NICE gives green light to life-extending drug combo for brain cancer
Children and teenagers with an aggressive form of brain cancer are set to benefit after a new life-extending drug combination treatment was today recommended for NHS use.
The treatment, described as a ‘step-change in care’ for children and young people with an aggressive form of brain cancer, has been backed by the National Institute for Health and Care Excellence.
“I am pleased we can recommend this new combination therapy that can give children longer without their tumour growing”
Helen Knight
Dabrafenib (Finlee) with trametinib (Spexotras) is a targeted treatment that can be taken at home rather than hospital and improves the length and quality of life for patients.
Dabrafenib in combination with trametinib – both of which are made by Novartis – is being recommended by NICE in final draft guidance for treating BRAF V600E mutation-positive glioma.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, and develop from the glial cells that support the nerve cells of the brain and spinal cord, noted NICE.
It said that BRAF was a specific gene mutation that causes the body to make faulty proteins, which in turn cause tumours to develop in the brain.
It is classified as either low-grade glioma (LGG), where tumours do not grow or grow slowly, or high-grade glioma (HGG), where tumours grow more rapidly and is usually fatal.
According to NICE, the treatments previously available for people with glioma were limited and included surgery, radiotherapy, chemotherapy, and best supportive care.
It is estimated that around 30 children in England could be eligible to take dabrafenib with trametinib.
Dabrafenib is given as tablets, which are taken twice a day, and trametinib is an oral solution, which is taken once a day.
The drugs are known as ‘cancer growth blockers’ and target the proteins affected by the BRAF gene, slowing or stopping the development of tumours. It is already used in several other forms of cancer.
The 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.
NICE highlighted that clinical trials showed the new treatment stalled the tumour growth in people with LGG for an average of more than two years – three times longer than current drugs.
Novartis has a confidential commercial arrangement for each medicine through a simple discount patient access scheme, which makes dabrafenib plus trametinib available to the NHS with a discount.
Helen Knight, director of medicines evaluation at NICE, said: “Diagnosis of a glioma brain tumour, which is often fatal for people with advanced high-grade glioma, can have a devastating impact on children and their families. Treatment options are limited, and we know they can be brutal.
“I am pleased we can recommend this new combination therapy that can give children longer without their tumour growing and offers them and their families a better quality of life,” she said.
She added: “This recommendation follows the licensing of both treatments for this type of cancer within the last three months.”
Professor Peter Johnson, NHS national clinical director for cancer, said: “It is fantastic news that this new and kinder 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.”
Under the guidance, dabrafenib with trametinib is recommended as an option for treating low-grade glioma (LGG) with a BRAF V600E mutation in children and young people aged one year and over who need systemic treatment.
It is also recommended as an option for treating high-grade glioma (HGG) with a BRAF V600E mutation in children and young people aged one year and over after at least one radiation or chemotherapy treatment.
Trial data found progression-free survival for people with LGG was 24.9 months with dabrafenib plus trametinib and 7.2 months with vincristine with carboplatin, another treatment combination. For those with HGG the median progression-free survival was nine months.
