Published: 24 April 2024
A new targeted drug combination treatment for children and young people with an aggressive form of brain cancer has been recommended by NICE.
The treatment has been described as a ‘step-change in care'. Its development was strongly supported by the NIHR.
The treatment is Dabrafenib (also known as Finlee) in combination with trametinib (also known as Spexotras, both made by Novartis). It is being recommended in final draft guidance for treating BRAF V600E mutation-positive glioma.
Gliomas are the most common type of brain cancer in children and young people. They develop from the glial cells that support the nerve cells of the brain and spinal cord. BRAF is a specific gene mutation which causes the body to make faulty proteins. This in turn causes tumours to develop in the brain.
It is classified as either:
- Low-grade glioma (LGG), where tumours do not grow or grow slowly.
- High-grade glioma (HGG), where tumours grow more rapidly and is usually fatal.
The current treatments for people with glioma are limited. They include surgery, radiotherapy, chemotherapy and best supportive care.
Professor Darren Hargrave is clinical professor in paediatric neuro-oncology at Great Ormond Street Hospital (GOSH) and Co-Theme lead for The Accelerating Novel Therapies theme of the NIHR GOSH BRC. He said:
"Having been involved in the original study that identified the role of BRAF in cancer and the clinical trials that led to the NICE approval of targeted therapies for childhood brain tumours with BRAF gene mutations, it is exciting to see these treatments becoming available to patients in England and Wales.
"The new combination therapy is an important advancement in the field of paediatric neuro-oncology that offers an alternative to chemotherapy for low-grade gliomas and provides an additional treatment option for relapsed high-grade gliomas, where overall response rates to the current therapy options have been as low as 20% or less."
Professor Hargrave led recruitment at GOSH for each phase of the trial and was the chair of the TADPOLE-G trial steering committee (the pivotal international trial behind the treatment). The research was also supported by NIHR Clinical Research Network.
Treatment for a potentially life-threatening brain tumour glioma may delay children's education, restrict socialising and have a lasting emotional impact. Side effects of chemotherapy include weight loss, seizures and headaches.
It is estimated that around 30 children in England could be eligible to take dabrafenib with trametinib. Dabrafenib is given as tablets. These are taken twice a day. Trametinib is an oral solution which is taken once a day.
‘Cancer growth blockers'
The medicines are known as ‘cancer growth blockers'. They target the proteins affected by the BRAF gene and slow down or stop the development of tumours. The combination is already used in several other forms of cancer.
The drug combination can be used by patients with LGG who are eligible for systemic treatment or for patients with HGG who have received at least one course of radiation or chemotherapy treatment.
Clinical trials show the new treatment stalls tumour growth in people with LGG for an average of more than two years – three times longer than current drugs.
Health Minister, Andrew Stephenson said: “A child's cancer diagnosis is devastating news for any family to process. This recommendation from NICE means eligible children will now be able to access an innovative, life-extending treatment.
“We want to improve outcomes for brain cancer patients and have provided £40 million for research projects to help do so. These collective steps show the importance of harnessing the latest treatments to improve outcomes for patients, including those with glioma diagnoses.”
NICE’s recommendation stemmed from NIHR-funded external review group reports. These are produced for NICE committees each year to inform their decision making. NIHR Technology Assessment Reviews (TAR) research teams provide NICE with the independent research it needs to inform its guidance committees. This happens through a variety of different types of specialised reports, suited to the decision-making process.
TAR research teams are funded by the NIHR but are academically independent. This means the public and committee can be confident that the results are an objective overview of the evidence base.
The full final draft guidance for dabrafenib with trametinib for treating BRAF V600E mutation-positive glioma in children and young people aged 1 year and over is on the NICE website.