World first trial to revolutionise treatment of brain cancer

A pioneering research study will trial multiple new treatments for people in the UK living with glioblastoma, an aggressive form of brain cancer.
Funded by Cancer Research UK and Minderoo Foundation, an Australian charity, the trial will for the first time enable researchers to adapt the treatment provided to each group of participants, meaning multiple new drugs can be tested in a short period. As part of the trial, every patient will also have their genome sequenced, enabling researchers to target their treatment with greater precision, the charities said.
Glioblastomas are the most common type of cancerous brain tumour in adults, making up nearly a third (32%) of primary brain tumours diagnosed in England between 1995 and 2017.
Due to the unique biology of the brain, however, finding new treatments has proven difficult. There have been over 1,000 brain cancer clinical trials over the past two decades, but these have not yielded significant breakthroughs.
To help address this, Cancer Research UK and Minderoo Foundation are each committing £1.68m (£3.36m total) to fund the “next-Generation aGile Genomically Guided Glioma platform trial”, also known as the 5G platform trial. The hope is that the unique approach taken by this trial will help speed up the development of new treatments whilst benefiting patients.
The 5G platform trial is sponsored by The Institute of Cancer Research, London, and conducted by the Joint Drug Development Unit at The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research (ICR).
Chief Executive of Cancer Research UK, Michelle Mitchell said:
“Brain tumours are notoriously hard to treat because we still don’t know enough about the biology of the disease, and current treatments are not effective enough.
“This new trial offers something different because researchers will use the DNA of participants to help them target treatments. Whole genome sequencing tests can give clues about how a cancer may have developed, how it will behave and what treatment options would work best.
“More research is needed, but it is hoped that the technology could one day be used within the NHS to improve the treatment and care of people with cancer.”
The 5G platform study is a world-first adaptive clinical trial platform for patients with brain tumours. The drug or drug combination a patient receives will be based on the specific genetic makeup of their cancer. The trial design also allows treatments developed for other types of cancer to be trialled in patients with brain cancer for the first time.
Incorporating flexibility into the design means that the drugs given to patients can be changed as real-time data is collected and incorporated, making it easier for researchers to try multiple treatment options across different groups of patients. This both benefits the trial itself and improves the likelihood of a positive outcome for participants.
Dr Juanita Lopez from the Institute of Cancer Research and Dr Richard Mair at the Cancer Research UK Cambridge Centre will co-lead the trial.
Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, London, Dr Juanita Lopez, said:
“We have seen progress made in better treating many types of cancer in recent decades, but less so in brain cancers like glioblastoma.
“The 5G platform trial presents a unique opportunity to work in new and exciting ways to find better treatment options for this patient population.”
Honorary Consultant Neurosurgeon at Cambridge University Hospitals NHS Foundation Trust and co-lead of the Brain Cancer Virtual Institute at the Cancer Research UK Cambridge Centre, Dr Richard Mair said:
“Brain cancers like glioblastoma are notoriously difficult to treat. I am excited to co-lead this world-first trial designed with this cancer type in mind.
“The use of whole genome sequencing for every patient on the trial performed in ‘real time’ enables us to be both agile and precise in our drug targeting. We hope that this approach will help patients with this cancer of unmet need.”
The first 5G platform trial sites opened in August 2024, with further sites planned across the UK as the study progresses. The drugs used in the study will be provided by pharmaceutical industry partners.
The trial involves researchers at the Cancer Research UK-funded ICR Clinical Trials and Statistics Unit (ICR-CTSU) and is supported by the National Institute of Health Research Biomedical Research Centre (NIHR BRC) at the Royal Marsden and the ICR. It is taking place in partnership with the Minderoo Precision Brain Tumour Programme, which is sponsored by Cambridge University Hospitals NHS Foundation Trust and the University of Cambridge.
The funding of this study forms part of Cancer Research UK’s long-term commitment to investing in brain cancer research. Cancer Research UK recently announced £8m additional funding for our two Brain Tumour Centres of Excellence.
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