The first ever targeted treatment for brain tumours in children has been approved for NHS patients, offering an ­alternative to chemotherapy, with fewer side effects.

The breakthrough comes after more than 20 years of research by University College London Professor Darren Hargrave, alongside researchers across the world.

The trial results found that a combination of the drugs Dabrafenib and Trametinib can be effective in treating children with rare brain tumours, called BRAF mutated gliomas. The BRAF gene was first discovered in the early 1980s.

In 2002, Professor Hargrave, a researcher at UCL and Great Ormond Street Hospital for Children, 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 Great Ormond Street in 2013 and the international TADPOLE-G study launched five years later, with final results published in 2023. Only recently 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’ll be available as part of standard care for NHS patients, rather than just those in a clinical trial. For children with low-grade gliomas, the normal course of treatment is either surgical removal if possible, or additional treatments such as ­chemotherapy and radiotherapy.

The low-grade glioma trial showed that the combination of Dabrafenib and Trametinib was four times more effective than chemotherapy, with 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, who was the chair of the TADPOLE-G trial steering group, 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.”

He added: “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.”