α-TOS – α-Tocopherol Succinate (CNC331), a Vitamin E analogue and prototype molecule for Mitocans CNC332 (MitoVE11S). CNC331 (α-TOS) was used to treat a mesothelioma cancer patient in the compassionate care setting.

ADME – Absorption, Distribution, Metabolism, and Excretion. A set of investigations carried out as part of preclinical drug development.

Allogeneic – Derived from another source; in the case of transplantation, the source of a graft is someone other than the recipient.

Antibody – Large Y-shaped proteins used by the immune system to identify and neutralise pathogens such as bacteria, viruses, and cancer cells.

Antigen – A molecule that interacts with components of the immune system, e.g. antibodies or T cells.

API – Active Pharmacological Ingredient. The component of a medicine responsible for the therapeutic effect. Other ingredients are carriers, water preservatives etc.

Apoptosis – The self-destruction and death of cells (including cancer cells) by natural processes in the same way that old or damaged normal cells die.

Autologous – Derived from the same source; in the case of transplantation, the graft is derived from the recipient.

Biologics – A broad class of drugs derived from biological sources, e.g. antibodies, genes, blood products, and cells.

Blockbuster – Biotech industry designation for drug that achieves annual sales in excess of USD$1 billion.

cGMP – Current Good Manufacturing Practice. Practices followed by the pharmaceutical and biotech firms to ensure that the products produced meet specific requirements for identity, strength, quality, and purity. FDA regulates these industries to ensure cGMPs are being followed.

Checkpoint inhibitor – A drug that inhibits the immunological checkpoints and thereby increases the magnitude of an immune response by breaking tolerance.

Clinical trial – The mechanism of assessing safety and efficacy of a novel drug in humans.

CMO – Contract Manufacturing Organisation that can for example manufacture drugs to cGMP standards.

CRO – Contract Research Organisation that can for example design, conduct and analyse clinical trials.

CTN – Clinical Trial – Notification, an Australian TGA scheme where the sponsor of a clinical trial will notify the TGA of its intent to carry out a clinical trial but the responsibility for the trial approval falls to the IHREC and the institution where the trial is to be conducted.

CXT Clinical Trial – Exemption, a TGA scheme where the sponsor of a clinical trial will seek approval to run the trial from the TGA, who then review all associated preclinical data.

Cytokine – Substances, such as interferon, interleukin, and growth factors, that are secreted by certain cells of the immune system and have an effect on other cells.

Dendritic cell vaccine – A therapeutic which involves the harvesting of the patient’s blood dendritic cells, priming them with tumour antigens and then re-infusing them back into the patient.

Drug modality – The type of drug substance, e.g. small molecule, peptide, antibody, DNA, RNA, cells, or other categories of therapeutic agents.

Efficacy – The degree to which a drug has the intended therapeutic effect of improving health by preventing or curing disease.

FDA – The US Food & Drug Administration, which approves the trialling and registration for new drugs in the USA.

Galactoside – A specific type of sugar that the body uses for specific biochemical purposes.

Genetic vaccine – A vaccine that consists of a DNA sequence that encodes a gene, which when transcribed and translated into protein will be the target of an immune response.

GM-CSF – Granulocyte-Macrophage Colony-Stimulating Factor.

IHREC – Institutional Human Research Ethics Committee. The committee that reviews proposals to carry out research on human subjects or tissues.

ICI – Immune checkpoint inhibitor. A relatively new class of “breakthrough” immunotherapies that reduce a tumour’s ability to suppress the immune response. ICIs are often described as “taking the brakes off” the immune response.

IND – Investigational New Drug. The FDA terminology for a drug in development. An application for an IND contains preclinical data required for the transition of the drug to clinical trial. This is a prerequisite in the US.

in vitro – Experiments conducted in a petri dish.

in vivo – Experiments conducted in animal subjects, for example mice or rats.

Malignant melanoma – Skin cancer of melanocytic (or mole) origin. Very aggressive tumours that metastasise (spread) quickly in comparison to other skin tumours. Mostly affects fair-skinned people and often caused by exposure to sunlight.

Mesothelioma – A tumour of the mesothelium, which line several of the body’s cavities. Most often caused by exposure to asbestos. It is a very aggressive and difficult to treat tumour.

Metastasis – The phenomenon of tumour spread from the primary site to distal secondary or tertiary sites. Generally, prognosis worsens with the degree of tumour metastasis.

Mitochondria – A subcellular organelle responsible for (among many things) the efficient conversion of carbohydrates and fats to CO2 and chemical energy in the form of ATP.

MitoVE11S – A vitamin E-succinate ester used to target the mitochondria of cancer cells. A proprietary compound of Cancure Limited.

Modality – See “Drug Modality”.

NCE – New Chemical Entity, a small chemical substance that is without precedent among regulated and approved drug products.

NEJM – New England Journal of Medicine, published by the Massachusetts Medical Society. NEJM is among the most prestigious peer-reviewed medical journals and the oldest continuously published one.

NDA – New Drug Application. Submitted to the FDA and contains the results of clinical trials. The FDA reviews the NDA, then decides whether to grant new drug status and allow marketing of the drug.

NME – New Molecular Entity is a drug or chemical that is without precedent among regulated and approved drug products. The NME designation indicates that a drug in development is not a version or derivative of an existing and previously investigated, trialled and approved substance.

NSCLC – Non-Small Cell Lung Cancer. An aggressive and difficult-to-treat cancer of the lung.

Patent term extension – An extension of patent life of up to 5 years to compensate a drug developer for the marketing time lost due to governmental regulations, which often necessitate 6–8 years of pre-market clinical testing.

Peptide vaccine – A vaccine comprising of fragments of proteins (peptides), which are usually synthetically manufactured.

Phase 1 clinical trial – A first in man study of a developmental drug. The primary endpoints in Phase 1 studies are toxicity and dosing. Not usually concerned with efficacy, although this can be addressed as a secondary endpoint if carried out in diseased subjects.

Phase 2 clinical trial – A clinical trial that addresses efficacy and safety of a developmental drug. Dosing is based on the data obtained from a Phase 1 study. Subjects are individuals that are suffering from the disease in question. This can include a placebo and carried out “double blind”, where neither the subject nor the attending physician is aware whether the drug or a placebo is administered.

Phase 3 clinical trial – A large clinical trial that is only conducted after a successful Phase 2 study. Usually double blind, placebo controlled. Many more subjects are enrolled in several centres, and it is often international.

RECIST – Response Evaluation Criteria in Solid Tumours, a standardised system of tumour evaluation before and after treatment that enables comparisons of experimental treatments.

Renal cell carcinoma – A tumour originating in the kidneys, usually aggressive and difficult to treat.

TGA – Therapeutic Goods Administration. The Australian regulatory authority responsible for review and approval of new drugs for marketing.

T cells – Immune cells that drive the immune response. Some T cells are responsible for cell-killing. T cells are important in anti-tumour immunity.

Vaccine – A substance that mimics a pathogenic agent (e.g. virus or tumour cell) and generates an immune response. The immune response targets the pathogen and neutralises its pathogenic effects.