NICE approval of combination treatment for paediatric brain tumour

24 April 2024 | Treatments, Campaigning and research

Novartis announced today that the National Institute for Health and Care Excellence (NICE) has published its Final Draft Guidance NICE has recommended the combination therapy of dabrafenib (Finlee®) and trametinib (Spexotras®) for the treatment of children with low-grade glioma (LGG) and high-grade glioma (HGG) who have a BRAF V600E mutation¹. BRAF V600E mutation is present in 15-20%² of LGGs and in 5-10%³ of HGGs in children, and is associated with poor survival outcomes and a less favourable response to chemotherapy².

The new treatment made available by NICE introduces a therapeutic option for children and young adults with relapsed/refractory mutation-positive paediatric high-grade glioma – an aggressive tumour – where there was previously no recognised standard of care³. The recommendation makes available for use on the NHS the liquid form of the treatment, suitable for patients as young as one year of age.

The NICE recommendation is based on data from the TADPOLE clinical trial led by Great Ormond Street Hospital for Children (GOSH) in London. Researchers from around the world contributed to the trial, which is the first study to test if the combination treatment can be used to treat BRAF mutated brain gliomas in very young children. The combination of dabrafenib + trametinib, may help to slow tumour growth by blocking signals associated with the BRAF and MEK kinases that are implicated in the growth of various types of cancer.

Current treatments for people with glioma are limited and include surgery, radiotherapy, chemotherapy, and best supportive care. NICE’s independent appraisal committee heard from patient experts that treatment for a potentially life-threatening brain tumour glioma can affect children’s education and socialising, and have a lasting emotional impact. Additionally, the side effects of current chemotherapy treatment can be severe, leading to people with glioma and their caregivers choosing to stop treatment.

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.”

Dr Lucy Wilkinson, Support Services Manager for Brain Tumour Support, reflected on the impact for families:

The approval of this new targeted treatment for paediatric gliomas is an important development for families affected. It also highlights the complex impact that a diagnosis can have for a child’s development and the wider effect on the whole family, making specialist support and wraparound care so vital. This less harsh, home-administered treatment therefore has potential to significantly improve quality of life for the whole family and is very much welcomed.

References:

1. NICE Guidance. Final Draft Guidance (FDG). Dabrafenib with trametinib for treating BRAF V600E mutation-positive glioma in children and young people aged 1 to 17 [ID5104].
2. Science Direct. CNS tumours The first study of dabrafenib in pediatric patients with BRAF V600–mutant relapsed or refractory low-grade gliomas . 
3. PubMed. Phase II Trial of Dabrafenib Plus Trametinib in Relapsed/Refractory BRAF V600-Mutant Pediatric High-Grade Glioma | Journal of Clinical Oncology 

About Novartis

Novartis is an innovative medicines company. Championing health and lives through pioneering NHS partnerships, innovative collaborations and a clear focus on the greatest healthcare challenges.  www.novartis.com/uk-en/