Glossary

A

Active Pharmaceutical Ingredient (API)

It is the substance in a pharmaceutical drug that is biologically active.

Allogenic

Being generically different although belonging to or obtained from the same species.

Anemia

A pathological deficiency in the oxygen-carrying component of the blood, measured in unit volume concentrations of hemoglobin, red blood cell volume or red blood cell number.

Angina Pectoris

Acute pain in the chest resulting from not enough blood reaching the heart muscle myocardial ischemia; pain is usually brought on by physical activity or emotional stress when those things increase the need for blood to supply the heart muscle. Some patients can predict the kinds of events that will precipitate an attack while others cannot. This condition is seen more often in men than in women, and in older persons than in younger persons. It is not a disease entity but a symptom of underlying disease in the arteries that supply the heart muscle. About 90 per cent of all cases can be attributed to coronary atherosclerosis.

Antiinflammatory

Counteracting or suppressing inflammation.

Antiischemic

Inhibiting or preventing ischemia.

Antineoplastic

Inhibiting or preventing the growth or development of malignant cells.

Antithrombotic

Preventing or interfering with the formation of a thrombus or blood clotting.

Apoptosis

Programm cell death; this physiological process is necessary for the elimination of superfluous, diseased, or damaged cells and the formation of new cells.

Ascites

An effusion and accumulation of serious fluid in the abdominal cavity.

Autologous

Derived or transferred from the same individual’s body.

C

Cancer

A group of diseases characterized by abnormal cells that grow and spread uncontrollably. A mass, or collection, of cancer cells called a malignant tumor frequently grows rapidly, invading and destroying nearby tissue. Cancer cells often travel to other body parts where they grow and replace normal tissue. This process, called metastasis, occurs as the cancer cells get into the bloodstream or lymph vessels.

Chemotherapy

A chemical that binds to and specifically kills microbes or tumor cells. In oncology, a drug therapy for cancer.

Chronic Lymphocytic Leukemia (CLL)

The most common form of leukemia in adults, in which the lymphocytes may look fairly normal but are not fully mature and do not deal effectively with infection. The malignant cells are found in the blood and bone marrow, collect in and enlarge the lymph nodes, and may crowd out other blood cells in the bone marrow, resulting in a shortage of red blood cells (producing anemia) and platelets (producing easy bruising and bleeding).

Clinical Trials

Drug testing in humans to assess safety, side effects and efficacy.

Coagulation

The process of clot formation.

Compassionate Use

A method of providing experimental therapeutics prior to final FDA approval for use in humans. This procedure is used with very sick individuals who have no other treatment options. Often, case-by-case approval must be obtained from the FDA for compassionate use of a drug or therapy.

Contract Research Organization (CRO)

An organization (commercial, academic, or other) contracted by the sponsor to perform one or more of a sponsor’s trial-related duties and functions. CROs are independent companies carrying out specialized functions of Pharmaceutical Research and Development such as Phase I, Phase II, Phase III, or Phase IV clinical trial.

Cytoprotective

Protecting cells from noxious chemicals or other stimuli.

Cytotoxic

Having a deleterious effect upon cells.

D

Deep Vein Thrombosis (DVT)

A blood clot (thrombus) in a deep vein in the thigh or leg. The clot can break off as an embolus and make its way to the lung, where it can cause respiratory distress and respiratory failure. DVT is sometimes called the “economy-class syndrome”. Even in young, healthy travellers, long stretches of time spent immobilized in the cramped seat of an aircraft with very low humidity sets the stage for formation of a blood clot in the leg.

Deoxyribonucleotide

A nucleotide having a purine or pyrimidine base bonded to deoxyribose, which in turn is bonded to a phosphate group.

E

Eco-Management and Audit Scheme (EMAS)

A management tool for companies and other organizations to evaluate, report and improve their environmental performance.

Embolism

The sudden blocking of an artery by a clot of foreign material (embolus) that has been brought by the blood current to the place where it has become lodged. The obstructing material is most often a blood clot, but it may be a fat globule, air bubble, piece of tissue, or clump of bacteria.

Endothelial

Pertaining to or made up of endothelium.

Endothelial cell

A thin, flattened cell, a layer of them lines the inside surfaces of body cavities, blood vessels, and lymph vessels, making up the endothelium.

Endothelium

The layer of epithelial cells that lines the cavities of the heart and of the blood and lymph vessels, and the serous cavities of the body.

European Agency for the Evaluation of Medicinal Products (EMEA)

A decentralized body of the European Union, with headquarters in London since January 1995. Its main responsibility is the protection and promotion of public and animal health, through the evaluation and supervision of medicines for human and veterinary use.

Expanded Access

A means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient with a serious disease or condition who cannot participate in a controlled clinical trial.

F

Fast Track

A formal mechanism to interact with the FDA using approaches that are available to all applicants for marketing claims.

Fibrin

An insoluble protein that is essential to clotting of blood, formed from fibrinogen by action of thrombin.

Fibrinolytic

Pertaining to, characterized by, or causing the dissolution of fibrin by enzymatic action.

Fludarabine

A chemotherapy drug used in the treatment of hematological malignancies.

Food and Drug Administration (FDA)

An agency in the U.S. federal government whose mission is to protect public health by making sure that food, cosmetics, and nutritional supplements are safe to use and truthfully labelled. The FDA also makes sure that drugs, medical devices, and equipment are safe and effective.

G

Glycopeptide

Any of a class of peptides that contain carbohydrates, including those that contain amino sugars.

Glycosaminoglycan

Any of the carbohydrates containing amino sugars occurring in proteoglycans, such as hyaluronic acid or chondroitin sulfate.

Good Manufacturing Practices (GMPs)

The quality system regulation overseen by the Food and Drug Administration (FDA), which includes requirements related to the methods used in, and the facilities and controls used for designing, manufacturing, packaging, labelling, storing, installing, and servicing of medical devices intended for human use.

Graft-Versus-Host disease (GVH disease)

A condition that occurs when immunologically competent cells or their precursors are transplanted into an immunocompromised recipient (host) that is not histocompatible with the donor. Because the host is immunocompromised, the graft is not rejected. Immunocompetent T lymphocytes derived from the donor tissue recognize the recipient's tissue as foreign or nonself and react with it, producing clinical manifestations including edema, erythema, ulceration, loss of hair, and heart and joint lesions similar to those of connective tissue disorders. This condition is a frequent complication of bone marrow transplantation.

H

Helicobacter Pylori

A gram-negative spiral bacterium that causes gastritis and peptic ulcers in humans; a history of H. pylori infection is associated with carcinoma of the stomach.

Hemocrit

The percentage by volume of packed red blood cells in a given sample of blood after centrifugation.

Hypotension

Abnormally low blood pressure.

I

Inflammatory Bowel Disease (IBD)

A group of chronic intestinal disorders characterized by inflammation of the large intestine.

Intent To Treat (ITT)

System of statistical analysis that, in assessing a drug, takes into account all patients, including those who discontinued the treatment, i.e. for side effects, and not only those who terminated the study.

Investigational Drug

A drug under study but not yet FDA approved.

Ischemia

An insufficient supply of blood to an organ, usually due to a blocked artery.

J

Jaundice

Yellowish discoloration of the whites of the eyes, skin, and mucous membranes caused by deposition of bile salts in these tissues. It occurs as a symptom of various diseases, such as hepatitis, that affect the processing of bile.

L

Low Angle Laser Light Scatter (LALLS)

A measurement technique largely employed for sensing very high molecular weight compounds.

M

Microangiopathy

Any disease of the capillaries, often applied to vascular changes in diabetes mellitus.

Mucositis

An inflammation of mucous membranes.

Myeloma

Myeloma is a cancer of plasma cells, a type of white blood cell found primarily in the bone marrow. In myeloma, a plasma cell becomes malignant and grows continuously, destroying normal bone tissue and crowding out normal blood cell production.

Multiple Myeloma

A malignancy of the plasma cells that affects multiple sites within the bone marrow and secretes all or part of a monoclonal antibody.

N

Named Patient Program

A program that offer physicians and their patients access to drugs which have not yet received approval for marketing by national health authorities.

Non-steroidal anti-inflammatory drug (NSAID)

Drugs with analgesic and antipyretic effects and which have, in higher doses, anti-inflammatory effects.

O

Occlusion

An obstruction or a closure of a passageway or vessel.

Oligodoxyribonucleotide

A polymer made up of a few (2–20) nucleotides.

Orphan Drug

Designation given to either a rare disease that affects fewer than 200,000 people, or a common disease that has been ignored because it is less prominent in the US compared with developing nations.

Orphan Medicinal Product Designation

Medicinal products that have been designated as orphan due to the rarity of the diseases they treat. Measures were first put in place to create incentives for developing orphan medicinal products in America in 1983. Incentives are required, as there are more than 5,000 identified diseases that affect less than 0.05% of the population for which there are currently no satisfactory treatments. This U.S. initiative has proved so successful in stimulating research that Japan, Singapore, and Australia have introduced similar regimes as has the EU in the form of Regulation 141/2000/EC on orphan medicinal products (Orphan Regulation).

P

Peptide

Any member of a class of compounds of low molecular weight that can be broken down to yield two or more amino acids; they are the constituent parts of proteins and are formed by loss of water from the ammonia and carboxyl ends of adjacent amino acids.

Phase I Trial

Initial testing in a small group of humans to determine safety, dosing and identify side effects associated with an investigational agent.

Phase II Trial

A human study, often involving more patients than a phase I trial, designed to determine efficacy and safety of an investigational agent.

Phase III Trial

A human study, often involving more patients than a phase II trial, to confirm an investigational agents efficacy, compare it to commonly-used therapies and generate long-term safety data.

Pivotal Trial

A clinical trial to evaluate the safety and efficacy of an investigational agent in patients who have the disease or condition to be treated. Pivotal trials usually represent the most rigorous demonstration of the therapeutics’ efficacy and safety, and serve as the basis for a New Drug Application (NDA) or Biologics License Application (BLA) submission to the FDA.

Plasma Cell

Any or the antibody-secreting cells found in lymphoid tissue and derived from B-cells upon lymphokine stimulation and reaction with a specific antigen. Also called plasmacyte.

Polydeoxyribonucleotide

A group of 13 or more deoxyribonucleotides in which the phosphate residues of each deoxyribonucleotide act as bridges in forming diester linkages between the deoxyribose moieties.

Preclinical

Drug testing in non-human models to assess safety, toxicity and efficacy.

Proliferation

To grow or multiply by rapidly producing new tissue, parts, cells, or offspring.

Prophylaxis

Prevention of or protective treatment for disease.

Prostaglandin

Any of a group of naturally occurring, chemically related, long-chain hydroxy fatty acids that stimulate contractility of the uterine and other smooth muscle and have the ability to lower blood pressure, regulate acid secretion of the stomach, regulate body temperature and platelet aggregation, and control inflammation and vascular permeability. They also affect the action of certain hormones. There are nine types of prostaglandins, designated by the letters A to I, with the degree of saturation of their side chains designated by subscripts 1, 2, or 3.

Proteolysis

The splitting of proteins by hydrolysis of the peptide bonds, with formation of smaller polypeptides.

Pulmonary Embolism

The lodgment of a blood clot in the lumen of a pulmonary artery, causing a severe dysfunction in respiratory function.

R

Rare Disease

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.

Regulatory Agency

A public authority or government agency responsible for exercising autonomous authority over some area of human activity in a regulatory or supervisory capacity.

Renal

Of, relating to, or in the region of the kidneys.

S

Stem Cell

Relatively undifferentiated cells of the same lineage (family type) that retain the ability to divide and cycle throughout postnatal life to provide cells that can become specialized and take the place of those that die or are lost.

Stem Cell Transplantation (SCT)

The use of stem cells as a treatment for cancer or other diseases. In this procedure, the stem cells are removed (or obtained) from a donor; before a transplant is done for cancer, the patient receives high-dose chemotherapy and/or radiation to destroy the malignant cells; the stem cells are then given to the patient in whom they can produce new blood and immune cells and replace the cells destroyed by the treatment; the stem-cell preparation is infused into a vein and, once in the bloodstream, the stem cells head straight for the bone marrow space.

Sulfatation

Any of several methods by which esters or salts of sulfuric acid (sulfates) are formed.

T

Thromboembolic

Related to the combination of thrombosis and its main complication, embolism.

Thrombosis

Blood cells reacting to a foreign object by clotting and blocking the artery.

Toxicity

The quality of being poisonous, especially the degree of virulence of a toxic microbe or of a poison.

Treatment Investigational New Drug (Treatment IND)

A protocol used to make promising new drugs available to desperately ill patients as early in the drug development process as possible. FDA will permit an investigational drug to be used under a treatment IND if there is preliminary evidence of drug efficacy and the drug is intended to treat a serious or life-threatening disease, or if there is no comparable alternative drug or therapy available to treat that stage of the disease in the intended patient population. In addition, these patients are not eligible to be in the definitive clinical trials, which must be well underway, if not almost finished.

U

Ulcer

A local defect, or excavation of the surface of an organ or tissue, produced by sloughing of necrotic inflammatory tissue.

Ulcerative Colitis

Inflammation of the colon and rectum. The cause is unclear although there are often antibodies to colonic epithelium and E. Coli strain 0119 B14.

V

Vascular

Pertaining to blood vessels or indicative of a copious blood supply.

Veno-Occlusive Disease (VOD)

A disease that sometimes occurs following high-dose chemotherapy or radiation, in which the blood vessels that carry blood through the liver become swollen and clogged.