The Press and Journal (Aberdeen and Aberdeenshire)
Boost for children with brain cancer
Anew targeted treatment which could transform the lives of children with brain tumours has been approved for use on the NHS.
The combination of dabrafenib and trametinib can now be used for patients aged one and over with either low-grade or high-grade glioma with the genetic mutation BRAF V600E.
Gliomas are the most common type of brain cancer in children and young people. They develop from the glial cells, which support the nerve cells of the brain and spinal cord.
A BRAF mutation is present in around 15%-20% of low-grade gliomas and around 5%-10% of high-grade gliomas in children.
The new drug therapy has been approved by the National Institute for Health and Care Excellence (Nice) after studies showed it lessened chemotherapy side-effects, improved children’s response rate to treatment, and improved the survival time without the disease getting worse.
Progression-free survival time went from 7.2 months with chemotherapy to 24.9 months under the new regime for those with low-grade glioma, and was nine months for those with high-grade disease.
In some cases, tumours completely disappeared, though longer term follow-up of patients is needed. The new treatment is expected to benefit around 60 children a year.
The combined therapy, which is used in some other illnesses, is less harsh, can be taken in tablet and liquid form, and can be given at home.
The two drugs work together by blocking the growth signal coming from the mutant BRAF protein and can slow or even stop the tumour from growing.
Dr Michele Afif, chief executive of the Brain Tumour Charity, 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.”
Suki Sandhu, whose son Raj Rana died aged eight in 2018, is a trustee at the Brain Tumour Charity and gave evidence to Nice.
Raj had six operations, chemotherapy, immunotherapy and proton beam therapy.
Ms Sandhu said: “The clinical trials on dabrafenib with trametinib show some really favourable results and I know if my son had had the opportunity to go on to such a trial, I would have jumped at it in a heartbeat.”