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World-first trial launches to revolutionise the treatment of brain cancer

A pioneering research study that's open at Addenbrooke's will trial multiple new treatments for people in the UK living with glioblastoma, an aggressive form of brain cancer.

A scientist placing a pipette into a tube, wearing a blue glove
Image credit: Minderoo Foundation

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.

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, an Australian charity, 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.

It is taking place in partnership with the Minderoo Precision Brain Tumour Programme , which is sponsored by Cambridge University Hospitals NHS Foundation Trust (CUH), and the University of Cambridge.

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.

Dr Richard Mair consultant neurosurgeon at CUH and Dr Juanita Lopez from the Institute of Cancer Research and will co-lead the trial.

A man smiling
Dr Richard Mair

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.

Dr Richard Mair

Dr Richard Mair, Honorary Consultant Neurosurgeon at CUH and co-lead of the Brain Cancer Virtual Institute at the Cancer Research UK Cambridge Centre, continued:

“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 Cambridge and London in August 2024, with further sites planned to open across the UK as the study progresses. The drugs used in the study will be provided by pharmaceutical industry partners. 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).

Targeted treatments

The 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, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, London, 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.”

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.

Michelle Mitchell, CEO, Cancer Research UK

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.

“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.”

Co-founder of Minderoo Foundation, Dr Andrew Forrest AO, said:

“Minderoo Foundation was established to take on tough and persistent challenges. There are few greater challenges than brain cancer. 

“Our investment in this ground-breaking study reinforces Minderoo’s commitment to being part of a world where cancer treatment options are not constrained, and hope is available for all – no matter how common or rare your cancer is."

This targeted treatment approach underpins the vision of the new Cambridge Cancer Research Hospital, a partnership between Addenbrooke's Hospital, the University of Cambridge and the Cancer Research UK Cambridge Centre.

By bringing world-leading scientists and clinical expertise together, the new hospital will deliver breakthroughs and innovations to change the way we detect and treat cancer, to diagnose the disease at a much earlier stage and deliver personalised, precision medicine.