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Opening a new frontier: First-in-class ‘epigenetic’ drug in clinical trials for people with advanced blood cancer

Wednesday 20 May 2015

Consultant haematologists and clinical trial leaders Associate Professor Mark Dawson (right) and Dr Michael Dickinson (left) in the Medical Day Unit at the Peter MacCallum Cancer Centre in East Melbourne.


A new front in the global effort to control cancer has opened with an international clinical trial of a new ‘epigenetic’ compound. The ground-breaking trial is the culmination of eight years of laboratory research led by Associate Professor Mark Dawson from the Peter MacCallum Cancer Centre.

The first-in-human trial is for people with leukaemia and multiple myeloma for whom all other treatment options have failed. It comes five years after Associate Professor Dawson made a landmark discovery while at The University of Cambridge showing this epigenetic therapy can be very effective in aggressive blood cancers.

Associate Professor Dawson, Consultant Haematologist and Head of the Cancer Epigenetics Laboratory at Peter Mac and Principal Investigator on the international trial, says epigenetic ‘BET inhibitors’ offer a new avenue in the treatment of cancer.  

‘Cancer epigenetics is based on a new wave of understanding that by manipulating the way DNA is packaged and deciphered in cancer cells, we can reinstate the normal checkpoints that restrict and impede the growth and survival of these cells.

‘In this way, epigenetic BET inhibitors effectively silence cancer-causing genes, helping cancer cells revert to healthy cells — in earlier laboratory trials completed at The University of Cambridge in the UK, this led to remarkable results in aggressive mouse models of leukaemia.’

Dr Michael Dickinson, Consultant Haematologist and Co-Principal Investigator on the trial says while it is early days for this phase I trial, involving patients at Peter Mac and five major cancer centres in the United States and the United Kingdom, it represents a unique opportunity for patients.

‘Five-year survival for almost all cancers has improved markedly over the last two decades, and these advances often begin with small, tightly-controlled, phase 1 trial recruiting a handful of carefully selected patients.

‘While overall statistics are slow to improve, in some individuals facing terminal cancer, access to early clinical trials of an untested and unproven therapy can offer meaningful hope.'

Caroline Hangay was the first Australian patient enrolled on the trial, after her leukaemia did not respond to chemotherapy.

'There was no beating around the bush, for my stage of disease and my treatment history, this trial was my only option.

‘I don’t know what the future holds, but things are now looking hopeful, more so than I thought they possibly could.’

Today is International Clinical Trials Day, commemorating 20 May 1747, the day on which James Lind started his famous trial comparing treatments for scurvy.

Please direct enquiries on this clinical trail to: lori.boren@petermac.org

To support cancer research at Peter Mac, click here.


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