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Many people think that brain tumours are exceptionally rare – yet around 16,000 patients a year in the UK are diagnosed with one, and one in three of us knows somebody affected, according to charity Brain Tumour Research. Incidence rates are rising, and brain tumours remain the biggest cancer killer of children and young adults under the age of 40. Yet, despite these bleak facts, only one per cent of the national spend on cancer research has historically been allocated to the disease.
A step forward
It made a welcome change this spring to read positive news, when it was announced in April that the National Institute for Health and Care Excellence (NICE) had recommended a groundbreaking new targeted drug combination treatment for children with an aggressive form of brain cancer. In the coming months, dabrafenib with trametinib will be available on the NHS to young patients aged one to 17 with gliomas – the most common type of brain tumour in children – with a specific mutation (BRAF V600E). Both made by Novartis, dabrafenib (also known as Finlee) will be given as tablets while trametinib (also known as Spexotras) is an oral solution.
“This new targeted treatment certainly brings hope for children who are diagnosed with this type of tumour,” says Dr Karen Noble, director of research, policy and innovation at Brain Tumour Research. “Currently, fewer than 30 per cent of children diagnosed with high-grade gliomas survive beyond five years. While the survival rate for low-grade gliomas is higher at 90 per cent, children undergo gruelling treatment, such as chemotherapy, which has harsh and lasting side effects,” she explains. “Trials in the US showed that this combination of drugs kept the tumour at bay without progression for around two years, which is a significant increase on the current average. What’s more, it lessened chemotherapy side effects and improved children’s response rate to treatment. So, although this is not a cure, this new treatment is less harsh and can be given at home – both factors which could significantly improve children’s quality of life.”
While the number of young patients who stand to benefit is relatively low (according to NICE, it’s estimated that around 30 children in England could be eligible), it’s nonetheless a significant step forward, heralded as the first new treatment for paediatric brain tumours in decades.
It may also yet pave the way for future developments. “Studies investigating dabrafenib in combination with trametinib involved patients aged up to 17 years,” says Dr Noble. “There is some evidence that the new treatment could have an effect in adults, but a full adult trial would be needed.”
Dabrafenib with trametinib will become available in the coming months for children aged one to 17.
Difficult to diagnose
More advances in brain tumour treatments cannot come quickly enough for patients, given that only 12 per cent will survive beyond five years of their diagnosis. The exact cause of primary brain cancer is still unclear, although it’s an area that scientists continue to explore. Recently, a major study of 250,000 mobile phone users – led by researchers at Imperial College London and Sweden’s Karolinska Institutet, published this spring in Environment International – found no link between long-term mobile use and an increased risk of brain tumours. What is known is that the disease is an incredibly complex one – with more than 100 different tumour types – and can present with a wide range
of symptoms.
“Brain tumours are notoriously difficult to diagnose because a patient’s symptoms are often very non-specific,” says Dr Noble. “The three most common symptoms are seizures (which can range from slight tingling in one hand or a short break in speech or awareness to full-blown seizures), focal symptoms (which can include forgetfulness or difficulty finding words), and physical symptoms (such as worsening coordination and headaches). Many of these symptoms can be attributed to other conditions or simply missed.”
As a result, continues Dr Noble, more than a quarter of patients are not diagnosed with a brain tumour until they are taken to A&E, unfortunately often when symptoms have increased in severity. However, by being aware of common red flags and the importance of early diagnosis, pharmacy teams are well-placed to spot potential warning signs.
“Due to the extra training given to pharmacy staff and the fact that it is now easier for patients or families to talk to a pharmacist long enough for a problem to be recognised, this is increasingly becoming a recognisable pathway for brain tumour referral,” says Dr Noble.
“Brain tumours remain the biggest cancer killer of children and young adults under the age of 40”
A forgotten type of cancer
For years, campaigners have complained that funding for brain tumour research has fallen behind other cancers. A 2023 inquiry report by the All-Party Parliamentary Group on Brain Tumours (APPGBT) highlighted that since records began in 2002, National Cancer Research Institute (NCRI) partners had funded £10 billion of cancer research, however, only £126 million of this had been spent on brain tumours, compared with £775 million on breast cancer and £551 million on leukaemia.
In 2016, a petition calling on the Government to fund more research into brain tumours – which my family started, having lost my brother Stephen to one in 2014 – topped 120,000 signatures. This prompted a lively debate at Westminster and was followed by the formation of a task and finish working group on brain tumour research. In 2018, after the group had published its report, the Government pledged £40 million over five years for research into brain cancer. But in 2023, the APPGBT’s report revealed that just £15 million had been awarded – with £6 million of this “not easily identifiable as relevant to brain tumours”.
“I was shocked to hear that £25 million had still not made it into the hands of researchers,” says my father Peter Realf, who sat as the sole patient carer representative on the task and finish group. “For too long, brain tumours have been a forgotten cancer and the Government must urgently do more for patients and their families.
“I believe that the will is there – among politicians, health professionals and charities – but something has to change, because the current system is too complicated and too slow. And unfortunately, given that 88 per cent of brain tumour patients will not survive more than five years, they simply don’t have the time to wait.”
“Ultimately one of the biggest challenges to finding a cure is the continued need for greater levels of investment into researching this uniquely complex disease,” adds Dr Noble. “Any ambition to take new discoveries from the scientist’s bench to the patient’s bedside is fundamentally underpinned by appropriate funding for discovery science and early-stage translational research – like that funded by Brain Tumour Research – which, in time, will deliver huge improvements in patient survival. But charities cannot, and should not, have to do it alone. The Government needs to declare brain tumours a critical priority and appropriately invest in research in order to deliver new treatments and, ultimately, cures.”
Hopeful breakthroughs
Though the search for a cure continues, there are some rays of hope on the horizon. “We’ve waited a long time for new drug developments, so the recent NICE announcement is good news,” says Peter. “I hope that this will encourage further research in this area.”
Meanwhile, Dr Noble says that four Brain Tumour Research Centres of Excellence are undertaking some exciting projects which could help to transform the future of brain tumour diagnoses and treatments.
“There has been encouraging news out of our Centres in recent months, including the findings of a clinical study which took place at our Imperial College London Centre, which could see a blood test help diagnose glial tumours, including glioblastoma (GBM), in the future,” says
Dr Noble. “Not only could this speed up the diagnosis of a brain tumour, but it could also eliminate the need for surgical biopsies which carry significant risk.
“At our Centre at the University of Plymouth, the team has used fruit fly models to uncover the ‘readying’ process which occurs just prior to brain tumour onset which could be vital for tumour growth – a discovery which could open up new avenues of research to find potential drug targets towards novel therapies for patients with glioma tumours.
“And they are also launching a clinical trial taking forward research into repurposing drugs used to treat HIV and AIDS for treating low-grade brain tumours.”
For Peter, each step forward is something to be celebrated. “Every trial, every breakthrough, gives patients something vitally important – hope,” he says. “It breaks my heart that there was no treatment that was able to save my son. But I hope that in the years to come, with more funding, we will find a cure.”
Find out more
- For further information on brain tumours, visit: braintumourresearch.org
- Read the NICE announcement: nice.org.uk/news/article/children-and-teenagers-with-an-aggressive-form-of-brain-cancer-set-to-benefit-after-nice-recommends-new-life-extending-drug-combination-treatment
- Take a look at the study by Imperial College London and Sweden’s Karolinska Institutet: news.ki.se/mobile-phone-users-who-talk-for-a-long-time-do-not-have-an-increased-risk-of-brain-tumours
- See The All-Party Parliamentary Group report on Brain Tumours: braintumourresearch.org/pages/campaigning-appg-on-brain-tumours.
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