Groundbreaking Brain Cancer Trial Opens Doors for New Hope in the US
A revolutionary clinical trial, aiming to transform the treatment of glioblastoma, the most aggressive form of brain cancer, has officially commenced in the United States. This world-first initiative offers a beacon of hope for patients battling this formidable disease, a journey that began with the pioneering efforts of Professor Richard Scolyer, Australia’s 2024 Australian of the Year.
Professor Mustafa Khasraw, a leading Australian medical oncologist based at Duke University in North Carolina, is spearheading the trial. His focus is to rigorously assess whether immunotherapy drugs can significantly improve outcomes for individuals diagnosed with the same lethal brain tumour that Professor Scolyer himself is confronting.
“I’m incredibly excited that this trial has commenced,” Professor Scolyer expressed. “It takes a considerable amount of time to get trials approved and initiated, but to be actively recruiting patients in the United States – and hopefully here in Australia soon – means we can truly begin to test these drugs and determine if they can make a tangible difference.”
The ambitious trial will involve multiple prestigious cancer centres across the US, including locations in Los Angeles, San Francisco, Houston, and New York, in addition to Duke University. The study is structured in two distinct phases.
Phase One: This initial stage is dedicated to meticulously evaluating the safety of the experimental treatment. A small cohort of newly diagnosed patients will receive the treatment to ensure its tolerability and identify any potential side effects.
Phase Two: Building on the safety data from the first phase, this larger stage will involve a significantly greater number of patients. Participants will be randomly assigned to receive either one or two immunotherapy drugs – Nivolumab and Relatlimab – administered before surgery. Following the surgical intervention, these immunotherapy treatments will be combined with more conventional therapies, such as radiotherapy and chemotherapy.
Professor Scolyer’s personal battle with glioblastoma, specifically the IDH wild-type variant, began in mid-2023. Recognising the grim prognosis and the limited advancements in conventional brain cancer treatments over nearly two decades, his colleague and fellow medical oncologist, Professor Georgina Long, proposed an audacious plan. Drawing inspiration from the success of immunotherapy in treating advanced melanoma patients, they decided to explore its potential for brain cancer.
“Even if it only afforded me a few extra months of life, it was going to yield crucial information quickly,” Professor Scolyer explained. “The fact that I am still alive and functioning well long after my diagnosis certainly provides a significant amount of hope.”
Professor Scolyer, alongside Professor Long, was devastated to learn in March of last year that his tumour had returned, defying the initial prognosis of only a few months to live. Despite his ongoing health challenges, he has been actively training for a charity bike ride in Tasmania, an endeavour he recently had to interrupt due to fracturing two ribs during a fall while running.
He passionately believes that Australia’s pioneering approach to brain cancer research, driven by this trial, will ultimately benefit patients worldwide. “Well done to Georgina for forging ahead with this treatment and establishing vital connections with experts in neuro-oncology, bringing them together with innovative ideas that have been pioneered in melanoma,” he stated.
Professor Khasraw, the principal investigator for the study, referred to the trial as “GIANT” – an acronym for Glioblastoma Immunotherapy Advancement with Nivolumab and Relatlimab. He described it as a testament to “team science,” uniting clinicians and scientists from numerous international institutions with the shared goal of “bringing innovation and hope to patients.”
Currently, three patients have been enrolled in the trial, with a fourth undergoing the initial screening process. While Professor Khasraw expressed his hope for Professor Scolyer’s long-term recovery, he emphasised the scientific imperative for data. “I’m a scientist,” he asserted. “I’m driven by data. In order to believe that something is truly effective, I need to see the clinical trial data.”
Nevertheless, Professor Khasraw acknowledged the trial as a significant step forward in glioblastoma treatment. “It’s a stepping stone to generate convincing data that this therapy, or a similar approach, is likely to improve patient outcomes at the population level,” he explained. “We don’t aim for one patient to do better; our ultimate goal is for all patients to experience improved outcomes.”
Professor Khasraw anticipates that the results of the trial, which will determine if the treatment is transformative, will be available in approximately two years. The foundation for this groundbreaking trial was laid by a peer-reviewed paper authored by Professor Long, which was published in the esteemed international journal Nature Medicine last year.
In Australia, the trial will be spearheaded by researchers at the Brain Cancer Centre, an organisation established by radio presenter Carrie Bickmore following the passing of her husband, Greg Lange, from brain cancer in 2011. The Australian arm of the trial will be led by Associate Professor Jim Whittle, a distinguished neuro-oncologist at Melbourne’s Peter MacCallum Cancer Centre and co-head of research strategy at the centre.
It is important to note that the Australian trial will exclusively recruit newly diagnosed glioblastoma patients. The underlying rationale is that immunotherapy is believed to be most effective when administered to tumours before they have been subjected to radiotherapy or chemotherapy.
Professor Whittle elaborated on the trial’s objective: “What this trial will do is establish whether this approach is feasible or effective for the treatment of glioblastoma.”
