Glossary of terms used on this website.
Acute Myelogenous Leukemia (AML): A quickly progressing disease in which too many immature white blood cells (not lymphocytes) are found in the blood and bone marrow. Also called acute myeloid leukemia or acute nonlymphocytic leukemia.
Ara-C : Cytarabine (cytosine arabinoside) is used to treat acute leukaemias, some head and neck cancers and non-Hodgkin’s lymphoma (NHL). This drug is one of a group of chemotherapy drugs called anti-metabolites. Anti-metabolites are similar to normal body molecules but they are slightly different in structure. These differences mean that anti-metabolites stop cells working properly instead of helping them. Anti-metabolites often stop cells making and repairing DNA.
Cytotoxic Compound: Cell-killing substance.
Cytarabine: Also known as Ara-C, is an generically available anticancer drug that is part of standard regimens for treating Acute Myelogenous Leukemia (AML). It belongs to the family of drugs called antimetabolites, and has a mechanism of action that includes inhibiting DNA replication and repair. (DeVita – Cancer)
de novo: In cancer, the first occurrence of cancer in the body.
DNA (deoxyribonucleic acid): The molecule present in every cell of the body that contains genetic information; a double-stranded molecule, shaped like a twisted ladder, consisting of four nucleotides in varying sequences that are paired with one another. The nucleotides, represented by the letters A, C, G, and T, are the building blocks of DNA.
Drug development process:
• Discovery: Identification of a biological, genetic or protein target linked to a particular disease, and subsequent lead identification of a potential drug that interacts with the target to help cure the disease or halt its progression.
• Preclinical: Comprehensive in vitro (lab dish) and animal testing of the drug candidate to establish its target specificity, toxicity in various doses and pharmacokinetics.
• Clinical Phase 1 : Human trials conducted to demonstrate the safety and early evidence of effectiveness or (efficacy) of an experimental drug or procedure. Tests are conducted to establish dosage, side effects and pharmacokinetics.
• Clinical Phase 2 : Trials with small numbers of patients conducted to identify drug performance characteristics effectiveness (optimal dosing, administration, key indication).
• Clinical Phase 3 : Pivotal trials conducted with larger patient populations to gather the additional information about effectiveness and safety that is needed to evaluate the overall benefit-risk relationship of the drug and to provide adequate basis for physician labeling.
• Approval: Data are analyzed and submitted for regulatory review. The U.S. submission to the FDA is called NDA (New Drug Application) or BLA (Biologic License Application); in Europe, data are submitted to the EMEA (European Medicines Evaluation Agency) in a submission called an MAA (Marketing Authorization Application). After analysis and review of the submission, the regulatory agency provides final approval.
Enzyme: A protein that speeds up chemical reactions in the body.
Gene: The fundamental physical and functional unit of heredity; a DNA segment that codes for (directs) the production of one protein.
Hematologic Malignancies: Cancers of the blood or bone marrow, such as leukemia or lymphoma. Also called hematologic cancers.
Hematopoietic Stem Cell Transplantion (HSCT): A procedure where bone marrow precursor cells are transplanted into a patient whose bone marrow has been prepared for transplantation by use of radiation, chemotherapy or both. These stem cells can come from the patient (so called “autologous” or transplants) or a donor (so called “allogeneic” transplants). This procedure is widely used in AML patients following induction of a remission with chemotherapy, such as with Ara-C or VNP10101M.
in vivo: In the body. The opposite of in vitro (outside the body or in the laboratory).
Metastasis: The spreading of tumor cells.
Myelodysplastic Syndrome (MDS): A syndrome characterized by “dysplasia” (abnormal maturation and release of bone marrow cells) in which the bone marrow does not make enough healthy blood cells. Also called refractory anemia, preleukemia and smoldering leukemia. This disease can be a precursor to the development of AML in some patients.
Patent: A grant made by the government giving the creator of an invention the sole right to make, use and sell the invention for a set period of time.
Prodrug: a pharmacological substance (drug) which is administered in an inactive (or significantly less active) form. Once administered, the prodrug is metabolised in the body in vivo into the active compound.
Protein: A molecule made up of amino acids that are needed for the body to function properly. Proteins are the basis of body structures such as skin and hair and of substances such as enzymes, cytokines, and antibodies.
Ribonucleotide Reductase Inhibitor (RRI) : A family of anticancer drugs that interfere with the growth of tumor cells by blocking the formation of deoxyribonucleotides (building blocks of DNA).
Small Cell Lung Cancer (SCLC) : An aggressive (fast-growing) cancer that usually forms in tissues of the lung and spreads to other parts of the body. The cancer cells look small and oval-shaped when looked at under a microscope. Also called oat cell cancer.
Temozolomide (Temodar®) : An anticancer drug that belongs to the family of drugs called alkylating agents. Temodar® is a registered trademark of Schering-Plough.