Tuesday, 10 January 2017

New anti-cancer drug, venetoclax which has the power to ‘melt away’ advanced forms of chronic lymphocytic leukaemia (CLL) was recently granted approval by the Australian Therapeutic Goods Administration (TGA).

The drug will be marketed as VenclextaTM and is approved for Australians with relapsed or refractory CLL with 17p deletion, a mutation that makes the disease resistant to standard treatment, as well as for people with relapsed or refractory CLL who do not have any other treatment options available.

How did this discovery come about?

In 1988, researchers at Walter and Eliza Hall Institute (WEHI) in Melbourne discovered BCL-2, a protein that enables cancer cells to survive. Since then a team of scientists worldwide has been working to find a way to ‘hit’ BCL-2 in order to stop cancer cell survival.

Venetoclax was discovered and developed with scientists from US pharmaceutical companies AbbVie and Genetech, as part of an international collaboration with WEHI.

Thanks to our supporters, the Leukaemia Foundation contributed to early work on the development of venetoclax, (formerly called ABT-199). This research was undertaken by Dr Kylie Mason, Professor Andrew Roberts and collaborators at WEHI through the Leukaemia Foundation’s National Research Program Grants-in-Aid in 2010 and 2012.

How does venetoclax work?

The drug works on the protein BCL-2 that prevents CLL cells from dying. Because these cells don’t die and continue to be made in the bone marrow, they begin to crowd out the blood, bone marrow and organs. The protein also protects the malignant CLL cells from chemotherapy, making them resistant to treatment.

Venetoclax switches off the BCL-2 protein, allowing the CLL cells to die naturally and making them susceptible to chemotherapy, so it is more effective in killing cancer cells.

What are the next steps?

Now that venetoclax has been approved by the TGA, it can be registered on the Australian Register of Therapeutic Goods (ARTG) and legally marketed and sold in Australia.

To make the drug affordable to the Australian public, the manufacturer can apply to the Pharmaceutical Benefits Advisory Committee to have the cost of the drug subsidised by the Australian government on the Pharmaceutical Benefits Scheme (PBS).

Venetoclax is not listed on the PBS. Historically the delay between TGA approval and PBS listing ranges from 14 to 31 months for cancer drugs.

Will this drug target other blood cancers?

Venetoclax is being combined with other approved drugs in Phase II and Phase III clinical trials in other blood cancers.

It is hoped venetoclax in combination with other drugs could benefit other hard to treat types of blood cancer, but further research is necessary.

For more information, visit http://www.wehi.edu.au/.