Personalized therapies are not yet a reality for children with medulloblastoma, a highly aggressive form of brain cancer.

Treatment involves invasive surgery, radiation to the whole brain and spinal cord, and high doses of chemotherapy. Not only does this approach not cure all patients – the survival rate is 60% – those that do survive face lifelong side effects of radiation on the developing brain, including intellectual disability, growth issues, early strokes, and hearing loss.

Future Leader, Dr. Vijay Ramaswamy and his research team have identified a strategy to reduce the amount of treatment children need to get better, specifically those with recurrent, high-risk medulloblastoma tumours.

One of the reasons this subset of tumors is so hard to treat effectively is because they’re resistant to radiation. A mutation in a gene called p53 makes the tumour cells evade death in response to the damage that radiation causes and as a result, the tumour cells persist.

“We wouldn’t have been able to figure this without support from Brain Canada.” 

Dr. Vijay Ramaswamy, Alvin Segal Family Foundation Future Leader in Canadian Brain Research

Dr. Ramaswamy and his team showed that blocking the DNA repair system in these cells made the radiation more effective at killing them.

With an additional $1.3 million grant from the Canadian Institutes of Health Research, Dr. Ramaswamy and his team are now testing this approach in mice using radiation combined with targeted drugs being developed to block the DNA repair system.  

Alexandria DeCarlo, a student in Dr. Vijay Ramaswamy’s lab at The Hospital for Sick Children. Credit: The Hospital for Sick Children

What’s the impact? 

Treatment for medulloblastoma has remained unchanged since the 1980s; it’s invasive, it’s toxic, and given that most of these tumours occur in young children, it brings heartbreaking, lifelong side-effects.

In the last decade, scientists have identified four distinct types of medulloblastoma tumours, each with unique growth mechanisms and clinical outcomes.

Dr. Ramaswamy and his research team focused on the most high-risk, recurring tumour types, given the pressing need for new ways to treat them. He and his team identified an Achilles heel in these tumours to target therapeutically, which promises to both increase the efficacy of treatment and reduce the long-term toxicity for this hard-to-treat tumour. 

“The grant allowed us to generate the results we needed to leverage more funding and explore a whole new direction for my lab. Thanks to Brain Canada, we were able to do something really innovative.” 

Dr. Vijay Ramaswamy
Dr. Vijay Ramaswamy (middle) and his team at The Hospital for Sick Children. Credit: The Hospital for Sick Children

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