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Developing a new treatment for high-risk brain cancer in children

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Every year in the UK, more than 400 children are diagnosed with a brain or spinal cord tumour.[1] A child with brain cancer may need extremely intensive treatment that can cause severe side effects. But sadly, it doesn’t work for everyone and the disease can come back. Professor John Anderson and Dr Laura Donovan of the UCL Great Ormond Street Institute of Child Health are developing a new treatment for children with certain high-risk brain tumours, which harnesses the power of their immune system to fight the disease. They hope this immunotherapy will be kinder than existing treatments and help save more children’s lives.

This project is jointly funded by Action Medical Research and LifeArc.

How are children’s lives affected now?

Medulloblastoma and high-grade glioma are types of childhood brain cancer that are often very difficult to treat. These tumours are often aggressive and spread into surrounding tissue, making it difficult to completely remove them surgically – and they are also often highly resistant to chemotherapy and radiotherapy.

“Many children with these brain tumours will face prolonged and gruelling treatment with surgery, chemotherapy or radiotherapy,” says Professor Anderson. “While these treatments can be life-saving, many will be left with long-term side effects that can seriously impact their quality of life.”

Unfortunately, the outlook for a child with high-risk brain cancer – which either does not respond well to treatment or comes back after treatment – is very poor.

“Sadly, many children with high-risk brain cancer will lose their lives at a young age,” says Professor Anderson. “There is an urgent need for gentler, more effective treatments that can offer hope to children and their families.”

How could this research help?

“We’re aiming to develop a cutting-edge new immunotherapy for children with high-risk medulloblastoma and high-grade glioma,” says Dr Donovan.

The new treatment – called chimeric antigen receptor (CAR) T cell therapy – has already proven effective for treating certain types of blood cancer. It involves collecting specific immune cells (called T cells) from a child’s blood, genetically modifying them in the laboratory, and putting them back into their body to fight the disease.

Previous funding from Action has helped us to develop CAR T cell therapy for children with another type of solid tumour called neuroblastoma – and we have run a clinical trial to evaluate the approach, with encouraging results,” says Dr Donovan.

The team now plans to engineer an improved version of CAR-T cells in the laboratory that are better at recognising and destroying brain cancer cells while avoiding healthy cells, and reducing the risk of side effects of the treatment.

“Ultimately, we hope this research will lead to a safe and effective treatment for children with these high-risk brain tumours – helping to save more lives and sparing children from life-changing side effects."

Dr Laura Donovan

Research table

Project details

Project Leader Professor John Anderson, BA MBBS MRCP PhD
Location Cancer Section, Developmental Biology and Cancer Programme, UCL Great Ormond Street Institute of Child Health
Project Team Dr Laura Donovan, BSc PhD
Grant Amount £246,014
Start Date
End Date
Duration 36 months
Grant Code (GN number) GN2927


  1. Children with Cancer UK. Spinal and brain tumours in children:  [website accessed 10 October 2022]


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