In a significant advancement for brain cancer treatment, researchers have published promising results in Nature Medicine detailing a novel immunotherapy approach for glioblastoma, one of the most aggressive forms of brain cancer. The experimental treatment, developed by Professor Georgina Long AO from the University of Sydney and Melanoma Institute Australia, marks the first documented use of neoadjuvant triple immunotherapy in glioblastoma patients.
The innovative treatment protocol involves administering three checkpoint inhibitor immunotherapies prior to surgical intervention. These drugs work by activating the immune system and programming T-cells to target and eliminate tumor cells. This approach draws from successful strategies previously employed in treating melanoma, including cases where cancer had spread to the brain.
Promising Initial Results
Analysis of the surgically removed tumor revealed encouraging findings. Compared to pre-treatment samples, the treated tumor demonstrated significantly increased diversity, abundance, and activation of immune cells. This enhanced immune cell presence suggests a robust immune response against the cancer. Notably, the treated patient has shown no clear signs of cancer recurrence after more than 18 months post-treatment.
"My hypothesis was that we could administer combination immunotherapy as first line treatment before surgery to boost the immune system and activate T-cells to target the brain tumor," explained Professor Long, the study's lead author. "This has never been done before."
Current Treatment Landscape
The development represents a potential breakthrough for glioblastoma treatment, a condition with historically poor outcomes. Current statistics paint a grim picture: patients face an average survival time of just 12 to 18 months, with only 25% surviving beyond one year and less than 5% reaching the three-year mark.
Advancing to Clinical Trials
The Brain Cancer Centre is now preparing to lead an Australian-led international clinical trial to rigorously evaluate this approach. Dr. Jim Whittle, a leading neuro-oncologist at Peter MacCallum Cancer Centre and The Royal Melbourne Hospital, will spearhead the investigation.
The upcoming trial will focus on testing double immunotherapy, with some patient cohorts receiving a combination of immunotherapy and chemotherapy. "While we are buoyed by the results of this experimental treatment so far, a clinical trial in a large group of patients would need to happen before anyone could consider it a possible breakthrough," Professor Long noted.
Research Infrastructure and Support
The trial will be conducted through The Brain Cancer Centre, established in partnership with WEHI and supported by the Victorian Government. The center brings together an extensive network of oncologists, immunologists, neurosurgeons, bioinformaticians, and cancer biology experts.
Dr. Whittle, who also leads a laboratory at WEHI, emphasized the meticulous nature of the trial development process: "We are pleased to be able to build on this exciting work by diving into the process of designing a clinical trial, which takes time, care and accuracy."
