SA researchers lead clinical study for chronic myeloid leukaemia

SA researchers are leading the world’s first frontline clinical study set to revolutionise the 21st century treatment of a type of leukaemia.

One hundred cancer sufferers are involved in a world-first $2m Adelaide-based trial of next-generation drugs to treat chronic myeloid leukaemia (CML).

Researchers are conducting the first frontline clinical study that is set to revolutionise the treatment of CML.

South Australian CML patients were among those in the trial to have received initial doses of the drug, asciminib, 30 days ago.

“There is no other patient in the world that has access to this frontline therapy,” the study’s co-lead researcher, Professor Tim Hughes, said.

“I think it has the potential to be a game-changer in the way we treat CML around the world.”

Prof Hughes holds senior positions at the SA Health and Medical Research Institute, Royal Adelaide Hospital and is the Beat Cancer professor at the University of Adelaide.

CML is a blood cancer that causes bone marrow to produce too many white blood cells.

Its treatment since the 1990s has been through tyrosine kinase inhibitor drugs that have turned CML from a deadly disease to one that can be managed.

But the drugs’ side-effects include increased risk of heart attack and stroke, with one-third of the patients using them struggling with major day-to-day side-effects such as tiredness and muscle pains.

Prof Hughes said the asciminib drug being trialled in SA targeted the CML cells more directly, with fewer damaging side-effects.

More than 4000 Australians are living with CML. It is estimated to become the most common form of leukaemia by 2040.

“The problem with CML treatment today is that while it is much better than it was in the 1990s, we have 50 per cent of patients who respond well, but will need treatment for life,” Prof Hughes said.

“Meanwhile, 25 per cent of all patients using these current drugs respond poorly or can’t tolerate their therapy – that’s a significant amount of unmet need.”

The trial will run for the next 12 months. If successful, it will pave the way for a much larger randomised international trial to progress it through various regulatory stages and then to market.