anemia and large organs like the spleen, liver, or lymph nodes.
Warfarin An anticoagulant drug that works by suppressing the production of clotting factors that rely on vitamin K for their production: factors II (prothrombin), VII, IX, and X. Originally isolated from a spoiled sweet clover that causes cattle to hemorrhage an die, it (more accurately, its original form) is named after its original patent holder, WARF, the Wisconsin Alumni Research Foundation. Its effectiveness in controlling pestilent rats and mice initially led to its commercial success. It has evolved into the most widely prescribed anticoagulant drug for people, and has saved countless lives.
Wells Small containers, similar to a tiny test tubes, used to grow cells or perform experiments in the laboratory. Culture dishes are often made with 96 wells each for screening monoclonal antibodies.
White Blood Cells Various types of blood cells involved in fighting infection. These cells are responsible for both therapeutic and pathological inflammation. Typically, they are divided into three subcategories, each with multiple further classifications: myeloid (from the marrow), lymphoid (from the lymph system), and monocyte/macrophage (scavengers).
XenoMouse Abgenix's proprietary transgenic mouse strain that contains human antibody genes and produces fully human antibodies.
Xenopus An African frog widely used in the study of vertebrate development.
X-linked Referring to genes and other DNA sequences residing on the X chromosome. X-linked traits are observed predominantly in males.
X-Ray Crystallography The technique for solving the three dimensional structure of complex molecules by forming a crystal of the molecule and analyzing the crystal's X-ray diffraction pattern. This technique was used to solve the structure of DNA, and is now used to solve the structure of proteins.
Yeast 2-Hybrid Analysis A technique that allows researchers to identify protein-protein interactions.
Yeast Model System Many researchers use yeast to do experiments that require nucleated cells (as opposed to bacteria) but take advantage of the rapid growth and uniformity of single celled organisms.
Yttrium-90 A radioactive isotope of the naturally occurring metallic element, Yttrium. It is used in the preparation of radiopharmaceuticals, typically for pituitary ablation and more recently for delivery of radiotherapy to specific cells primarily in cancer.
Zidovudine (AZT) Chemically known as azidothymidine, it is a drug used against human immunodeficiency virus (HIV), typically in combination with other drugs. Branded formulations are GlaxoSmithKline's Retrovir and Combivir, the latter also containing another anti HIV drug, lamivudine (3TC), for convenience.
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