Venetoclax has generated a lot of interest from cancer researchers over many years. The drug is based on a discovery made in the 1980s at Melbourne’s Walter and Eliza Hall Institute and this year showed remarkable results in clinical trials for adults with chronic lymphocytic leukaemia (CLL), with most patients with an advanced form of CLL achieving either a partial or complete response. Venetoclax was fast-tracked for approval for clinical use for adults in the US.
Its target, BCL-2, is a cell survival protein and the product of an oncogene, a cancer-causing gene. After the success of venetoclax trials in adults, a next step was to test its effectiveness against children’s leukaemia, where it was seen as a potential alternative to navitoclax, a drug with some unpleasant side effects.
The paediatric leukaemia study was a collaboration between Walter and Eliza Hall Institute and Children’s Cancer Institute. Professor Lock’s laboratory at Children’s Cancer Institute is the leukaemia testing site for the Pediatric Preclinical Testing Consortium (PPTC), which is supported by the US National Cancer Institute. The site, the only PPTC site outside the US, generates high-quality preclinical data to prioritise therapies using a successful model developed here by Professor Lock and his team. Several drugs we have prioritised have now been accelerated into clinical trials for children with leukaemia . . .