A new approach for treating childhood medulloblastoma

01 March 2013

Professor Geoff Pilkington, University of Portsmouth

Medullobastoma is the most common malignant brain tumour of childhood. Although 70 per cent of patients can be cured, the aggressive treatments leave many survivors with significant mental and physical disabilities. Professor Pilkington is developing a new approach to the treatment of medulloblastoma that could impact on both survival rates and quality of survival for young patients.

Amount of grant: £196,928  |  Date of award: March 2013


Medulloblastoma is the most common malignant brain tumour of childhood. Whilst around 70 per cent of affected children can now be cured, this comes at a high cost as the aggressive nature of the treatment leaves survivors with a poor quality of life, often suffering serious physical and mental disabilities.

The problem is that this type of tumour is complex and currently both good- and bad- prognosis patients are grouped together and receive the same aggressive treatment.

Using cutting edge molecular genetic techniques, Professor Pilkington’s team is unravelling the biology of medulloblastoma. They have identified the genetic signalling pathways that underlie development of the disease.

Medulloblastomas are known to have an accumulation of a protein called GD3. GD3 is normally involved in cell death but, crucially, in medulloblastoma it is modified in such a way that it does not function normally and thus cannot drive the death of aberrant cells.

The team has already shown, in the lab, that reversing this modification results in increased cell death. Their previous gene therapy approach to this work was successful but the gene that was previously used has some associated problems that rendered it unsuitable for progression to clinical trial. They now need to try an alternative approach to take the technique forward to clinical trial.

About the research team

Professor Pilkington has worked in neuro-oncology research for more than 40 years. His Cellular and Molecular Neuro-oncology laboratories at the University of Portsmouth represent the flagship centre for dedicated brain tumour research in the south of England.

Professor Pilkington has assembled a multi-disciplinary and international research team, with years of experience and each bringing different areas of expertise to the project.

Collaborators include Dr Michael Taylor from the Hospital for Sick Children in Toronto, whose laboratory leads the world in medulloblastoma research.

What difference will this project make?

New treatment approaches for medulloblastoma are desperately needed, not only to improve survival rates but, crucially, to reduce the devastating morbidity associated with current treatments.

If successful, the current project will take Professor Pilkington’s new treatment approach a step closer to clinical trial.

Ultimately, this approach could impact significantly on both survival and quality of survival for young patients with medulloblastoma.

Read more: About childhood brain tumours | Other brain tumour research | Brain tumour initiative


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