Glossary of Terms - Biotechnology

adjunct— An auxiliary treatment that is secondary to the main treatment.

 

adjuvant— Substance or drug that aids another substance in its action.

 

angioedema—Swelling of the mucous membranes, tissues beneath the skin, or an internal organ due to an allergic reaction.

 

ankylosing spondylitis— An inflammatory disorder of unknown cause that primarily affects the spine. The vertebrae may fuse together and form a rigid back that is impossible to bend.

 

antisense— An antisense drug is the mirror or complementary image of a small segment of messenger RNA (mRNA), the substance that carries instructions (“sense”) from the genes to the cell’s protein-making machinery. The anti-sense drug readily binds to the mRNA strand, keeping it from transmitting its instructions to the cell, and thus inhibiting the production of an unwanted protein.

 

application submitted— An application for marketing has been submitted by the company to the Food and Drug Administration (FDA).

 

biotechnology— The collection of processes that involve the use of biological systems. For some of the industries, these processes involve the use of genetically engineered organisms. For the purpose of this report, only those products that involve recombinant DNA, monoclonal antibody/hybridoma, continuous cell lines, cellular therapy, gene therapy and vaccines technology are included.

 

Clostridium difficile— A bacterium that produces an irritating toxin that causes a form of colitis characterized by profuse, watery diarrhea with cramps and low-grade fever.

 

colony stimulating factor (CSF)— Protein responsible for controlling the production of white blood cells.

 

Crohn’s disease— A subacute chronic gastrointestinal disorder, involving the small intestine, characterized by patchy deep ulcers that may cause fistulas and a narrowing and thickening of the bowel.

 

cytomegalovirus (CMV)— A DNA virus related to the herpes virus, affecting mostly neonatal

infants and immunocompromised individuals. CMV can occur without symptoms or result in mild flu-like symptoms.

 

erythropoietin— Hormone that stimulates red blood cell production in the bone marrow.

 

Fast Track— A process designed to facilitate the development and expedite the review of drugs to treat serious diseases and fill an unmet medical need. The status is assigned by the U.S. Food and Drug Administration. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious diseases. Generally, determining factors include whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may be potentially superior to existing therapy. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process. The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.

 

Gaucher’s disease— An inherited disease caused by a lack or deficiency of an enzyme (glucocerebrosidase). Primarily affects the liver, spleen and bone marrow, usually resulting in death.

 

gene therapy— Therapy at the intracellular level to replace or inactivate the effects of disease-causing genes or to augment normal gene functions to overcome illness.

 

glioblastoma— The most common primary brain tumor and one of the most aggressive forms of brain cancer, primarily affecting adults over the age of 50.

 

growth factors— Factors responsible for regulating cell proliferation (rapid and repeated reproduction), function and differentiation.

growth hormone—Pituitary hormone that stimulates the growth of long bones in prepu- bertal children.

 

heat shock protein— A natural adjuvant (a substance that enhances the immune response to an antigen) linked to molecular signals or antigenic peptides, which are capable of stimulating an immune response. The heat shock protein (HSP) core is an invariable molecule that remains the same for each patient; the antigenic peptides are patient- and tumor-specific or pathogen-specific. When purified in a certain way, HSP-peptide complexes constitute the “fingerprint” of the cell from which they were derived. When injected into a patient, these antigenic peptides are re-expressed on the surface of important immune cells—macro-phages—that play a major role in activating the immune system, resulting in a much more potent immune response than the one achieved by the expression of these same peptides by the tumor or pathogen-infected cell.

 

hemophilia A and B— Hemophilia A, the “classic” hemophilia, is a genetic bleeding disorder due to deficiency of the coagulation factor VIII. Hemophilia B, or “Christmas” disease, is caused by deficiency of coagulation factor IX.

 

hepatitis— Inflammation of the liver with accompanying liver cell damage or death, caused most often by viral infection, e.g., hepatitis B, and C.

herpes simplex virus—A strain of herpes virus that may lie dormant in nerve tissue and can be reactivated to produce painful sores of the anus or genitals.

 

human papillomavirus— Viral agent of warts, believed to be contagious and usually harmless, but it can lead to cervical cancer.

 

interferon— A glycoprotein naturally produced by cells that interferes with the ability of a virus to reproduce after it invades the body.

 

interleukin— An endogenous substance that stimulates the production of different types of white blood cells or leukocytes.

 

ischemia— Insufficient supply of blood to an organ or tissue, which can cause organ damage such as an ischemic stroke.

 

lymphoma— Cancers in which the cells of lymphoid tissue, found mainly in the lymph nodes and spleen, multiply unchecked. Lymphomas fall into two categories: One is called Hodgkin’s disease, characterized by a particular kind of abnormal cell. All others are called non-Hodgkin’s lymphomas, which vary in their malignancy according to the nature and activity of the abnormal cells.

 

mAb (monoclonal antibodies)— Large protein molecules produced by white blood cells that seek out and destroy harmful foreign substances.

 

macular degeneration— A progressive disorder that affects the central part of the retina, causing gradual loss of vision. It is a painless condition, usually affects both eyes and is common in the elderly.

 

metastases/metastatic—Cancers that have spread from the primary or original cancer site. Cancer cells can break away from a tumor and travel to other areas of the body through the bloodstream or the lymph system. They are often more difficult to treat than primary tumors.

 

multiple myeloma— A malignant condition characterized by uncontrolled proliferation of plasma cells (a class of white blood cells) in bone marrow. Symptoms include pain and destruction of bone tissue, numbness and paralysis, kidney damage, anemia, and frequent infections.

 

myelodysplastic syndromes (MDS)— Blood disorders that ultimately are fatal. Patients usually succumb to infections or bleeding. The term “preleukemia” has been used to describe these disorders.

 

myeloid— Pertaining to, derived from, or manifesting certain features of the bone marrow. In some cases, myeloid also pertains to certain types of non-lymphocyte white blood cells found in the bone marrow.

 

myocardial infarction— Damage to the heart muscle caused by stoppage or impairment of blood flow to the heart, also known as heart attack.

 

neuroblastoma— A tumor of the adrenal glands or sympathetic nervous system (the part of the nervous system responsible for certain automatic body functions, such as the “fight or flight response”).

neutropenia—An abnormally low neutrophil count (certain white blood cells).

 

Orphan Drug— A drug to treat a disease that has a patient population of 200,000 or less in the United States, or a disease that has a patient population of more than 200,000 and a development cost that will not be recovered from sales in the United States.

 

peripheral vascular disorder— The obstruction of blood supply to the extremities, particularly the legs, caused by atherosclerosis.

 

Phase 0— First-in-human trials conducted in accordance with FDA’s 2006 guidance on exploratory Investigational New Drug (IND) studies designed to speed up development of promising drugs by establishing very early on whether the agent behaves in human subjects as was anticipated from preclinical studies.

 

Phase I—Safety testing and pharmacological profiling in humans.

 

Phase II— Effectiveness and safety testing in humans.

 

Phase III— Extensive clinical trials to demonstrate safety and efficacy in humans.

 

pulmonary hypertension— High blood pressure in the arteries that supply blood to the lungs.

 

pulmonary fibrosis— Scarring and thickening of lung tissue.

 

respiratory syncytial virus (RSV) disease— One of the most important causes of lower respiratory tract disease in children, accounting for more than 90 percent of cases of bronchiolitis.

 

restenosis— A condition where an artery plugs up again following treatment to open it up.

 

sepsis— A condition associated with a serious bacterial infection of the blood.

 

short bowel syndrome— The malabsorptive condition that arises after significant segments of the small intestine are removed.

 

systemic lupus erythematosus (SLE)— The most serious form of lupus, a chronic autoimmune disorder causing inflammation and damage to multiple organs.

 

thrombocytopenia— A reduction in the number of platelet cells in the blood, which causes a tendency to bleed, especially from the smaller blood vessels.

 

ulcerative colitis— A chronic inflammation and ulceration of the lining of the colon and rectum. It causes bloody diarrhea and mainly involves the left colon.

 

uveitis— Inflammation of the uvea, the middle layer of the eye.