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Latest international precision medicine clinical trials now available to blood cancer patients in Australia

Tuesday June 29, 2021

Click here to watch the interview with Prof Paula Marlton.

In 2015 the Leukaemia Foundation established a Trials Enabling Program (TEP) to help Australians living with blood cancer access the latest research and treatments through international clinical trials, without leaving the country.

Partnering with the nation’s leading blood cancer clinical trial group the Australasian Leukaemia & Lymphoma Group (ALLG), this innovative program has helped many Australians access new clinical trials and novel therapies, years ahead of their expected availability on the Australian market.

Under TEP, two new trials have now successfully launched at hospital sites across the country, representing the first studies to occur in Australia in collaboration with the Haemato Oncology Foundation for Adults in the Netherlands (HOVON), and the German-Austrian Acute Myeloid Leukaemia Study Group, Germany (AMLSG).

The trials represent new hope for Australians living with acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS), with Australia’s participation in the trial expected to help improve access to new therapies for patients with these blood cancers.

Acute myeloid leukaemia is an aggressive type of blood cancer and MDS is a group of blood cancers which affect the production of normal blood cells. It is well-known that, over time, around one-third of MDS patients will go on to develop AML.

The risk of an individual with MDS going on to develop AML depends on a patient’s MDS subtype, varying from a 2 in 10 chance for people with low-risk MDS to a 4 in 10 chance for people with high-risk MDS. Although differences in cell biology exist between MDS and MDS that has developed into AML, these two diseases are genetically related. In almost every case where MDS has gone on to develop into AML, disease progression is associated with clonal evolution and mutations in particular genes.

Both clinical trials aim to understand whether the addition of new targeted treatments to existing standard treatments will improve outcomes for patients.

Around 30% of patients with newly-diagnosed AML experience changes in the FMS-like tyrosine kinase 3 gene (FLT3). Adding therapies like midostaurin which target these gene changes can improve survival in these patients. The HO156/AMLM24 trial, led by HOVON, will investigate whether either midostaurin or a more potent FLT3-targeted therapy, gilteritinib, can improve survival for these patients when added to intensive chemotherapy.

The trial’s Australian arm has officially opened, with the first patient now recruited at The Alfred Hospital in Melbourne. A further 24 sites are planned to open across the country with recruitment of 80 AML patients anticipated over a three-year period, while recruitment across 16 European countries, which began in late 2019, is targeted to reach 798 patients over coming years.

Chief Investigator A/Prof Andrew Wei from The Alfred Hospital in Melbourne, who is leading the Australian trial, said: “This is a globally significant and potentially practice-changing trial that we are delighted to be participating in nationally with the support of the ALLG and Leukaemia Foundation. It will also provide our patients with access to new and potentially beneficial drugs in the frontline setting and after stem cell transplant.”

The ALLG AMLM23 clinical trial is now also underway for patients newly diagnosed with specific sub-types of AML and MDS to explore the impact of adding precision therapies, ivosidenib and enasidenib, to standard treatment.

Around 20% of patients with newly diagnosed AML have genetic changes or mutations in two proteins – IDH1 or IDH2. The AMLM23 trial is targeting patients with IDH1 or IDH2-postive AML or MDS with excess blasts (MDS-EB2) who have not undergone previous chemotherapy.

Ten patients have already been recruited to the trial at hospital sites in Townsville, Melbourne, Perth and Sydney, with plans for the trial to open at a total 20 sites across Australia and recruit a total 80 patients over four years.

Recruitment for the AMLM23 clinical trial is currently open at a further 125 sites across nine European countries and is targeted to reach 968 participants by 2023.

The Australian arm of this trial is being led by Principal Investigator Professor Paula Marlton, Head of Leukaemia and Deputy Director of Haematology at the Princess Alexandra Hospital, who said the trial was an important example of how international collaborations can help improve access to new therapies for Australians with blood cancer.

Leukaemia Foundation CEO Chris Tanti said these trials will deliver new therapeutic drugs to blood cancer patients’ years ahead of the expected availability on the Australian market.

“We know that enabling access to clinical trials results in a better understanding of the disease, and a positive clinical trial outcome could have a major impact on how patients are managed in the future locally and globally,” he said.

“Our research shows less than 20% of Australians living with blood cancer who want to participate in a clinical trial are able to access one[1]. It is our priority to break down barriers for patients and ensure they have access to the latest clinical trials, treatments and information they need right here in Australia to not only survive their blood cancer but also live well after a diagnosis.”

Australasian Leukaemia & Lymphoma Group CEO Delaine Smith said there is an urgent need for new and highly effective therapies for patients with AML.

“These two trials are key vehicles by which improvements in survival rates will be achieved. Clinical trials are essential to the improvement and care of patients and collaborating with prestigious international groups means we can accelerate research and processes for new medicines for Australian patients.”

Enabling access to novel and specialised therapies, including through increasing access to clinical trials, is a key priority of Australia’s first National Strategic Action Plan for Blood Cancer – the blueprint to achieving the blood cancer community’s united vision to see zero lives lost to blood cancer by 2035.

The Australian AMLM23 and AMLM24 clinical trial projects were kindly supported by the estate of Florence Brown. If you are interested in supporting similar projects, please contact the Leukaemia Foundation on 1800 620 640.

The Leukaemia Foundation provides practical, emotional and educational support for Australians diagnosed with a blood cancer. If you or someone you know has been diagnosed with AML or MDS, please contact your treating physician to understand more about the ALLG AMLM23 and AMLM24 trials or contact the Leukaemia Foundation by visiting


Blood cancer signs and symptoms:
Symptoms of all blood cancers can sometimes be subtle or even similar to other conditions, such as a flu. However, ongoing symptoms like recurrent infections, increased fatigue, night sweats, bone pain, bruising or enlarged lymph nodes should be immediately discussed with your GP or specialist. Early diagnosis can play a key role in surviving blood cancer, so it is crucially important that you are examined and treated properly. If you or someone you love is diagnosed with a blood cancer, reach out to the Leukaemia Foundation on 1800 620 420. Accessing our support is free of charge.

[1]  3,227 people living with blood cancer responded to the survey featured in the State of the Nation: Blood Cancer in Australia report, providing a good representation of sub-types, States, regional status, age and private health insurance status.

Last updated on January 3rd, 2023

Developed by the Leukaemia Foundation in consultation with people living with a blood cancer, Leukaemia Foundation support staff, haematology nursing staff and/or Australian clinical haematologists. This content is provided for information purposes only and we urge you to always seek advice from a registered health care professional for diagnosis, treatment and answers to your medical questions, including the suitability of a particular therapy, service, product or treatment in your circumstances. The Leukaemia Foundation shall not bear any liability for any person relying on the materials contained on this website.