Australian protein discovery leads to tablet that “melts away blood cancer”

22 August 2016
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Group photo
More than 100 institute staff and students have been
involved in the development of venetoclax
A potent anti-cancer treatment co-developed and trialled in Melbourne was granted approval for use in patients by the powerful US Food and Drug Administration (FDA) in April 2016.

The announcement recognises that the drug, venetoclax, is a successful new therapy for patients with chronic lymphocytic leukaemia (CLL). It is also a strong indicator that the drug is on track to becoming registered and available in Australia.

Venetoclax was developed based on a landmark discovery in 1988 by Walter and Eliza Hall Institute scientists. They found that a protein called BCL-2 promoted cancer cell survival. Ever since then scientists around the world have been trying to find a way to target BCL-2 as a treatment for certain cancers.

Professor Andrew Roberts, a clinical haematologist and head of clinical translation at the Walter and Hall Institute in Melbourne said trials had shown patients responded to venetoclax with a very substantial reduction of leukaemia cells in their body.

“Some patients have remained in remission four years after their treatment began,” Professor Roberts said of the trials.

“In many cases we have seen the cancerous cells simply melt away.”

The drug is effective in killing cancer cells in approximately 80 per cent of people with very advanced forms of CLL. Twenty per cent of patients achieved a complete remission, where the leukaemia was no longer seen on routine tests. Its US approval is for patients with CLL who have a specific chromosomal abnormality called a 17p deletion and who have been treated with at least one other therapy.

Professor Roberts said a small number of patients had such a profound response to venetoclax that even very sensitive research tests were unable to detect any remaining leukaemia in their bodies.

CLL is one of the most common forms of leukaemia. In Australia more than 1000 people are diagnosed with the cancer every year.

Walter and Eliza Hall Institute director Professor Doug Hilton said the development of venetoclax had set the foundation for building towards the “dream of a cure for CLL”. It reflects the critical importance of robust medical research funding in Australia.

“Research that ultimately leads to major advances and cures can take decades to be developed and venetoclax is representative of that trajectory,” Professor Hilton said.

“This is a prime example of the tangible benefits of investment in Australian medical research at every stage – from idea through to discovery to development. Any royalties that flow to the institute from the FDA’s approval of venetoclax will be invested back into research that will improve health outcomes for patients in Australia and abroad.”

Venetoclax was co-developed for use by US pharmaceutical companies AbbVie and Genentech, a member of the Roche group, and was discovered by AbbVie scientists as part of a joint research collaboration with Walter and Eliza Hall Institute scientists.


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