CHILDREN with deadly brain tumors could get an 18-month survival boost with the first new NHS drug in almost 20 years.
The drug regulator has approved the gene-targeting drug combination dabrafenib and trametinib for children with glioma brain cancer.
Around 30 seriously ill children could be eligible every year in England.
In trials, those under 18 who received the drugs saw their cancer stop growing for two years – 25 months – compared with seven months for standard chemotherapy.
Some have even seen their tumors disappear completely – although more studies are needed to prove the drugs’ potential as a cure.
It causes fewer side effects and can be taken orally at home rather than at a hospital appointment.
Dr Michele Afif, head of the Brain Tumor Charity, said: “This may only affect a small population, but it is of huge significance for them and their loved ones.
“We hope this is the first of many new treatments that will ensure our community can live longer and better.”
The charity said 2005 was the last time a new medicine was approved by the NHS for children with brain tumours.
“THIS WILL MAKE A REAL DIFFERENCE”
AALIYAH, 12, (pictured above) received the drug combination in a clinical trial at Great Ormond Street Hospital after being diagnosed with a brain tumor at an ophthalmology appointment.
She said: “I managed to start secondary school with my friends and attend practically every class.
“I was also able to stay at home instead of in the hospital for treatment and continue my hobbies, such as being a majorette.
“I’m delighted that other teenagers and children can now have this treatment instead of chemotherapy as it will make a real difference.”
The drug combination will be offered on the NHS to under-18s who have high- or low-grade gliomas in the brain or spinal cord with a mutation in the BRAF gene.
Existing treatments can be brutal
This mutation makes chemotherapy less effective, but the drugs can seek out and destroy the proteins it produces, blocking tumor growth.
Helen Knight, from the National Institute for Health and Care Excellence, said: “Treatment options for glioma brain tumors are limited and we know they can be brutal.
“I am pleased that we can recommend this new combination therapy that can give children more time without tumor growth and offer them and their families a better quality of life.”
Professor Peter Johnson, director of cancer at NHS England, said the drugs are “very effective” and added: “It’s fantastic news that it will now be available on the NHS.”
Gliomas are rare, but they are the most common type of brain cancer in children and young people, affecting around 180 people a year.
Symptoms of brain tumors may include headaches, nausea, seizures, drowsiness or fainting, eye problems, or changes in personality or behavior.
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