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Dabrafenib with trametinib is a targeted treatment that can be taken at home rather than hospital and improves the length and quality of life for patients.

A new targeted drug combination treatment, described as a ‘step-change in care’ for children and young people with an aggressive form of brain cancer, has today (Wednesday, 24 April) been recommended by NICE. 

Dabrafenib (also known as Finlee) in combination with trametinib (also known as Spexotras, both made by Novartis) is being recommended in final draft guidance for treating BRAF V600E mutation-positive glioma.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, 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. The current treatments for people with glioma are limited and include surgery, radiotherapy, chemotherapy, and best supportive care. 

Patient experts told NICE’s independent appraisal committee that treatment for a potentially life-threatening brain tumour glioma can delay children’s education, restrict socialising, and have a lasting emotional impact. They highlighted the side effects of chemotherapy, which can include weight loss, seizures, and headaches can lead to people with glioma and their caregivers choosing to stop treatment. 

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 medicines are known as ‘cancer growth blockers’ and target the proteins affected by the BRAF gene and slow down or stops 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 the tumour growth in people with LGG for an average of more than two years – three times longer than current drugs. 

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.  

“NICE is determined to get the best care to patients fast and ensure value for the taxpayer. 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 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.”

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.” 

The company 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.  

The research for this treatment was supported by the NIHR Clinical Research Network. 

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.