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Protein discovery leads to tablet that “melts away blood cancer”

August 26, 2016

A potent anti-cancer treatment co-developed and trialled in Melbourne was granted approval for use in patients by the US Food and Drug Administration (FDA) in April 2016.

The approval was announced this week and it recognises that the drug, venetoclax, is a successful new therapy for patients with chronic lymphocytic leukaemia (CLL). CLL is one of the most common forms of leukaemia in Australia and the Western hemisphere – it accounts for approximately one-third of new leukaemia cases.

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

Cancer researchers at WEHI

A team effort by WEHI researchers.

Professor Andrew Roberts, a clinical haematologist and head of clinical translation at WEHI 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. The 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.

WEHI 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 and worldwide cialis overnight delivery.

“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.

More than 100 WEHI staff and students have been involved in the development of venetoclax and it was co-developed for use by two US pharmaceutical companies.

The original news post was published on the WEHI website. Image of

The Australian Cancer Research Foundation (ACRF) has supported cancer research at WEHI by providing three research grants, AUD$ 5.5M in total. The establishment of the ACRF Centre for Therapeutic Target Discovery at WEHI assisted with this discovery and has supported scientists in testing primary human samples against a range of drugs.

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