SA researchers make massive leukaemia breakthrough

Adelaide researchers say their advance in treating a form of blood cancer is the “biggest breakthrough in chronic myeloid leukaemia treatment this century”.

• $1.25m grant for SAHMRI researcher to outsmart cancer
• Get the most out of your Advertiser digital subscription
• New tool to help newly diagnosed cancer patients

Adelaide researchers leading an international clinical trial say they have made “the biggest breakthrough in chronic myeloid leukaemia (CML) treatment this century.”

The project has demonstrated the safety and efficacy of a next-generation treatment, a new kinase inhibitor called asciminib.

CML is a blood cancer that causes bone marrow to produce too many white blood cells. This excess of mutant white blood cells interferes with normal blood cell production.

The study’s lead author, Professor Tim Hughes, said the development of the original tyrosine kinase inhibitor (TKI) called imatinib in the 1990s changed CML “from a death sentence to a disease that in many patients could be managed until they lived to a ripe old age.”

“But while imatinib and subsequent TKIs have been very effective at improving survival, they frequently cause serious side-effects,” he said.

TKIs slow or stop the excess production of white blood cells but the ones currently approved for use are not well targeted, so while they attack leukaemic cells they also damage healthy cells.

Prof Hughes, who holds senior positions at the SA Health and Medical Research Institute, University of Adelaide and Royal Adelaide Hospital, says the new inhibitor asciminib “selectively blocks the mutant kinase present in the leukaemic cells.”

“This trial of 150 patients showed asciminib is highly effective, even in patients who had failed to respond to several other TKIs,” Prof Hughes said.

“Equally as important, it is well tolerated by patients and appears to have significantly less long- term ill-effects compared to current treatments. Asciminib is the biggest breakthrough in CML treatment this century.”

More than 4000 Australians are living with CML however the increasingly high survival rate brought about by TKI treatment means the disease is estimated to be the most common form of leukaemia by 2040.

The results of this clinical trial written with co-authors Dr David Yeung and Associate Professor David Ross who also hold senior positions in SAHMRI and other major Adelaide medical and research institutions were published in the New England Journal of Medicine.