Victorian researchers have made a global breakthrough, uncovering unprecedented insights into how a new drug can slow the growth of low-grade gliomas (LGG) — a type of brain cancer that mostly affects young adults.
This is the first-ever clinical trial run through the BrainPOP (Brain Perioperative Platform), led by The Brain Cancer Centre and backed by a $16 million investment from the Victorian Government.
Why this matters
Low-grade gliomas are a slow-growing but devastating type of brain cancer that significantly impacts the lives of patients, many of whom are young adults in their prime. Characterised by a specific mutation gene called IDH, current treatments are limited and LGG have long been considered incurable.
Thanks to the pioneering BrainPOP clinical trial program, led by The Brain Cancer Centre, this innovative approach involves collecting tumour samples before and after treatment, enabling researchers to learn exactly how therapies work within the brain.
They have been able to directly study how a new drug, Safusidenib, works inside the brain. This oral therapy targets the IDH1 gene mutation found in many LGGs.
What the trial showed
BCC funded research teams from The Royal Melbourne Hospital, WEHI and Peter MacCallum Cancer Centre collected tumour samples before and after treatment to track the drug’s effect — a global first for brain cancer.
The results, published in Nature Medicine, show how the drug can change tumour activity at a molecular level, giving researchers powerful clues about how to better personalise treatment in the future.
“This study reveals the power of BrainPOP as a safe and effective platform for accelerating our understanding of new treatments and their real-world impact in the brain.”
Dr Jim Whittle, Co-Head Research Strategy, The Brain Cancer Centre, Laboratory Head WEHI, Medical Oncologist Peter MacCallum Cancer Centre
“This trial is not only a revolution in the way we test new treatments but brings new opportunities for this most deserving group of patients with a devastating disease.”
Professor Kate Drummond, Director of Neurosurgery, RMH
The trial was supported by an incredible $16m investment from Victorian Government and involved patients who had not yet undergone chemotherapy or radiation.
Recognition on the world stage
Nature Medicine is one of the most prestigious scientific journals in the world. Think of it as the “front page of world science.” Only the most important and groundbreaking discoveries — the kind that could change how we understand medicine, technology, or the universe — are chosen to be published there.
Getting research into Nature Medicine means researchers work has been recognised globally as being of the highest quality and significance. It’s a bit like winning an Oscar in film, or receiving a gold medal at the Olympics — it signals that the work is among the very best in the world.
What’s next
“For the first time, we’ve seen what a drug is doing in the brain with incredible detail, helping us to clearly identify the next steps for personalising treatment and predicting who would most benefit.” Dr Jim Whittle
While it is too soon to know if, ultimately, these medications will improve outcomes or extend lives of these patients, this trial proves that BrainPOP is a safe and effective way to test new treatments and speed up discoveries. Plans are now underway for pivotal studies of Safusidenib in diffuse IDH1 mutant gliomas.
“I’m incredibly proud of the team, from multiple institutions, that delivered this clinical trial and its results. Researchers and the community should take the chance to celebrate these results with us. The fact that we were able to bring this concept to life was a world first at the time and is an extraordinary achievement. We also understand the responsibility to not rest, that this is just one step – we’re already focused on the chance to do more to support the brain cancer community now and in the future” Sam McGuane, CEO The Brain Cancer Centre
Victorian Minister for Economic Growth and Jobs Danny Pearson said:
“We will continue to support our world class researchers to make these life-changing discoveries, and cementing Victoria as a leader in medical research,”
About BrainPOP
BrainPOP is a pioneering trial platform that allows researchers to test new therapies directly in the brain — something never done before due to the complexity of neurosurgery. This Brain Cancer Centre funded program connects hospitals and laboratories across Melbourne’s biomedical precinct, aiming to transform the design of brain cancer clinical trials and the development of new treatments.
The collaborative trial program led by The Brain Cancer Centre brings together the expertise of researchers and clinicians across the Melbourne Biomedical Precinct including Murdoch Children’s Research Institute, Peter MacCallum Cancer Centre, The Royal Children’s Hospital, the University of Melbourne, the Royal Melbourne Hospital and WEHI.
The new study drew on the clinical and surgical expertise of the RMH, including Prof Drummond, and the multidisciplinary approach of WEHI’s Brain Cancer Research Laboratory – co-led by Dr Whittle, Dr Sarah Best and Dr Saskia Freytag – which combines deep knowledge of clinical oncology, fundamental cancer biology and bioinformatics to accelerate research outcomes.
Collaborators included University of Melbourne, The Florey, Metabolomics Australia and MoleQlar Analytics (Germany). The research was supported by AnHeart Therapeutics, a Nuvation Bio Company, and the Victorian Government.
The BrainPOP research team (left to right): Professor Kate Drummond, Dr Jim Whittle, Dr Saskia Freytag, Dr Sarah Best.
Brain scans from a patient in the world-first clinical trial, where researchers were able to compare tumour samples taken before treatment (left) and after treatment (right). The pioneering Brain Perioperative clinical trial platform aims to accelerate drug development and improve outcomes for brain cancer patients.
