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Terms/Glossary


Glossary of terms
Acyclovir Medicine to treat several viral diseases
AML Acute Myeloid Leukaemia - most common acute leukaemia
Anaemia Low levels of red blood cells (Low haemoglobin levels)
Anthracyclines A class of medicines to treat several cancer types
Antimetabolites A class of medicines to treat several cancer types
Anti-viral effect Effect against virus infections
API Active Pharmaceutical Ingredient - the active component of a medicine
Ara-C (cytarabine) Medicine to treat blood cancers  - parent compound to CP-4055
Aza-C (azacitidine ) Medicine to treat blood cancer - parent compound to CP-4200
Biodegradable Degradable in a biological environment/system
Bioavailability The fraction of an administered dose of unchanged drug that reaches the circulation
Bio-distribution Distribution of a medicine to different organs and tissues in the body
Cancer A group of diseases in which cells grow unrestrained in an organ or tissue in the body; can spread to tissues around it and destroy them or be transported through blood or lymph pathways to other parts of the body
Carcinoma Cancer that begins in the skin or in tissues that line or cover the internal organs
Cardiovascular Related to blood vessels and/or the heart
Cell The fundamental unit of life. Each cell contains a complete set of an organism's genetic material. An organism is made up of many specialised cells of diverse functions
Central Nervous System (CNS) Related to the brain and/or spinal cord
Chemotherapy Classes of medicine to treat or control disease (mostly used to describe cytotoxic cancer therapy)
Clinical data Data from studies in man, as opposed to pre- or non-clinical data derived from laboratory studies, disease models and animal studies
Clinical trial Research study conducted with patients to scientifically evaluate new therapies or diagnostic tests
Combination Therapy Combining two or more medicines to treat the same disease
Composition of Matter Patent In describing the subjects of patents, these words usually applies to a new chemical entity (NCE). The patent may also include the functionality and proposed use of the compound
CR (Complete Response/ Complete Remission) Characterizes the level, or lack of, drug effect. See also CRp, PR and SD. By RECIST defined as disappearance of all detectable cancer
CRp A subgroup of CR used in within blood cancer
Cytarabine (ara-C) Medicine to treat blood cancers  - parent compound to CP-4055
Cytomegalovirus (CMV) Cytomegalovirus is a member of the herpes virus family. CMV can cause serious infections
Cytotoxic Medicines capable of killing cells - a class of anticancer medicines
Deamination Chemical reaction involved in deactivating several medicines inside cells
dFdC (gemcitabine) Medicine to treat several cancer types - parent compound to CP-4126
dFdCTP Gemcitabine-triphosphate - the active metabolite of gemcitabine
DLT Dose limiting toxicity; a side effect limiting the amount of medicine given
DNA Abbreviation for deoxyribonucleic acid. The substance of heredity; a large molecule that carries the genetic information that cells need to replicate and to produce proteins
Docetaxel Medicine to treat several cancer types
Double-Blind Study A comparative study in which neither the patient nor the physician knows whether the patient is receiving the treatment of interest or the control treatment
Elacytarabine Proposed International Non-proprietary Name (= generic name) for CP-4055
Enzyme Protein that catalyse (facilitates) chemical reactions without being changed itself
Equimolar Equivalent number of molecules of chemical substances
Excretion The eliminating of medicine from the body
FDA Food and Drug Administration (US drug regulatory authority)
First-Pass Metabolism The transformation of the medicine, as it passes, for the first time, from the gut though the liver.
Formal Toxicology Program Animal studies required prior to human exposure
Gemcitabine Medicine to treat several cancer types -  parent compound to CP-4126
GMP Good Manufacturing Practice; a set of quality principles guiding the manufacturing/production of pharmaceutical products
Haematological Related to the blood
hENT1 Human Equilibrative Nucleoside Transporter 1 = a protein known to mediate transport of nucleosides into human cells
Hepatitis B and C Particular virus infections of the liver
Herpes Genitalis A particular virus infection of the genitals
Herpes Labialis A particular virus infection at or close to the mouth
IMP Investigational Medicinal Product - formulation of a drug under development.
IND Investigational New Drug - medicine used in clinical studies in humans in the USA (not yet approved by the FDA for regular use)
Indication The valid reason to use a certain medicine or procedure
INN International Non-proprietary Name or generic name; the name used for the active ingredient of a drug (e.g. gemcitabine)
In vitro This term refers to experiments performed in an artificial environment like a test tube or culture media
In vivo This term refers to experiments performed in a living body or organism - most often in animals
Intellectual Property (IP) Any product of the human intellect that is unique, novel, unobvious, and has some value in the marketplace, such as a patented invention, trade mark, original design or the practical application of a good idea
Intraperitoneal Inside the abdominal cavity
Intratumoural Inside the tumour
Intravenous Inside the vein (blood vessel carrying blood on its journey back to the heart)
Leukaemia Group of cancer diseases of the blood
Lipids (Greek: lipos = fat) Fatlike substances, characterised by their insolubility in water and solubility in organic solvents such as alcohol, ether and chloroform. Storage lipids in the body are called triglycerides and the lipids which constitute the structural basis for cell membranes are called phospholipids
Liposomal Formulation A method of delivering a medicine to the body by encapsulating or incorporating the medicine in microscopic lipid vesicles called liposomes
LVT Lipid vector technology -  The modification of active drugs by the chemical binding of these compounds to certain selected lipids
Lymphoma A neoplasm of lymph tissue that is usually malignant
Malignancy Cancerous growth, in which a mass of cells grows in an uncontrolled fashion, with a tendency to create daughter cells that break away and grow elsewhere (metastases), invading and damaging surrounding tissue
Malignant Melanoma Type of skin tumour that is characterised by the cancerous growth of melanocytes, which are cells that produce a dark pigment called melanin
MDR Multidrug resistance; within cancer treatment: adaptation of tumour cells to anticancer drugs in ways that make the drugs less effective
MDS Myelodysplastic syndrome - Previously called pre-leukaemia. A mix of haematological diseases that may develop into AML
Metabolism Biochemical reactions that occur in a living organism, as well as the energy exchanges and transformations that accompany them. Drug metabolism: The process by which enzymes alter or break down drugs. The metabolism allows the drug to carry out its therapeutic role and eventually be eliminated from the body.
Metastases The spread of cancer from one part of the body to another
Mismatch repair Ability of the cell to repair damaged DNA (caused by chemotherapy or other factors)
MTA Material transfer agreement - Agreement often used between companies or between company/academic institution that allows one party to perform experimental work with a compound from the other party
MTD Maximum tolerated dose - the highest dose of a medicine that can be given without unacceptable side effects
New Chemical Entitiy (NCE) A new drug (active ingredient) which has a unique molecular structure
NDA New Drug Application - Application for approval in the US
NIH National Institutes of Health - the US top governmental health authority
Non-inferiority study Comparative clinical study design where the goal is to demonstrate that the product is not inferior to the comparato
NSCLC Non Small Cell Lung Cancer; Sub group of lung cancer representing approx. 80% of all lung cancers
Oncology The study of the causes, properties, disease progression and treatment of cancer
Oral Cancer Drug Medicine to treat cancer and which is taken via the mouth
Orphan Drug A term that refers to a drug that treats rare diseases. Companies can apply to FDA and EMEA to obtain so-called Orphan Drug Designation which gives both support by the authorities and market exclusivity for a fixed number of years.
Oxaliplatin Medicine to treat cancers
PD (Progressive Disease) Characterises the level, or lack of, drug effect. Defined by RECIST as above 20% growth in the sum of the longest diameters of the largest lesions/tumours. See also CR, CRp, PR, and SD.
PR (Partial Response) Characterizes the level, or lack of, drug effect. Defined by RECIST as ≥ 30% decrease in the sum of the longest diameter of target lesions/tumours. See also CR, CRp, PD, and SD.
Pharmacodynamic The study of the mechanism of action of medicines and their effects on the body
Pharmacogenetic The study of the mechanism of action of medicines at a genetic level
Pharmacokinetic The study of absorption, distribution, biotransformation and excretion of medicines, including the timelines of these processes
PoC Proof of concept
PoP Proof of principle
Preclinical study A test of a new drug in animals or in vitro ( in a test tube) conducted to gather evidence justifying a study in humans
Pro-drug A pharmacologically inactive compound that converts to the active form of the drug within the human body. It is generally designed to overcome problems associated with stability, toxicity, lack of specificity, or limited bioavailability
RECIST Response Evaluation Criteria in Solid Tumours; a set of internationally accepted criteria used to define responses to treatment of solid tumours.
RD Recommended dose as defined in phase I clinical studies
SD (Stable Disease) Characterises the level, or lack of, drug effect. Defined by RECIST as between +20% and - 30% change in the sum of the longest diameters of the target lesions/tumours. See also CR, CRp, PR, and PD
Shingles A herpes virus infection
Solid Tumour Cancer of body organs other than blood, bone marrow, or the lymphatic system, e.g. lung cancer, colorectal cancer, malignant melanoma, etc.
Sustained Level Maintained at a level without interruption or weakening
Superiority study Comparative clinical study design where the goal is to demonstrate that our drug is superior to the comparator
Targeting Agents Drugs that are designed to be active through interfering with specific receptors/mechanisms of the cell
Taxanes A group of medicines to treat several cancer types
Therapeutic Index (TI) The ratio of therapeutic benefit to side effects. The higher the therapeutic index, the greater the efficacy in relation to its side effects
Tolerability The subjective ability of patients to tolerate the effects of therapy
TTP Time to Progression; The time until a disease starts to progress in a patient