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An oral drug combination by Novartis showed promise in treating a subgroup of patients suffering from a common childhood brain cancer in a trial. In the mid-stage trial, 47 per cent of the patients that were given the two drugs Tafinlar and Mekinist saw their tumours shrink, far above a rate of 11 per cent in a comparative group of participants on standard chemotherapy, the drugmaker said on Monday, June 6. The participants, aged one to 17 years, were suffering from low-grade gliomas (LGG), the most common childhood brain cancer. The trial only included those who were found to have a mutation known as BRAF V600, a genetic contributor in about 15 per cent to 20 per cent of paediatric LGG cases. Among further results of the trial with 110 participants, the median time without disease progression was 20.1 months for those given the Novartis drug combo, compared to 7.4 months on chemotherapy. The new oral treatment candidate also caused less severe side effects than burdensome chemotherapy.

After receiving approval from the National Institute for Health and Care Excellence (NICE), the NHS is preparing to roll out a new combination treatment for brain tumours in children and young people in England. Dabrafenib with trametinib would be the first ever targeted treatment for children (aged 1-17) with gliomas that have a specific genetic mutation, the health service said. The treatment, which can be taken at home, has been found to slow the progression of the disease by over threefold compared to standard chemotherapy in children with low-grade gliomas that have a BRAF V600E mutation. Clinical trials have shown that it causes fewer side-effects than chemotherapy. NICE approved the combination treatment on Wednesday 24 April. It will be available on the NHS in the coming months.