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Molecular Screening and Therapeutics clinical trials

Cancer is usually considered a disease of older people, with mortality rates increasing with age for most cancers. One of the key characteristics of rarer cancers, however, is that these cancers place a great burden on children and young families. In every age group (Baby Boomers, Gen X, Gen Y, and even children) ‘rare’, high-mortality cancers are the most common cause of disease-related death in Australia.

Reasons for the high mortality rates for less common cancers include difficulty in diagnosis (as they are seen less often), standard treatments are often less effective and there is limited access to new therapies.

While there is excellent evidence that participation in clinical trials is associated with better outcomes, these patients have little access to standard clinical trials because they are not financially feasible to run for the small numbers of patients with one ‘rare’ cancer or another.

The Genomic Cancer Medicine Program has developed targeted approaches to inhibit tumour growth and immunotherapies to stimulate an immune response that delivers long-lived tumour destruction. These medicines, which work on differing cancer mechanisms, offer new opportunities for patients. The Program is working to understand more about the heritable causes of cancer and risk management, and developing new diagnostic techniques and treatments to improve the lives of individual patients, and health outcomes for all Australians.


Traditionally clinical trials in humans for new drugs have been conducted in four to five phases, taking around eight to ten years to complete. The financial costs of conducting cancer clinical trials have risen from less than US$10,000 per patient in 1980, to around $US47,000 in 2011. Average development costs are estimated at around US$3.6 billion dollars per drug. The cost of drug development, which must be recouped by pharmaceutical industry, already limits access of some patients to important treatment options, and is an important component of future public health care costs.

Because there are fewer patients with these cancers, it isn’t possible to follow the usual progression of clinical trials mechanisms for these small groups of patients.

While phase 1, 2 and 3 studies are, and will remain, critical to drug development, they are costly and slow. New trial designs, such as the MoST clinical trials, which sit between a phase 1 toxicity trial and a phase 2 efficacy trial and include genetic testing of cancers, offer opportunities for trialling targeted treatments.

Eligibility for participation in MoST clinical trials is independent of where the cancer arises in the body, as MoST personalises experimental treatment based on an individual’s unique personal and cancer genetic profile. In these trials, rather than focusing on a tumour’s location, such as the ovaries, patients with say ovarian cancer, pancreatic cancer, sarcoma and other cancers, but who have a shared harmful variant, are treated with a drug that may target the variant.

Clinical trials are generally used to test a new treatment, with some patients getting the new drug and the others getting an existing drug or placebo (a pill that has no effect). The MoST clinical trials test multiple treatments at the same time and all participating patients in a MoST trial receive a treatment. The advent of genomic medicine means that treatment is guided by the genetic make-up of the patient and their illness.

All component studies of the Genomic Cancer Medicine Program have been approved by the St Vincent’s Hospital Sydney Human Research Ethics Committee, which operates under the requirements of the National Health and Medical Research Council’s National Statement on Ethical Conduct in Human Research (2007), the Australian Code for the Responsible Conduct of Research (2007), and for research specific to NSW, the NSW Supplement to the National Statement (2008).

How it works


Joining the MoST Clinical Trial

First, all patients, and their tumours where possible, are genomically screened to see if they are suitable for a trial and if there are variants that can guide the treatments that can be trialled. These trials are looking to see if a treatment will work, or work more effectively than another treatment.

Professor Thomas and his team are investigating how targeted therapies work and are trying to find new biomarkers that can predict which patients will benefit from these treatments.

After screening, patients are offered either:
1. MoST clinical trials, including immunotherapies
2. Clinical trials outside MoST that use molecular eligibility criteria
3. Other biomarker-guided treatments outside MoST.

All participants, including those with no ‘actionable’ biomarkers, are informed of the results of the screening of their tumour tissue through their own doctors.

MoST has already shown that genomic cancer profiling can identify treatable options for a significant portion of patients who previously had none.


Precision Cancer Immunotherapy

The MoST protocol is also conducting clinical studies to test new immunotherapy drugs in patients with high-mortality cancers. The first MoST immunotherapy substudy tests a combination of so-called ‘immune checkpoint inhibitor’ drugs that take the brakes off the anti-tumour immune response, enabling immune cells to attack cancer cells. A second immunotherapy study, for patients with specific genetic abnormalities in their tumour, combines a targeted treatment with a checkpoint inhibitor.

Although immunotherapies are proving to be effective in many cancer types, they do not work in all patients. Garvan researchers are looking to find biomarkers that can predict which patients will benefit from specific treatments targeting the immune system and to better understand how immunotherapies work to fight cancer. With this knowledge, the team aims to develop a more precise approach that tailors treatment with immunotherapy to individual patients based on the characteristics of their immune system and its interactions with tumour cells.

The immunotherapy trials will allow us to understand how these immune biomarkers influence the anti-tumour response and help develop a precision immunotherapy approach where treatment can be personalised.

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