First new brain tumour drug for 20 years could give children an extra 18 months with family
CHILDREN with deadly brain tumours could get an 18-month survival boost with the first new NHS drug for almost 20 years.
The medicines watchdog has approved the gene-targeting drug combination of dabrafenib and trametinib for children with glioma brain cancers.
Around 30 critically ill children could be eligible each year in England.
In trials, under-18s who received the drugs saw their cancer stop growing for two years – 25 months – compared to seven months for standard chemotherapy.
Some have even seen their tumours disappear completely – although more studies are needed to prove the drugs’ potential as a cure.
It causes fewer side effects and can be taken by mouth at home instead of on a hospital visit.
Dr Michele Afif, chief of the Brain Tumour Charity, said: “This may only affect a small population but it’s of huge significance to them and their loved ones.
“We hope that this will be the first of many new treatments that will ensure our community can live longer and better lives.”
The charity said 2005 was the last time a new drug was approved for the NHS for children with brain tumours.
AALIYAH, 12, (pictured above) has been receiving the drug combination on a clinical trial at Great Ormond Street Hospital after being diagnosed with a brain tumour in an eye appointment.
She said: “I’ve been able to start secondary school with my friends and go to pretty much all my lessons.
“I’ve also been able to be at home, rather than staying in hospital for treatment, and carry on my hobbies such as majorette.
“I’m really pleased that other teenagers and children will now be able to have this treatment instead of chemotherapy, it will make a real difference.”
The drug combo will be offered on the NHS to under-18s who have high- or low-grade gliomas in their brain or spinal cord with a BRAF gene mutation.
Existing treatments can be brutal
This mutation makes chemotherapy less effective, but the medications can seek and destroy the proteins it makes, blocking the tumour’s growth.
Helen Knight, at the National Institute of Health and Care Excellence, said: “Treatment options for glioma brain tumours 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 offer them and their families a better quality of life.”
Professor Peter Johnson, NHS England’s cancer director, said the drugs are “very effective” and added: “It is fantastic news that this will now be available on the NHS.”
Gliomas are rare but the most common type of brain cancer in children and young people, affecting around 180 per year.
Symptoms of brain tumours can include headaches, sickness, seizures, drowsiness or fainting, problems with the eyes, or personality or behaviour changes.