‘Pediatric treatment’ for brain cancer in children is offered by NHS in England
The NHS is to offer children with brain tumors in England a groundbreaking new targeted drug therapy to tackle the disease – a development that charities are hailing as the biggest breakthrough in decades.
Gliomas are the most common form of brain cancer in children, but experts say the standard treatment with chemotherapy can be brutal and grueling, and also carries the risk of side effects such as weight loss, seizures and headaches.
A pediatric drug treatment has been given the green light by the National Institute for Health and Care Excellence (Nice). Studies have shown that it caused fewer side effects, improved children’s response to treatment, and improved their survival time without worsening the disease.
Dabrafenib with trametinib was shown to stop tumor growth for more than three times as long as standard chemotherapy for children with low-grade gliomas that have a specific genetic mutation, while also helping to prevent many of the serious side effects of chemotherapy.
The treatment will initially be available on the NHS in England for people aged 1 to 17 years with low- or high-grade gliomas who have a BRAF V600E mutation.
The combination treatment, which can be administered at home rather than in hospital, works by targeting the proteins made by the altered BRAF gene that are responsible for uncontrollable tumor growth.
Gliomas grow in the brain or spinal cord and can be low-grade, where tumors grow slowly, or high-grade, where they grow faster and can be fatal. Approximately 150 children in the UK are diagnosed with low-grade gliomas and around 30 with high-grade gliomas each year.
Clinical trials showed that the treatment, which had fewer side effects than chemotherapy, halted the growth of low-grade gliomas for an average of about two years (24.9 months) – more than three times as long as standard chemotherapy (7.2 months). says England.
In some cases, the tumors disappeared, although longer-term follow-up of patients is needed.
Dabrafenib is given as soluble tablets 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 tumor growth.
Dr. Michele Afif, the CEO of the Brain Tumor Charity, said: “We are delighted that Nice has approved the first new treatment for pediatric brain tumors in decades.
“While this will only affect a small population, it is of great importance to them and their loved ones and represents real progress. We hope this will be the first of many new treatments that will help our community live longer, better lives.”
Suki Sandhu, whose eight-year-old son Raj died six years ago from high-grade glioma, welcomed the rollout of the new therapy on the NHS.
“I had to make the decision to stop chemotherapy treatment for my son because he was suffering from terrible side effects after years of harsh treatment. It was one of the hardest decisions I’ve ever had to make… We need pediatric drugs and new treatments for people with brain tumors, like this treatment, and I’m hopeful that other families will continue to have access to these medications and, hopefully , stay disease-free for longer and live a good quality life.”
Prof. Peter Johnson, National Clinical Director for Cancer at NHS England, said: “It is a significant step forward in the treatment which has been shown to be easier to deliver than chemotherapy and highly effective at blocking the growth of the disease, giving children a better quality of life and longer.
“It can also be taken at home, meaning children and teenagers can spend less time in hospital for treatment and more time with their loved ones and doing things they enjoy.”