Projects we fund

Overview

Medulloblastoma is the most common malignant brain tumour of childhood. Whilst around 70% 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.

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. About childhood brain tumours Brain tumour initiative