Children in Scotland diagnosed with a brain tumour that has a specific genetic mutation can now be treated on the NHS with a kinder, more effective targeted therapy.
The Scottish Medicines Consortium has today approved prescribing dabrafenib to treat children aged one and above who have been diagnosed with a low- or high-grade glioma that has the BRAF V600E mutation: https://scottishmedicines.org.uk/medicines-advice/dabrafenib-finlee-full-smc2667/
It’s used in combination with another medicine, trametinib, and both can be administered at home as they are taken orally. This means fewer hospital visits.
The news follows the approval given in April this year by the National Institute for Health and Care Excellence (NICE).
Current standard treatment for the condition typically involves surgery – where that’s possible – chemotherapy and/or radiotherapy. But less than 30% of children diagnosed with high-grade gliomas survive for five years or more and the treatment often has brutal side effects.
Outcomes for low grade gliomas – the most common childhood brain tumour – are better at a 90% survival rate following surgery. But chemotherapy may also be prescribed whereas this treatment means improved quality of life for patients as clinical trials showed its side effects were rare.
The combination medication is the first targeted treatment to be made available on the NHS for paediatric gliomas. It is already licensed for use in the USA, and for use in the UK in adults with the BRAF mutation who have some types of lung and skin cancers.
The two drugs work together to block the growth signal coming from the mutant BRAF protein, slowing or even stopping the tumour from growing.
Brain tumours affect around 500 children a year in the UK, of whom between 150 to 175 may have a glioma and fewer still will have the BRAF mutation. But despite the small numbers involved, this is the first progress made in treating the disease in years.
In 2008, research carried out by Dr David Jones which was funded by The Brain Tumour Charity (then known as the Samantha Dickson Brain Tumour Trust), identified a BRAF mutation that was common in pilocytic astrocytoma brain tumours.
This was the first time that a specific genetic change had been detected in these tumour types and it paved the way for drug development as it could then become a target for diagnostic tests, changing the research landscape for low grade brain tumours in children.
Dr Jones now leads the EVEREST Centre in Germany, which carries out research into paediatric low grade brain tumours.
Dr Michele Afif, Chief Executive at The Brain Tumour Charity, said: “We are delighted that the Scottish Medicines Consortium has joined NICE in approving the first new treatment for paediatric brain tumours in decades.
“We hope that this will be the first of many new treatments that will ensure our community can live longer and better lives.”
To find out more about The Brain Tumour Charity, visit:
www.thebraintumourcharity.org/]www.thebraintumourcharity.org/