Researchers have identified a potential approach to stop the growth of the most common type of brain tumor in children.

UNC Lineberger’s Timothy Gerson, MD, PhD, and colleagues reported in the journal Development that by blocking a signal called GSK-3, they could control tumor growth in a subtype of medulloblastoma. Their preclinical findings may provide clues to a possible new targeted treatment strategy.

«This work could lead to new insights into developmental brain malformations and also to new treatments for medulloblastoma that may spare the severe side effects of radiation and typical chemotherapy,» said Gerson, who is an associate professor in the UNC School of Medicine Department of Neurology.

While as many as 80 percent of children with medulloblastoma survive long-term with radiation and chemotherapy, researchers said there is a need to improve therapies in order to limit debilitating side effects from those treatments, as well as to develop treatments that work for children whose cancer don’t respond to treatment.

«For one of the more common subtypes of medulloblastoma, we found we can target a signaling pathway to block tumor growth,» said Jennifer Ocasio, PhD, the study’s first author and a former graduate student in the UNC School of Medicine Neuroscience Curriculum. «By targeting this pathway, we think this would be a way to sidestep effects of radiation and chemotherapy that have a lot of side effects because we are blocking growth rather than killing these cells.»

In laboratory studies using mice and cells, the researchers focused on a tumor subtype that accounts for about one-third of medulloblastoma cases. In this type of brain tumor, the Sonic Hedgehog cellular signal helps trigger a series of signals in developing brain cells that lead to an overgrowth of neurons in the cerebellum, which controls balance, speech and other activities.

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Materials provided by UNC Lineberger Comprehensive Cancer Center. Note: Content may be edited for style and length.