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Researchers Discover Promising Immunotherapy Combination for Medulloblastoma and Other Pediatric Brain Cancer Types


One in four children does not survive medulloblastoma, and although standard treatments can cure some patients, those who survive often live with devastating long-term side effects. The research community with support from the Pediatric Brain Tumor Foundation and other nonprofits has been working diligently to further immunotherapy as a safer and more effective treatment for pediatric brain tumors like medulloblastoma.

Researchers at Sanford Burnham Prebys have discovered that combining immunotherapy with the tumor necrosis factor (TNF) protein can eliminate medulloblastoma brain tumors in mice. The discovery, published in Nature Neuroscience, is expected to lead to a clinical trial to test the benefits of the treatment in pediatric patients. The findings also hold promise for other pediatric brain cancer types including diffuse intrinsic pontine glioma (DIPG).

“I’ve studied medulloblastoma for more than 20 years. I’ve seen many therapies that prolong survival in mice. But this is the first time I have ever seen a therapy essentially melt the tumor away,” says Robert Wechsler-Reya, PhD, senior author of the paper. Wechsler-Reya is a professor and director of the Tumor Initiation and Maintenance Program at Sanford Burnham Prebys, program director of the Joseph Clayes III Research Center for Neuro-Oncology and Genomics at the Rady Children’s Institute for Genomic Medicine, and member of the Pediatric Brain Tumor Foundation’s Research Advisory Network.

“We look forward to testing this approach in the clinic and are hopeful that this discovery might be able to save children’s lives.”
The work of study authors Dr. Robert Wechsler-Reya, Dr. Oren Becher, Northwestern University, and Dr. Michael Taylor, Hospital for Sick Children, was supported in part by the Pediatric Brain Tumor Foundation.

Learn more about this promising breakthrough in Sanford Burnham Prebys’ press release.