Melbourne-based neurosurgeon leads new drug trial in brain cancer

Australian researchers have achieved a global-first, using an innovative process to understand how a new drug suppresses tumour activity in brain cancer, offering hope to patients with low-grade gliomas (LGG).

One Australian is diagnosed with brain cancer every five hours, and more children die from brain cancer than from any other disease. Survival rates have barely shifted in the past three decades, with 80% of patients diagnosed with brain cancer dying within five years. LGGs, a slow-growing type of brain cancer, profoundly affect patients’ lives, many of whom are young adults in the prime of life.

Researchers from the Royal Melbourne Hospital (RMH), Walter and Eliza Hall Institute of Medical Research (WEHI), and the Peter MacCallum Cancer Centre studied the efficacy of Safusidenib, originally developed by Daiichi Sankyo and licensed to AnHeart Therapeutics for development and commercialisation outside Japan, in this patient cohort.

“We want Victorian brain tumour patients to have care that is equal to anywhere in the world,” said Professor Kate Drummond, Director of Neurosurgery at RMH and the trial’s lead investigator. “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 Drummond highlighted the positive response from patients, many of whom were excited to participate in the trial, “even though the trial required two operations and intensive treatment.”

“Brain cancer patients are desperate for new treatments, and clinical trials like this are exactly what is needed,” she added.

Dr Jim Whittle, medical oncologist specialising in neuro-oncology at Peter Mac and Laboratory Head at The Brain Cancer Centre and WEHI, explained the trial’s significance.

“These types of [perioperative] trials are vital for advancing drug development but with the complex and sensitive nature of neurosurgeries, this approach has not previously been used in brain cancer,” Dr Whittle said. “This new study reveals the power of [Brain Perioperative platform] BrainPOP as a safe and effective platform for accelerating our understanding of new treatments and their real-world impact in the brain.

“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,” he added.

The trial was supported by a $16 million investment from the Victorian Government and involved patients who had not yet undergone radiation or chemotherapy. Participants took the drug prior to any other cancer treatment, a world first for IDH inhibitors.

While it is too early to determine if Safusidenib will improve outcomes or extend lives, plans are now underway for pivotal studies in diffuse IDH1 mutant gliomas.

“We are deeply grateful for the Victorian Government’s support for the BrainPOP platform, which is vital to our ultimate goal of radically transforming outcomes for patients with brain cancer,” Dr Whittle said.

Victorian Minister for Economic Growth and Jobs Danny Pearson described the results as promising for brain cancer patients.

“We will continue to support our world-class researchers to make these life-changing discoveries, and cementing Victoria as a leader in medical research,” he said.

Further trials using the BrainPOP platform are already in development. Patients who meet the entry criteria are offered participation by their treating practitioners at participating hospitals.

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 Mac, The Royal Children’s Hospital, the University of Melbourne, RMH, and WEHI.

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