A groundbreaking experimental treatment for glioblastoma, one of the deadliest brain cancers, has been published in Nature Medicine, bringing new hope for patients.
The treatment, developed by Professor Georgina Long AO from the University of Sydney, is now set to be tested in a large-scale clinical trial led by researchers at The Brain Cancer Center.
Glioblastoma is an aggressive and fast-growing brain cancer with a poor survival rate. Most patients live only 12 to 18 months after diagnosis. Only a quarter survive beyond one year, and less than 5% make it past three years. Finding an effective treatment has been a major challenge, as the cancer is resistant to most existing therapies.
A New Approach to Treatment
Professor Long, a medical oncologist and expert in melanoma treatment, applied her knowledge of immunotherapy to glioblastoma. Immunotherapy is a type of treatment that stimulates the body’s immune system to attack cancer cells.
In this case, she used a combination of three checkpoint inhibitor drugs before surgery—an approach known as neoadjuvant triple immunotherapy.
Checkpoint inhibitors work by activating T-cells, the immune system’s attack cells, to target and destroy tumors. The goal of using this therapy before surgery was to strengthen the immune response against the tumor, making it easier to remove and reducing the chances of the cancer returning.
The published paper describes the results of this experimental treatment on a glioblastoma patient. When the tumor was removed, it showed a higher number of active immune cells compared to before the treatment.
This suggests that the therapy helped trigger a strong immune response against the cancer. At the time the paper was submitted, the patient had remained cancer-free for over 18 months—significantly longer than the average survival time.
“This has never been done before,” said Professor Long. “My hypothesis was that using immunotherapy before surgery could activate T-cells to fight the brain tumor. It worked in melanoma, and now we are testing whether it can work in glioblastoma.”
Moving Toward a Clinical Trial
While these early results are promising, a larger trial is needed to determine whether this treatment is truly effective. To scientifically test the approach, an international clinical trial will be launched within a year, led by The Brain Cancer Center.
Unlike the initial case, the trial will test a two-drug (double immunotherapy) approach. Some patients will also receive chemotherapy to see if combining treatments improves outcomes. Dr. Jim Whittle, a neuro-oncologist at the Peter MacCallum Cancer Center and The Royal Melbourne Hospital, will lead the study.
“This is an exciting step forward,” said Dr. Whittle. “We now need to carefully design a clinical trial that will allow us to test this treatment in a large group of glioblastoma patients.”
Professor Long, who helped secure access to the necessary drugs for the trial, emphasized the importance of further testing. “While we are encouraged by the early results, a clinical trial is essential before anyone can consider this a real breakthrough.”
What This Means for Brain Cancer Research
Glioblastoma has long been one of the most challenging cancers to treat, with little progress in improving survival rates. Immunotherapy has revolutionized the treatment of other cancers, such as melanoma, but it has not yet been widely used for brain tumors.
This study offers a potential new path forward, suggesting that boosting the immune system before surgery may help fight the cancer more effectively.
If the clinical trial confirms the success of this approach, it could change how glioblastoma is treated in the future. While the trial is still in its early stages, it represents a significant step toward improving outcomes for patients with this devastating disease.
The Brain Cancer Center will announce recruitment details for the trial at a later date. Participation will be limited to eligible glioblastoma patients.
The research findings can be found in Nature Medicine.
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