A Curative Approach for Chronic Myeloid Leukaemia (CML)

Chronic myeloid leukaemia (CML) is predicted to become the most prevalent leukaemia in Australia within the next 20 years. Only about 25% of CML patients achieve the best outcome – treatment-free remission (TFR).

Around 50% still require lifelong treatment and a further 25% develop resistance or intolerance to treatment.

This project is one of several that researchers at SAHMRI are involved in that are improving the length and quality of life for people with CML.

This research focuses on improving outcomes by determining the key genetic events driving drug resistance and disease progression. This will further develop the new, highly targeted drug asciminib which promises to revolutionise CML therapy and remove the current barriers to TFR - the ultimate goal of CML therapy today.

Asciminib is the latest generation tyrosine kinase inhibitor (TKI) treatment for CML. The introduction of TKI treatment at the turn of the millennium has improved CML survival from approximately 20% at 10 years to almost normal life expectancy however CML is still a fatal disease for 5-10% of patients. The reasons for early progression to acute phase disease and for drug resistance need to be explored before the death rate can be reduced further.

Thousands of people in Australia are facing lifelong dependence on expensive drugs that often cause side effects which can be serious. Over the past 10 years this team and others have shown that carefully selected people with CML with excellent responses to therapy can stop treatment and around half of them will remain in TFR. Currently however, there are no accurate and reliable predictors of who will relapse and who will remain in TFR.

This project is funded by the National Health and Medical Research Council