Personalised Medicine (T1)
Speakers
- Dr Penny Wilson, Technology Strategy Board, UK
- Associate Professor Grant McArthur, Head of the Translational Research Group and Head of the Molecular Oncology Laboratory, Peter MacCallum Cancer Centre
- Professor Robyn Ward, Professor of Medicine & Clinical Associate Dean, Prince of Wales Clinical School, UNSW. Chair of the Medical Services Advisory Committee (MSAC)
Session co-chairs
- Paul Cohen, Paul Cohen Consulting
- Sonia Yip, NHMRC Clinical Trials Centre, The University of Sydney
Synopsis
Personalised medicine uses the knowledge of genetics to predict the development of disease and to tailor medical treatment to an individual so as to obtain the best outcome. Speakers will cover some of the emerging issues and trends in personalised medicine including (1) the need for companion diagnostics for patient selection (2) impact on clinical trial design and patient subgroups (3) genetic biomarkers as predictors of disease and (4) changes in the MSAC process and in particular how funding agencies may better assess co-dependent technologies.
Full Text
Sequencing of cancer genomes suggest that many attractive drug targets are found in about 10% of cancers. New clinical trial designs are needed to demonstrate improvements in survival compared to standard treatment with the use of new targeted drugs. . Equally important is the validation of diagnostic tests that are robust to reliably identify subsets of patients most likely to benefit. This creates profound challenges to align the dual development of diagnostic test with the therapeutic, so-called co-diagnostic tests.
Examples will be given from the field of oncology including melanoma and rarer diseases such as dermatofibrosarcoma protuberans (DFSP). These will illustrate scenarios where a strong scientific rationale has guided trials of therapies in small subsets of patients who are predicted to most likely to benefit from a new drug regimen on the basis of their individual biology. How the targeted therapy imatinib was approved for the treatment of DFSP (a disease which always harbours a translocation of the COL1A1 and PDGFB genes), based on a Phase 2 study of only 12 patients will be examined. More recently the challenges in melanoma are more difficult. For example the current analysis of the KIT inhibitor nilotinib in melanoma is being assessed in a randomised study with traditional endpoints and statistical power, however only 2% of melanoma patients have KIT mutations yet the research necessitates a global study with standardization of molecular diagnostic testing. Melanoma is also an example of a disease with a number of possible novel targets are present at low frequencies including groups of mutations such as NRAS, CDKN2A, CCND1, CDK4, PTEN, and AKT that might predict benefit from combination
The development of a co-diagnostic test in these two disease types will be discussed. The benefits and hurdles of obtaining regulatory approval of these tests will be covered.
In conclusion, the study of the biology of the cancer and targets coupled with innovative trial design and validation of surrogate markers and the development of robust diagnostic tests to identify responsive patients is essential to allow patients to benefit from the genomics revolution in cancer.
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