New NHS treatment could transform the lives of children with brain cancer. It will be available in the coming months for young people aged 1-17 with low-grade or high-grade gliomas that have a BRAF V600E mutation.
Chemotherapy is the standard treatment for gliomas, but experts say it can be gruelling for children, and carries the risk of side-effects such as weight loss, seizures and headaches.
The new drug therapy has been approved by the National Institute for Health and Care Excellence (NICE) after clinical trials showed it reduced chemotherapy side-effects, improved children’s response rate to treatment, and improved the survival time without the disease getting worse.
Professor Peter Johnson, NHS National Clinical Director for Cancer, said: ‘It is fantastic news that this new precision treatment for children and young people with this type of brain tumour will now be available on the NHS – it is a significant step forward in treatment that has been shown to be easier to take than chemotherapy and very effective in blocking the growth of the disease, helping children have a better quality of life for longer.’
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Gliomas grow in the brain or spinal cord and can be low grade, where tumours grow slowly, or high grade, where they grow more rapidly and may be fatal.
Clinical trials have found that the new combination treatment of dabrafenib with trametinib stalled growth of low-grade gliomas for around two years (24.9 months) on average, over three times as long as standard chemotherapy (7.2 months).
Dabrafenib is given as dissolvable tablets that are taken twice a day, and trametinib is an oral solution taken once a day. The drugs work together by blocking the growth signal from the mutant BRAF protein and can slow or even stop the tumour from growing.
With about 150 children diagnosed with low-grade gliomas every year in the UK and about 30 diagnosed with high-grade gliomas, experts have welcomed the new treatment as a significant step forward in offering children a better life.
Dr Michele Afif, Chief Executive at The Brain Tumour Charity, said she is ‘delighted that NICE has approved the first new treatment for paediatric brain tumours in decades. Though this will only affect a small population, it’s of huge significance to them and their loved ones and represents real progress. We hope that this will be the first of many new treatments that will ensure our community can live longer and better lives.’