May 28, 2024

The NHS is offering children with brain tumours England A ground-breaking new targeted drug therapy to tackle the disease – a development charity is hailing as the biggest breakthrough in decades.

Gliomas are the most common type of brain cancer in children, but experts say the standard treatment of chemotherapy can be cruel and debilitating, and also carries the risk of side effects such as weight loss, seizures and headaches.

A kinder drug therapy got the green light from the National Institute for Health and Care Excellence (Nice). Studies have shown it causes fewer side effects, improves children’s response rate to treatment and their survival time without the disease worsening.

Dabrafenib with trametinib was found to stop the growth of tumors for more than three times as long as standard chemotherapy for children with low-grade gliomas who have a specific genetic mutation, while also helping to reduce many of the harsh side effects of chemotherapy save.

The treatment will initially be available on the NHS in England for people aged one 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. Around 150 children are diagnosed with low-grade gliomas in the UK each year and around 30 are diagnosed with high-grade gliomas.

Clinical trials showed that the treatment, as well as having fewer side effects than chemotherapy, stopped 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), NHS England says.

In some cases, tumors have disappeared, although long-term follow-up of patients is necessary.

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 the tumor from growing.

Dr Michele Afif, Chief Executive of the Brain Tumor Charity, said: “We are delighted that Nice has approved the first new treatment for pediatric brain tumors in decades.

“Although this will only affect a small population, it is of great importance 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.”

Suki Sandhu, whose eight-year-old son Raj died of a high-grade glioma six years ago, welcomed the introduction of the new therapy on the NHS.

“I had to make the decision to stop the chemotherapy treatment for my son because he had terrible side effects after years of harsh treatment. It was one of the hardest decisions I’ve ever had to make… We need friendlier drugs and new treatments for those with brain tumors, like this treatment, and I hope that other families will continue to gain access to these drugs and, hopefully , stay disease-free longer and live good quality lives.”

Prof Peter Johnson, NHS England’s national clinical director for cancer, said: “This is an important step forward in treatment which has been shown to be easier to take than chemotherapy and very effective at blocking the growth of the disease , which helps children to have a better quality of life for longer.

“It can also be taken at home, meaning children and teenagers can spend less time in hospital with treatment and more time with their loved ones doing things they enjoy.”

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