Professor Grant Dewson from WEHI will co-direct the team to advance precision medicine and biomarker discovery for Parkinson’s disease, alongside Professor Glenda Halliday from the University of Sydney.

Parkinson’s was first discovered in 1817 and is now the fastest growing neurodegenerative condition in the world. Why are there no drugs to stop or slow its progression?

Parkinson’s has no single cause. It is complex, with links to several gene mutations as well as environmental influences. Currently there is no cure and the only drugs available help control the wide range of symptoms people with Parkinson’s experience daily.

By the time someone is diagnosed it’s estimated they have already lost over 50% of the brain cells that produce dopamine. The exact cause of these losses is not yet fully understood.

These factors combined, and the expense of drug discovery projects, make the search for new medicines difficult, but new technology along with multi-discipline collaboration is accelerating research.

What are precision medicines and how do they differ from more traditional medicines?

Traditional medicine uses a one-size-fits-all approach. Everyone with a particular condition will be prescribed the same drug that might work well for some and not for others.

Precision medicine tailors the treatment to an individual guided by factors including genetics, environment and lifestyle. This patient-centered approach is designed to make medicines more effective and minimise side-effects, and is now used in many cancer therapies.

Why is the search for biomarkers for Parkinson’s so important?

Biomarkers are measurable disease indicators, ideally by a simple test of blood or urine.

Having a biomarker that will identify Parkinson’s in its early stages before symptoms become apparent will allow people to access treatments that more effectively delay the progression of the disease. It could also lead to a national screening program like we have for bowel and breast cancers.

Biomarkers are also important for tracking disease progression, individual responses to treatments and the effectiveness of new disease-modifying drugs in clinical trials.

Looking forward five years to the end of this project, what do you hope to have achieved?

This Centre of Research Excellence provides important foundations for closer collaboration across Parkinson’s research, enabling us to leverage combined resources, personnel and expertise.

Through this collaborative approach we aim to better understand the complexities of Parkinson’s and progress new disease-modifying therapies and biomarkers towards the clinic – helping improve the lives of the more than 200,000 Australians living with Parkinson’s today and future generations.

The Centre of Research Excellence for Parkinson’s Disease: Advancing Precision Medicine is led by WEHI and the University of Sydney in partnership with the Florey Institute of Neuroscience and Mental Health, University of Tasmania, Monash Health, Neuroscience Research Australia and the Royal Melbourne Hospital.

Professor Andrew Wei’s team will lead a national Centre of Research Excellence to transform acute myeloid leukaemia outcomes by combining cutting-edge science, advanced technologies and clinical expertise to drive discovery and fast-track new treatments.

Why is acute myeloid leukaemia so difficult to treat, and who is most at risk?

Acute myeloid leukaemia (AML) is a rare but aggressive blood cancer that primarily affects older adults, though it can occur at any age. Around 1200 Australians are diagnosed each year. Despite advances in care, the five-year survival rate remains low at just one-in-four.

Treatment often involves strong chemotherapy and sometimes stem cell transplants, however these options are often too toxic for older patients who make up most cases. Many patients relapse because current therapies don’t adequately address the biological diversity of AML.

A key cause of treatment failure is the persistence of minimal residual disease (MRD) – small numbers of leukaemia cells that survive initial treatment and can drive relapse. Genetic changes within AML cells are highly variable and certain high-risk changes, like mutations in the TP53 gene, are especially resistant to treatment and associated with poor outcomes.

How will the Centre of Research Excellence improve early detection and prevent relapse?

The new Centre of Research Excellence will bring together leading experts in AML biology, molecular genetics and clinical trials to develop more effective strategies to treat, monitor and prevent leukaemic relapse. This includes validating the potential of new drugs and combinations, and applying advanced technologies to unmask how drug resistance develops.

Researchers will bring new ultra-sensitive MRD detection tools to the clinic to aid earlier identification of relapsing disease and use this information to guide pre-emptive, targeted interventions. We will also conduct cutting-edge clinical trials aimed at identifying more effective treatment approaches for the most difficult to treat leukaemias. And a collaborative network will be established to collect patient-derived samples to accelerate discovery and translation.

What new treatments and models are being explored for high-risk AML?

Centre of Research Excellence researchers are investigating a range of targeted therapies that exploit specific vulnerabilities in AML cells. These include inhibitors of proteins which help cancer cells survive, such as FLT3, BCL2 and IDH1.

For patients with TP53-mutated AML – a subtype with extremely poor prognosis – researchers are exploring immune-stimulating agents like STING agonists and inhibitors of cancer survival proteins such as MCL1 and BCLXL.

Combination therapies are also being developed to enhance the effectiveness of existing drugs, such as menin inhibitors, and we are building advanced lab models to track how AML evolves and responds to treatment.

How will the Centre of Research Excellence build a stronger AML research and clinical workforce?

The Centre of Research Excellence will invest in training the next generation of AML researchers by funding multiple PhD and postdoctoral positions. Trainees will gain hands-on experience in genomics, bioinformatics and clinical trial design. Our collaboration across six major institutions, alongside industry and consumer partners, will help fast-track new discoveries into clinical care.

The Centre of Research Excellence in Acute Myeloid Leukaemia is led by WEHI in partnership with the Peter MacCallum Cancer Centre, the Royal Melbourne Hospital, Monash Medical Centre and Alfred Health.

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Header image: Professor Grant Dewson (left) and Professor Andrew Wei (right)