New UK research targets the deadliest form of childhood cancer

• 420 children are diagnosed with brain or central nervous system (CNS) tumours every year in the UK.
  
• Brain/CNS tumours are the second most common type of childhood cancer.
  
• 220 children die from cancer annually in the UK. Brain/CNS tumours are the leading cause of childhood cancer deaths (ages 0–14).

With Brain Tumour Awareness Month starting today, Children with Cancer UK is highlighting the urgent statistics on the deadliest form of childhood cancer and announcing two newly funded research projects aimed at delivering more effective treatments and improving the long-term wellbeing of young brain tumour patients.

420 young people are diagnosed each year, with many facing lifechanging side effects because of current therapies. Scientists are advancing pioneering projects, funded by Children with Cancer UK, to improve survival and protect long-term quality of life.

The experiences of families like Alfie’s, who was diagnosed with medulloblastoma at nine, underline just how critical these developments could be.

The two new brain tumour research projects funded by Children with Cancer UK are:

Targeting metastatic medulloblastoma – led by Dr Laura Donovan, UCL Great Ormond Street Institute of Child Health, London.

Dr Laura Donovan’s team is investigating why medulloblastoma, the most common malignant brain tumour in children, becomes significantly more dangerous when it spreads.

Using advanced models and cell-tracking techniques, researchers aim to identify the small population of tumour cells responsible for metastasis. Understanding these mechanisms will help inform the development of next generation immunotherapies designed to prevent tumour spread and reduce long term side effects.

Early MRI predictors of quality of life in paediatric ependymoma (EPiQ) – led by Professor Shivaram Avula, Alder Hey Children’s Hospital NHS Foundation Trust, Liverpool.

Ependymomas arise from cells that line the cerebrospinal fluid spaces of the brain and spine. This project analyses early MRI scans before and after treatment to identify which children may be at greater risk of long-term cognitive or functional difficulties.

The research aims to provide clinicians with earlier indicators so they can tailor support and rehabilitation, helping to preserve children’s quality of life during treatment and recovery.

Dr Laura Donovan said:

Paediatric brain tumours remain some of the most complex and challenging cancers we treat. Continued research is essential if we are to change outcomes for children. The developments emerging across the field are encouraging, with a growing emphasis on safer, more targeted therapies that improve both survival and long term wellbeing.

Alfie’s story

In September 2019, nine-year-old Alfie from Wolverhampton was diagnosed with medulloblastoma after weeks of headaches, vomiting and fatigue. He underwent major surgery, six weeks of radiotherapy and eight months of chemotherapy.

Following surgery, Alfie developed posterior fossa syndrome, a condition affecting movement and speech, resulting in him having to learn to walk and talk again. This is just one of the many late effects harsh cancer treatments can cause, and why it is important we continue to invest into kinder treatments to allow children like Alfie to enjoy life after their cancer diagnosis.

Despite the challenges, Alfie was known for his humour and kindness, lifting spirits on the ward through small gifts and jokes. His family continues to support others affected by childhood cancer and is working with Children with Cancer UK throughout Brain Tumour Awareness Month.

Kerry, Alfie’s mum, said:

Nothing can prepare you for hearing the words ‘your child has a brain tumour’. Watching Alfie go through surgery, radiotherapy and months of chemotherapy was heartbreaking, yet he still found the strength to bring joy to others.

Last December, Alfie celebrated his 16th birthday. Five years after completing treatment, the aggressive therapies that saved his life have also left him with lifelong disabilities. As a result of the late effects of his brain tumour, he continues to experience learning challenges at school. Despite this, Alfie is thriving. He is preparing to begin a supported animal care college course and volunteering his time to help other families facing childhood cancer.

Kerry added:

Alfie has overcome so many challenges. His resilience, kindness and determination inspire everyone around him. Watching him endure such aggressive treatment showed us how urgently children need better options. Research gives families hope.

Alfie said:

There were some really hard days, but I focused on staying positive and helping others. If my experience can help improve treatment for children in the future, that means a lot.

Gavin Maggs, Chief Executive of Children with Cancer UK, said: 

Brain tumours continue to have the most devastating impact on young lives. This is why investing in high quality research is absolutely essential. The projects we’re funding are not just advancing scientific knowledge, they have the potential to fundamentally change how these cancers are diagnosed and treated, offering children safer and more effective options than ever before. With the public’s support this March, we can accelerate breakthroughs so that more children survive and live well after treatment.

Children with Cancer UK have released a video about brain tumours in children.

To support lifechanging research and family services, visit www.childrenwithcancer.org.uk