A peer-reviewed paper detailing the experimental cancer treatment developed by Professor Georgina Long AO, University of Sydney medical oncologist and Medical Director of the Melanoma Institute Australia, has today been published in Nature Medicine, paving the way for a clinical trial to be conducted by researchers at the Brain Cancer Centre in Melbourne.
The paper details the experimental treatment given to a patient who was diagnosed with glioblastoma, a highly aggressive brain cancer. Professor Long used her expertise in immunotherapy and drew on melanoma science to devise, lead and administer the treatment. It is the first documented use of neoadjuvant triple immunotherapy in glioblastoma, involving a combination of three checkpoint inhibitor immunotherapies (drugs that activate the immune system, instructing T-cells to kill tumour cells) administered prior to surgery.
When resected, the tumour treated with immunotherapy showed increased diversity, abundance, and activation of immune cells compared to the tumour prior to receiving immunotherapy. These immune cells may recognise and attack cancer cells: their increased presence may suggest a strong immune response. At the time of final submission of the paper on 9 January 2025, the patient had no clear signs of cancer recurrence after more than 18 months.
“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 tumour -- an approach I had previously developed successfully in both stage 3 melanoma and melanoma that had spread to the brain,” said lead author, Professor Georgina Long. “This has never been done before and what this trial will do is establish whether this approach is feasible or effective for the treatment of glioblastoma.”
An Australian-led international clinical trial will scientifically investigate the efficacy of the approach within a large cohort of eligible glioblastoma patients and could commence within a year. It will trial the use of double immunotherapy. In some patients, it will be combined with chemotherapy.
The Brain Cancer Centre has world-leading expertise in glioblastoma and was established in partnership with the Walter and Eliza Hall Institute of Medical Research (WEHI) with support from the Victorian Government.
“I am delighted to be handing the baton to Dr Jim Whittle, a leading Australian neuro-oncologist at the Peter MacCallum Cancer Centre, The Royal Melbourne Hospital and Co-Head of Research Strategy at the Brain Cancer Centre to commence a broader scientific study to scientifically determine if – and how – this process might work in treating glioblastoma,” Professor Long said, who also secured drug access for the clinical trial.
“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,” she said.
Dr Whittle said: “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. When that process is complete, the result will be a world first clinical trial that enables us to thoroughly test the hypothesis against a representative sample of patients.”
People with glioblastoma have an average survival time of 12 to 18 months, with only 25 percent of patients surviving more than one year and less than five percent surviving more than three years.
Last week, Professor Long published a paper in The Lancet Oncology which detailed the development of a dual combination immunotherapy which has seen to a seven-year survival rate in 51 percent of patients with melanoma that had metastasised in the brain. The previous survival rate was 16 weeks.
About Professor Georgina Long
Professor Georgina Long AO is the Medical Director of the Melanoma Institute Australia and chair of melanoma medical oncology and translational research at the University of Sydney. A former Australian of the Year for her contributions to melanoma research and treatment, she has led melanoma drug development and clinical trials at the Melanoma Institute Australia, transforming experimental drug therapies to be a standard of care for melanoma patients, including neoadjuvant drug therapy.
About Dr Jim Whittle
Dr Jim Whittle is the Co-Head of Research Strategy of The Brain Cancer Centre and co-designed the Brain Perioperative Clinical Trial Platform (BPOP), a platform funded by the Victorian State Government, which will be used across many clinical trials. BPOP is delivered by partners at the Royal Melbourne Hospital, Peter MacCallum Cancer Centre and WEHI.
DISCLOSURE
As this announcement deals with the launch of a clinical study, the patient subject of the experimental treatment published in Nature Medicine has not been named in the media release.
Commencement of recruitment for the clinical trial will be announced by The Brain Cancer Centre at a later date and will be limited to eligible patients.
The authors received no specific funding for this work. Drug company Bristol Myer Squibb provided the drugs used for the experimental treatment. Investigators were supported by the Melanoma Institute Australia, National Health and Medical Research Council Investigator grants, Cancer Institute NSW grants, NSW Health Early-Mid Career grants, the University of Sydney Medical School Foundation, the CLEARbridge Foundation, and PhD scholarships from the University of Sydney and Melanoma Institute Australia.
The author declare competing interests. For the full list of author contributions and competing interests, please visit Nature Medicine.
Research:
Long, G. et al. (2025), Neoadjuvant Triplet Immune Checkpoint Blockade in Newly Diagnosed Glioblastoma (Nature Medicine). DOI: 10.1038/s41591-025-03512-1
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