A next-generation treatment for CML, asciminib, is the biggest breakthrough in CML treatment this century, according to Professor Timothy Hughes.

He spearheaded development and testing of the new treatment and is lead author of the results of an international study, published in the New England Journal of Medicine, in December 2019.

Asciminib is a new tyrosine kinase inhibitor (TKI) that is well tolerated and does not have the serious side-effects of the original TKI, imatinib. In the 1990s, imatinib changed CML from a death sentence to a disease that could be managed and meant many patients lived into old age.

While imatinib and subsequent TKIs approved for use in Australia are very effective at improving survival, they also frequently cause serious side-effects because they are not well targeted. In addition to attacking leukaemic cells, they also damage healthy cells which can result in vomiting, diarrhoea and muscle pain.

Professor Hughes said asciminib selectively blocks the mutant kinase present in the leukaemic cells and an international clinical trial involving researchers in Adelaide has demonstrated the safety and efficacy of asciminib.

“This trial of 150 patients showed asciminib is highly effective, even in patients who’d failed to respond to several other TKIs,” said Prof. Hughes about the Phase I dose-escalation study.

“Equally as important, it’s well tolerated by patients and appears to have significantly less long-term ill-effects compared to current treatments.”

While more than 4000 Australians currently live with CML, by 2040 CML is estimated to become the most common form of leukaemia, due to the increasingly high survival rate. This has resulted from TKI treatment.

Lisa McNeil, who was diagnosed with CML in late-2001 aged 30, went on the asciminib trial in mid-2015. She is still on the trial and describes her experience with this drug as “a lot better than the old TKIs”.