Leading charity welcomes NICE approval for therapy for children with rare brain tumours
The Brain Tumour Charity says it’s ‘delighted’ that a new targeted therapy, for some childhood brain tumours, which is kinder and more effective than current treatments, has been approved for NHS usage by NICE.
Following a review of its cost and clinical effectiveness, a combination of the drugs dabrafenib and trametinib are being recommended, by NICE, for patients aged one and above who have been diagnosed with glioma – a certain type of brain tumour.
These diagnoses can include diffuse midline glioma, formerly known as DIPG, astrocytoma or ependymoma – which also has a genetic mutation called BRAF V600E.
Typically, less than 30% of children diagnosed with high grade gliomas will survive for five years or more. Outcomes for low grade gliomas, the most common childhood brain tumour, are better – at a 90% survival rate following surgery.
The current standard of care is radiotherapy and chemotherapy. These aren’t always effective for these types of tumours and can have brutal side effects like hair loss, nausea, bruising and low blood count.
The new treatment is taken in tablet and liquid form so can be administered at home with few side effects.
The combination medication is the first targeted treatment to be made available on the NHS for paediatric gliomas. It is already licensed for use in the USA, and for use in the UK in adults with the BRAF mutation who have some types of lung and skin cancers.
The two drugs work together to block the growth signal coming from the mutant BRAF protein, slowing or even stopping the tumour from growing.
‘First new treatment for paediatric brain tumours in decades’
‘We are delighted that NICE has approved the first new treatment for paediatric brain tumours in decades,’ says Dr Michele Afif, Chief Executive at The Brain Tumour Charity.
‘Though this will only affect a small population, it’s of huge significance to them and their loved ones and represents real progress.’
‘We hope that this will be the first of many new treatments that will ensure our community can live longer and better lives.’
Brain tumours affect around 500 children a year in the UK, of whom between 150 to 175 may have a glioma.
Estimates vary, but the BRAF mutation is present in 15 to 20 per cent of paediatric low grade, and between five to seven per cent of high grade gliomas, in children.
Suki Sandhu lost her son Raj to a high grade glioma in 2018 when he was eight, but she’s delighted the treatment could now help other children.
Suki, a trustee at The Brain Tumour Charity, and a consultee who gave evidence to the NICE Technical Appraisal on behalf of patients, said Raj was diagnosed when he was two.
His treatment included six operations, chemotherapy, immunotherapy and proton beam therapy in the USA.
Clinical trials: show ‘some really favourable results’
‘I am so pleased NICE has approved the use of these new drugs,’ says Suki. ‘Many years ago, I had to make the decision to stop chemotherapy treatment for my son as he was suffering with horrible side effects after years of harsh treatment.’
‘It was one of the hardest decisions I have ever had to make as it was a clear sign we were running out of options, and it was likely I would lose him.’
‘The clinical trials on dabrafenib with trametinib show some really favourable results and I know if my son had had the opportunity to go onto such a trial I would have jumped at it in a heartbeat.’
‘We need kinder drugs and new treatments for all the brain tumour community. I am hopeful other families will go on to get access to these drugs and, hopefully, remain disease-free for longer and live good quality lives.’
Aaliyah, 12, from Ipswich, joined the clinical trial in 2020. This was 12 months after being put on ‘watch and wait’ for an inoperable brain tumour that was picked up following a routine eye test.
‘Three months after we started the trial, we had an MRI,’ says Aaliyah’s mum, Amie.
‘That was the first time I felt able to look at the scans. When I saw the before and after, that was probably the day a massive weight was lifted off my shoulders. I didn’t expect it to shrink that much, I knew we’d made the right decision.’
Aaliyah says she’s grateful she’s been able to have treatment at home.
Starting secondary school with friends
‘I’m able to just take tablets twice a day and go to the hospital every few months, rather than be in hospital to have chemotherapy.’
‘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 do feel proud in a way that I’ve been part of a trial that has helped other people, and I am thankful to the team who have supported me.’
‘I’m really pleased that other teenagers and children will now be able to have the tablets instead of chemotherapy, without needing to be part of a trial. That’s a pretty big thing to have been part of and will make a real difference.’
‘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,’ says Helen Knight, director of medicines evaluation at The National Institute for Health and Care Excellence (NICE).
‘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 within the last three months.’
‘We are humbled to be part of an international scientific community effort in developing targeted therapies based on the unique genetic features of a patient’s tumour and welcome the NICE decision to make the treatment available for patients in England and Wales,’ says Marie Andrée Gamache, Country President at manufacturers Novartis UK and Ireland.
‘This recent appraisal is an example of how we can address the biggest clinical and public health challenges in the UK through collaboration and make the progress that’s needed for patients to benefit most.’
‘We are working with the Scottish Medicines Consortium to provide equitable access to the treatment across the UK.’
The Tumour Marker
In 2008, research carried out by Dr David Jones which was funded by The Brain Tumour Charity, then known as the Samantha Dickson Brain Tumour Trust, identified a BRAF mutation that was common in pilocytic astrocytoma brain tumours.
This paved the way for drug development as it was the first time that a specific genetic change had been detected in these tumour types.
It could then become a target for new treatments and diagnostic tests, changing the research landscape for low grade brain tumours in children.
Dr Jones now leads the EVEREST Centre in Germany, which carries out research into paediatric low grade brain tumours and is funded by The Brain Tumour Charity.
NICE’s decision was influenced by the results of the TADPOLE-G study, published in the New England Journal of Medicine and the Journal of Clinical Oncology.
This showed the clinical benefits of a combination of the medications in BRAF mutated paediatric gliomas.
Seeing treatments becoming available to patients in England and Wales
‘Having been involved in the original study that identified the role of BRAF in cancer and the clinical trials that led to the NICE approval of targeted therapies for childhood brain tumours with BRAF gene mutations, it is exciting to see these treatments becoming available to patients in England and Wales,’ says Professor Darren Hargrave, clinical professor in paediatric neuro-oncology at Great Ormond Street Hospital.
‘The new combination therapy is an important advancement in the field of paediatric neuro-oncology that offers an alternative to chemotherapy for low-grade gliomas and provides an additional treatment option for relapsed high-grade gliomas, where overall response rates to the current therapy options have been as low as 20% or less.’
The Brain Tumour Charity is currently funding more of Professor Hargraves work and is the UK’s largest dedicated brain tumour charity, committed to fighting brain tumours on all fronts.
It invests in pioneering research in the UK and internationally to increase survival and improve treatment options and raises awareness of the symptoms of brain tumours to bring about earlier diagnosis.
It also offers a comprehensive support and information service for anyone who is affected by a brain tumour diagnosis, including a support and information line, fact sheets, online peer-to-peer support and a dedicated Children and Families Service.
Open letter calling for National Brain Tumour Strategy
The Brain Tumour Charity recently delivered its Open Letter, signed by more than 52,000 people, to Health Ministers at all four UK Governments.
It calls for the disease to be prioritised in a National Brain Tumour Strategy to enable speedier diagnoses, optimal aftercare and increased investment in research into brain tumours.