A new targeted treatment which could transform the lives of children diagnosed with glioma has been approved for use on the NHS.
The combination of dabrafenib and trametinib can now be used for patients aged one and over with either low-grade or high-grade glioma with the genetic mutation BRAF V600E. The two drugs, which can be taken in tablet and liquid form, work together by blocking the growth signal coming from the mutant BRAF protein and can slow or even stop the tumour from growing.
Gliomas are the most common type of brain tumour in children and young people, with around 150 children diagnosed with a low-grade glioma and 30 children diagnosed with a high-grade glioma every year. A BRAF mutation is present in around 15-20% of low-grade gliomas and 5-10% of high-grade gliomas in children.
At present, fewer than 30% of children diagnosed with high-grade gliomas survive beyond five years. While the survival rate for low-grade gliomas is higher at 90%, children undergo gruelling treatment, such as chemotherapy, which has harsh and lasting side effects.
The new drug therapy has been approved by the National Institute for Health and Care Excellence (NICE) after studies showed it lessened chemotherapy side effects and improved children’s response rate to treatment.
The treatment also increased progression-free survival time from 7.2 months with chemotherapy to 24.9 months under the new regime for those with low-grade glioma, and nine months for those with high-grade disease. In some cases, tumours completely disappeared, though longer term follow-up of patients is needed.
Aaron Wharton was diagnosed with a grade 3 anaplastic ependymoma aged four in 2020. He spent 10 weeks in hospital undergoing two surgeries and developed meningitis twice. He also underwent proton beam therapy and chemotherapy. At times, his parents were optimistic that his treatment was working, but tragically, three years after his diagnosis, in April 2023, they lost their only child at the age of seven.
His mum, Nicola (pictured top with Aaron and husband Lee), said: “It is wonderful to hear a new targeted treatment for paediatric gliomas has been approved for use on the NHS. I truly hope this will positively benefit other families going through this incredibly hard journey. Aaron was robbed of his life and from being with us. It’s unbelievable that with all the advancements in so many other cancers there is so little available for those diagnosed with brain tumours and especially children, so this is very welcome news.”
Dr Karen Noble, our Director of Research, Policy and Innovation, said: “This is a significant step forward and great news for our community. The current treatments for paediatric brain tumours can be gruelling and have challenging side effects that can significantly impact quality of life.
“Dabrafenib and trametinib represent the first approved targeted combination-therapy treatment for paediatric tumours. The treatment is less harsh and can be given at home, meaning it will significantly improve children's quality of life and give families a much-needed kinder and more effective option.”
Related reading: