Landmark decision
This is a landmark decision that will improve treatment options for children who have low and high-grade gliomas with a BRAF V600E mutation.
The approval of this treatment marks the first new treatment in decades. It has the potential to improve survival and quality of life for children facing this devastating diagnosis.
We are pleased that the National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) have approved the use of these drugs on the NHS. It is such positive news for the brain tumour community!
This announcement builds on many years of research, some of which we funded nearly 20 years ago. And also involved our Trustee, Suki Sandhu who shared her personal experience at the NICE Technical Appraisal. The Charity has also been a registered stakeholder throughout the appraisal process.
We are delighted that NICE and the SMC have 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.”
Dr Michele Afif – CEO of The Brain Tumour Charity
Dabrafenib and trametinib
Dabrafenib and trametinib are drugs known as ‘cancer growth blockers’. They target tumour cells that are growing and multiplying uncontrollably as a result of a DNA mutation in the BRAF V600 gene.
These drugs help to slow or stop the growth of tumour cells by blocking proteins that cause cells to uncontrollably grow and multiply.
Between 30 and 60 children a year in England could be eligible for this treatment which involves taking dabrafenib tablets twice a day and trametinib in liquid form once a day.
Clinicians estimate that the BRAF V600E mutation affects 15-20% of paediatric low-grade gliomas and between five and 10% of paediatric high-grade gliomas. (1,2).
Longer and better lives
Clinical trials show that the combination of dabrafenib and trametinib is more effective than radiotherapy and chemotherapy, and that it has fewer side effects in both children with low and high-grade gliomas – helping children to live longer and better lives.
What’s next?
NICE approved the treatment in April 2024, followed by SMC approval in September 2024. The NHS now has to make this treatment available to eligible children.
The approvals in England and Scotland are important steps for ensuring equity of access for the UK. Often, decisions taken by NICE are adopted by the All Wales Therapeutics and Toxicology Centre in Wales too. But we recommend you speak to your treatment team about eligibility and access to this medication.
Treatment for children like Aaliyah
Aaliyah, 12, joined the TADPOLE-G clinical trial in 2020, 12 months after being put on ‘watch and wait’ for an inoperable brain tumour that was picked up following a routine eye test.
This clinical trial assessed how effective combination treatment with dabrafenib and trametinib was against low and high-grade childhood gliomas with a BRAF V600E mutation.
Aaliyah is grateful she’s been able to have treatment at home. She is also proud to have been part of the clinical trial, led by researchers at Great Ormond Street Hospital in London.
I’m able to just take tablets twice a day and go to the hospital every few months, rather than be in hospital to have chemotherapy. I’ve been able to start secondary school with my friends and go to pretty much all my lessons. I’ve also been able to be at home, rather than staying in hospital for treatment, and carry on my hobbies such as majorette.”
Aaliyah
She added: “I do feel proud in a way that I’ve been part of a trial that has helped other people, and I am thankful to the team who have supported me. I’m really pleased that other teenagers and children will now be able to have the tablets instead of chemotherapy, without needing to be part of a trial. That’s a pretty big thing to have been part of and will make a real difference.”
Aaliyah’s mum, Amie, said: “Three months after we started the trial, we had an MRI. That was the first time I felt able to look at the scans. When I saw the before and after, that was probably the day a massive weight was lifted off my shoulders. I didn’t expect it to shrink that much, I knew we’d made the right decision.”
References
- Bouffett, E, et al. (2023). ‘Dabrafenib plus Trametinib in Pediatric Glioma with BRAF V600 Mutations’, The New England Journal of Medicine 389(12), 1108-1120. Link to paper.
- Hargrave, D, et al. (2023). ‘Phase II Trial of Dabrafenib Plus Trametinib in Relapsed/Refractory BRAF V600–Mutant Pediatric High-Grade Glioma’. Journal of Clinical Oncology 41(31), 5174-5183. Link to paper.