An achievement 30 years in the making that has given hope to sufferers of terminal blood cancers has lead to a deal worth up to US$325 million for a Melbourne medical research institute.

The massive windfall from the partial sale of royalty rights for the cancer-busting treatment venetoclax will not only reward the Walter and Eliza Hall Institute for its decades of scientific experimentation and discovery, but will secure its future as a global centre for medical research. 

“Venetoclax demonstrates what success can look like for a collaborative, entrepreneurial and innovative medical research institute and why investment in basic research is so important,” the Institute’s director Professor Doug Hilton said.

“The Institute’s mission is to make discoveries for humanity and this income will help us deliver on that. It will enhance and accelerate our ability to make fundamental discoveries that can be translated into better treatments, bringing real benefits to patients on a global scale, as well as benefiting the Australian economy.”

A portion of the income will go towards enhancing and accelerating the discovery and translation of new medicines, as well as the acquisition of state-of-the-art dynamic imaging technology.

The development of the anti-cancer treatment began in 1987 with the discovery by PhD candidate David Vaux of a cancer gene — Bcl-2 — that stops cells from being able to kill themselves. His discovery was hailed as a milestone in the study of cancer genes, and now venetoclax is used to stop Bcl-2, allowing cancer cells to self-destruct. 

In a 2013 clinical trial, the drug was used to treat advanced leukaemia patients for whom no conventional treatment options were left. As a result, 84 per cent experienced remission, despite an average of four failed prior treatment regimens. The trial patients were typically incurable, yet almost 25 per cent achieved complete cancer clearance.

In the years since that initial Bcl-2 discovery, more than 400 scientists have harnessed developments in technology to contribute to this life-saving project, according to the Institute’s cancer and haematology laboratory head Professor David Huang.

Robotics and automation, X-ray crystallography and nuclear magnetic resonance were all deployed in efforts to stop Bcl-2’s dirty work, with the Australian Synchrotron’s arsenal of supercharged technologies essential to the work.

“The key elements that you need for drug discovery are automation and robotics and that's critical because they will allow you to, from tens of thousands of small molecules, identify things that will specifically target Bcl-2,” Huang said.

“The other thing that's critical in terms of technology is the ability for us to realise how the drugs can target Bcl-2. We have a detailed molecular picture and that really is very much driven by technology such as X-ray crystallography, and we've been incredibly lucky in Melbourne because the Australian Synchrotron is based here.”

Now, with venetoclax on the market and the Institute’s financial security assured, Huang said it’s a special moment.

“I think for somebody like me to have been involved in the project, from basic research discovery on to making a difference to patients, and then for the Institute to be rewarded in the commercial sense for that, that’s very satisfying, a very special moment. It almost beats the eureka moment when you make a new discovery,” Huang said.

A subsidiary of the Canada Pension Plan Investment Board has acquired the rights to a portion of future venetoclax royalties with an upfront cash payment of US$250 million and potential milestone payments of up to US$75 million. The Institute will retain partial royalties in the groundbreaking treatment. 

Therapeutic regulatory bodies in Australia, Canada, the US and the EU have approved venetoclax for treating people with previously treated chronic lymphocytic leukaemia with 17p deletion. Venetoclax is jointly commercialised by Genentech, a member of the Roche Group, and AbbVie.



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