Monash researchers to run world-first leukaemia “platform” trial

A/Prof Andrew Wei in clinic. A/Prof Wei researches AML.
Still from "Meet our research champion: Dr Andrew Wei"

by Anne Crawford

A national clinical trial program led by Monash University researchers testing drugs that may help patients with an aggressive form of leukaemia has been awarded a federal government grant of more than $1.5million.

The program, investigating treatments for Acute Myeloid Leukaemia (AML), is being funded by the Medical Research Future Fund’s (MRFF) Rare Cancers, Rare Diseases and Unmet Needs Clinical Trials Program.


It is being led by Adjunct Associate Professor Andrew Wei from the Australian Centre for Blood Diseases (ACBD) and Adjunct Associate Professor John Reynolds from Epidemiology & Preventive Medicine in Monash University, in collaboration with the Australasian Leukaemia and Lymphoma Group. Associate Professor Wei’s ACBD colleague Professor David Curtis was also awarded one of the 19 MRFF grants (see story).

“It is an amazing feeling to find out that this new medical research fund is really going to accelerate validation of new drugs into patient care more rapidly than ever,” Associate Professor Wei said. “It will be a great catalyst for Australian research.”

AML, which destroys a patient’s capacity to make blood, causes anaemia, infection and bleeding. Intensive chemotherapy is the main form of treatment but 50-70% of patients relapse afterwards.

The “platform trial” – the first of its kind for this disease state – will test the efficacy of multiple new drugs at the same time, aiming to ‘erase’ residual disease left over after chemotherapy to prevent relapses.

“Rather than just testing one drug at a time, waiting years for the result and then moving on to the next trial, we’re trying to increase the efficiency of finding more effective treatments quickly by testing several new drugs in parallel,” Associate Professor Wei said.

“The platform design also means more patients receiving potentially active treatments and less patients randomised to observation,” he said.

The program would be ongoing, trialling new drugs as they become available.

Associate Professor Wei is building on research conducted with venetoclax, a drug which has shown positive effects in older patients with AML. “This platform trial will now examine if this drug can be useful for younger patients.

“The program will determine whether a new drug, such as venetoclax, has potential to become the standard of care after chemotherapy by substantially reducing the chance of the disease coming back and possibly the need for a stem cell transplant,” he said.

The program is set to start in six months.