Young people with brain cancer to benefit from first ever targeted treatment

NICE has recommended the first ever targeted treatment for children with a specific form of brain cancer.

The combination treatment of dabrafenib (Finlee) and trametinib (Spexotras) is recommended for people aged one to 17 with low- and high-grade gliomas with a specific genetic mutation (known as BRAF V600E mutation-positive glioma).

Dabrafenib is given as dissolvable tablets which are taken twice a day and trametinib is an oral solution which is taken once a day.

Professor Peter Johnson, NHS National Clinical Director for Cancer, said the therapy is “very effective in blocking the growth of the disease” and avoids the need for chemotherapy, enabling patients to have “a better quality of life”.

Gliomas the most common type of brain cancer found in young people

Gliomas are the most common type of cancerous brain tumour in children and young people. The tumours develop in the glial cells (such as astrocytes and oligodendroctyes) which support neuron function in the brain and spinal cord.

Grade I or II tumors are termed low-grade gliomas (LGG), while Grade III and IV tumours are classified as high-grade gliomas (HGG). Around 150 children are diagnosed with LGG every year in the UK and around 30 are diagnosed with HGG, with up to one fifth having the BRAF gene mutation.

Symptoms of gliomas include seizures, cognitive symptoms (such as memory loss), headache, nausea and vomiting, weakness or numbness.

The current treatments for people with glioma are limited and include surgery, radiotherapy, chemotherapy, and best supportive care.

30 children expected to benefit from new treatment

BRAF causes the body to make faulty proteins which in turn cause tumours to develop in the brain. These tumours are more resistant to chemotherapy and are therefore harder to control.

Dabrafenib with trametinib works by targeting these proteins, and clinical trials show that the combination treatment stops the disease progressing for more than three times as long as standard chemotherapy (24.9 months vs 7.2 months).

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.

It is estimated that around 30 children in England could be eligible for the treatment, which is expected to be available in the coming months.

Treatment will help brain cancer patients avoid harsh side effects of chemotherapy

Patient experts told NICE’s independent appraisal committee that this new treatment will help brain tumour patients avoid the harsh side effects of chemotherapy such as weight loss, seizures, and headaches, which can lead people with glioma to stop treatment.

Aaliyah, 12, who has glioma, explained how taking dabrafenib and trametinib has benefitted her.

“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,” she said. “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.

“I’m really pleased that other teenagers and children will now be able to have this treatment instead of chemotherapy, without needing to be part of a trial, it will make a real difference.”

Dr Michele Afif, Chief Executive at The Brain Tumour Charity, has welcomed NICE’s approval. He said: “We are delighted that NICE has 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.”