| 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 |



