Leukaemia drug breakthrough wins Victoria Prize

Melbourne professors Andrew Roberts and John Seymour have won a Victoria Prize for Science & Innovation for their development of an anti-cancer drug.

The $50,000 prize acknowledged the duo’s leadership in bringing a new anti-blood cancer drug into clinical practice through world-leading clinical trials.

The impact has been massive. It is now in use by thousands of patients globally, keeping their cancer under control for many months and in some cases, years. The drug, Venetoclax, is a pill. Taken daily, it switches off a protein that causes cancerous leukaemia cells to die in patients with chronic lymphocytic leukaemia (CLL). As with any medical discovery of this magnitude, there were many people involved in its testing, however, Professor John Seymour, Haematology Director at the Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, as well as Professor Andrew Roberts, Head of the Walter & Eliza Institute of Medical Research, are the ones with their names on the prize.

Professor John Seymour"We can’t emphasise enough that this was truly a team effort," says Andrew, acknowledging both the decades of development, as well as their recent prize win. "We’re representing many teams of researchers at the Walter and Eliza Hall Institute and in Victorian hospitals, not to mention our partners in industry without whom this wouldn’t have happened."

John, adds: "A remarkable feature of the development of this drug to date has been the critical involvement of Melbourne researchers – both laboratory and clinical – at each of the key steps which makes local skill and expertise so integral to the success."

Certainly, the drug breakthrough is further endorsement that Victoria is a leading medical research hub, particularly in cancer research involving hospitals, research institutes and industry collaboration. These have been nurtured via the Victorian Comprehensive Cancer Centre (VCCC), an alliance of ten medical organisations committed to cancer control, of which the Peter MacCallum Cancer Centre and The Walter and Eliza Hall Institute of Medical Research are just two members.

Victoria has always been Australia’s home of innovation and it is this culture, backed by a highly skilled workforce and world class research institutes, that has made it the epicentre of Australia’s biotech sector.

Professor Andrew Roberts in the labratory smiling at the cameraWhile getting Venetoclax to market has been three decades in the making, Andrew says the state of Victoria has a culture of taking on scientific challenges and sticking to the task until it’s done – noting this is especially true in medical research. According to him, the origins of Venetoclax went through a similar unrelenting development period.

"This drug follows from a totally unexpected research discovery in Melbourne 30 years ago. The first 15 years were about basic research – working out exactly how to tackle the Achilles' heel of some cancers – and only then were we able to focus on making the right drug,” says Andrew. “The drug was generated about 10 years ago and co-developed by American pharmaceutical companies, AbbVie and Genentech. The first patient was treated on a trial in Melbourne seven years ago, and the drug was approved for standard use in CLL patients in Australia just under two years ago."

That may seem like a mighty long time to receive the clinical thumbs up, but Andrew says "the process of development has gone extremely quickly for this drug over the last decade."

He recalls that when the drug became the first of its kind to be approved for clinical use, his reaction was one of "great excitement, enormous relief, and a real sense of hope for improving outcomes for patients now and in the future."

Speaking of which, the duo’s next big cancer research goal is to work out how to best combine this drug with other drugs to enable some patients with CLL to be cured. They’ll also be exploring how best to use this drug in other blood cancers, including lymphoma and other leukaemias.

"We and others are beginning to explore which other forms of cancer may also benefit from Venetoclax treatment," says John, “and just last month, the drug was approved in combination with chemotherapy agents in the US for another form of leukaemia (acute myeloid leukaemia) in elderly patients without other effective treatment options. This expansion of the areas where the drug is showing benefit for patients is very gratifying."

More about Venetoclax

  • For some cancers, BCL2 is the key protein that keeps the cancer alive. So when you turn off BCL2, the cancers cells die. 
  • Chronic lymphocytic leukaemia (CLL) is one cancer that relies on BCL2 for survival. That’s where Andrew and John first trialled Venetoclax, and where it’s now approved in many countries for routine use in those whose CLL has returned after initial therapy.
  • Venetoclax can be given alone or in combination with another anti-cancer drug for CLL. It causes the CLL to melt away and then keeps it under control for many months or years. It has a major benefit in most patients and how long those benefits can last is still being worked out.

Footnote: Last month, we interviewed the other winner of the Victoria Prize: Professor Doug MacFarlane. He won in the physical sciences category for his work in storing renewable energy.