Australian researchers have achieved the most promising advance in brain cancer treatment in two decades, with a new clinical trial showing that a targeted drug can shrink previously incurable tumours by up to 90 per cent.
As reported by Natasha Robinson in The Australian, the pioneering Brain POP (Brain Perioperative) clinical trial platform has provided unprecedented insights into how brain cancer drugs work. Led by The Brain Cancer Centre in collaboration with the Royal Melbourne Hospital, the Walter and Eliza Hall Institute of Medical Research (WEHI), the Peter MacCallum Cancer Centre, The Florey, and MoleQlar Analytics (Germany), the platform allows doctors to examine tumours before and after surgery to track exactly how a treatment interacts with cancer cells.
A key partner in the study was Metabolomics Australia, enabled by Bioplatforms Australia via NCRIS. The team used advanced chemical analysis to measure small molecules (metabolites) in tumour samples from patients treated with a drug that blocks a faulty enzyme called mutant IDH. They measured levels of a chemical called 2-hydroxyglutarate (2-HG), which builds up when this enzyme does not work properly. A reduction in 2-HG levels indicated that the treatment was working as intended.
The trial targeted low-grade gliomas, a slow-growing but incurable form of brain cancer. Ten patients were treated with an oral inhibitor known as safusidenib, designed to attack the IDH1 gene mutation. Several patients experienced tumour shrinkage of up to 90 per cent. Importantly, the trial confirmed that the drug successfully crossed the blood–brain barrier, something long thought unattainable.
Professor Kate Drummond, Director of Neurosurgery at the Royal Melbourne Hospital and lead investigator, described the platform as revolutionary. “This disease hasn’t had a new treatment since about 2005. With Brain POP, we can now see if a drug is reaching the brain and working against the tumour, which completely changes how we design clinical trials.”
A strength of Brain POP is its use of spatial biology, which creates detailed three-dimensional maps of tumours. This technology shows not only how the cancer responds to treatment but also how cells interact with each other and the immune system. Such knowledge could accelerate the development of personalised therapies and help repurpose existing drugs.
Dr Jim Whittle, a medical oncologist at Peter Mac and laboratory head at The Brain Cancer Centre and WEHI, said: “With Brain POP, we can look directly at how brain cancer cells respond to treatment. This allows us to move towards more precise and personalised therapies.”
While the initial results focused on low-grade gliomas, researchers believe the platform could also be applied to aggressive glioblastomas.
The findings, published in Nature, mark a turning point in brain cancer research. By enabling researchers to see how drugs truly interact with tumours, Brain POP offers new hope for patients and a faster path to treatment. This achievement also highlights the critical role of national research infrastructure, with NCRIS providing the advanced capabilities that make discoveries of this scale possible.
Information in this article has been adapted from The Australian.