Study offers childhood brain tumour treatment hope

In collaboration with the Press Association

Results from a new study suggests that pilocytic astrocytoma - the most common type of brain tumour seen in children - may be driven by a specific set of gene faults. The discovery could pave the way for new treatments for the disease.

Researchers funded by the Samantha Dickson Brain Tumour Trust, Cancer Research UK and ALSAC found that every one of the pilocytic astrocytomas tested contained either a fault in a gene called KRAS, or a situation where the BRAF or RAF1 genes had become attached to other genes.

All three gene faults have previously been linked to cancer, and there are currently drugs in development that target the molecular pathways they are involved in. The scientists have raised the possibility of using such drugs to help treat children with pilocytic astrocytoma.


Childhood brain tumours are the leading cause of cancer-related deaths in children as existing treatments are not always effective. While surgery remains an option many tumours are inoperable due to their proximity to critical brain structures.

Study leader Professor David Ellison, of St Jude Children's Research Hospital in Memphis, believes this research, published in the Journal of Pathology, has important implications for treatment.

"Our more detailed molecular understanding of pilocytic astrocytoma will also be useful in diagnosis, enabling pathologists to use genetic tests to distinguish among different types of childhood brain tumours to guide better treatment decisions," he commented.

Dr Lesley Walker, Cancer Research UK's director of cancer information, said: "It's often more difficult to treat brain tumours successfully because of the sensitive position of the tumour. This research adds to our understanding of the cancer pathways that cause these tumours to form, and we think this study will be vital in guiding future research into targeted treatments for the disease."

References Activation of the ERK/MAPK pathway: a signature genetic defect in posterior fossa pilocytic astrocytomas. J Pathol. (2009), DOI: 10.1002/path.2558. Sheer D. et al.