Developed by a team of international and Australian scientists, venetoclax has just received Australian approval. The anti-cancer drug has the power to "melt away" certain advanced forms of chronic lymphocytic leukaemia (CLL) - the most common type of leukaemia in Australia, with 1300 people diagnosed each year.
Venetoclax's approval is for patients with relapsed or refractory CLL with 17p deletion - a mutation that makes the disease relatively resistant to standard treatment options - as well as for patients with relapsed or refractory CLL for whom no other treatment options are available.
Walter and Eliza Hall Institute of Medical Research director Professor Doug Hilton AO welcomed news of the drug's approval, most importantly for patients with limited treatment options.
"The fact that Australians with hard-to-treat chronic lymphocytic leukaemia can now benefit from a drug like venetoclax demonstrates how critically important medical research is to the health of our community," Professor Hilton said.
"TGA approval of venetoclax is a major milestone in a journey spanning decades of powerful and innovative research by teams of leading scientists, clinicians and entrepreneurs, including more than one hundred researchers at Melbourne's Walter and Eliza Hall Institute of Medical Research."
The timeline of discovery began at the Walter and Eliza Hall Institute in 1988, with the identification of BCL-2, a protein enabling cancer cells to survive. Professor Hilton said scientists worldwide had subsequently been trying to find a way to "hit" BCL-2, in order to stop cancer cell survival.
"Like a lethal arrow, venetoclax flies straight to the heart of BCL-2," Professor Hilton said.
Venetoclax was discovered and developed with scientists from US pharmaceutical companies AbbVie and Genentech, as part of an international collaboration with the Walter and Eliza Hall Institute. The first clinical trials for venetoclax started in Melbourne at the Institute's Victorian Comprehensive Cancer Centre partners The Royal Melbourne Hospital and Peter MacCallum Cancer Centre and were led by Australian haematologists.
Professor Andrew Roberts, a clinical haematologist at The Royal Melbourne Hospital and cancer researcher at the Walter and Eliza Hall Institute and the University of Melbourne, said venetoclax was being combined now with other approved drugs and undergoing phase 2 and phase 3 clinical trials in other blood cancers.
"The hope is that venetoclax, potentially in combination with other approved drugs, could benefit more patients including those with other hard-to-treat types of blood cancer," Professor Roberts said. "Ongoing research suggests that this drug will be very active against other cancers, so this milestone may just be the beginning."
[Walter and Eliza Hall Institute of Medical Research]