New treatment for brain tumours approved following decades of work

The first ever targeted treatment for brain tumours in children has been approved for NHS patients, following over 20 years of research by a UCL clinician scientist.

Professor Darren Hargrave, who is a researcher at the UCL Great Ormond Street Institute of Child Health and consultant at Great Ormond Street Hospital for Children (GOSH), was the Chair of the TADPOLE-G trial steering group, alongside researchers across the world.

The results of the trial, published in the New England Journal of Medicine  and the  Journal of Clinical Oncology in 2023*, found that a combination of the drugs Dabrafenib and Trametinib can be effective in treating children with rare brain tumours, called BRAF mutated gliomas.

This week the two drugs, which are already used to treat some adult cancers, have been approved by the National Institute for Health and Care Excellence (NICE). This means they will be available as part of standard care for NHS patients, rather than just those on a clinical trial.

For children with paediatric low-grade gliomas, the normal course of treatment is either full surgical removal if possible or additional treatments like chemotherapy and radiotherapy. The low-grade glioma trial showed that the combination of Dabrafenib and Trametinib was four times more effective than chemotherapy and had half the side effects.

Meanwhile, children with high-grade gliomas after surgery require both radiotherapy and chemotherapy. Unfortunately, less than 20% of patients respond well to this treatment. But when the children were treated with Dabrafenib and Trametinib in the trial, the response rate increased to 56%.

Professor Hargrave said: "The new combination therapy is an important advancement in the field of paediatric neuro-oncology that offers an alternative to chemotherapy for low-grade gliomas and provides an additional treatment option for relapsed high-grade gliomas, where overall response rates to the current therapy options have been as low as 20% or less."

The BRAF gene was first discovered in the early 1980s. In 2002, Professor Hargrave was part of the Cancer Genome Project at the Wellcome Sanger Institute that identified BRAF mutations in human cancer.

Over the next eight years, multiple studies showed how the same mutations could also occur in childhood cancer.

In 2011, Professor Hargrave began working with pharmaceutical companies to design a clinical trial to target these mutations with Dabrafenib and Trametinib.

The first trials for the drugs began at GOSH in 2013 and the international TADPOLE-G study launched five years later - with final results published in 2023.

Professor Hargrave said: "As I was involved in the original study that identified the role of BRAF in cancer and the clinical trails that led to the NICE approval of targeted therapies for childhood brain tumours with BRAF gene mutations, it is exciting to see these treatments becoming available to patients in England and Wales."


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