A new study from Johns Hopkins Kimmel Cancer Center suggests that blocking fructose metabolism could boost the immune system's ability to fight group 3 medulloblastoma, a deadly childhood brain cancer. Published in Acta Neuropathologica Communications, the research conducted in mice indicates that disrupting how tumor cells generate energy may slow disease progression and enhance response to immunotherapy.
Group 3 medulloblastoma is the most aggressive subtype of the disease, with a five-year survival rate below 50% even with intensive treatment. Standard therapies including surgery, radiation, and chemotherapy often cause long-term side effects in young patients. The Johns Hopkins team focused on the metabolic vulnerabilities of these tumors, specifically their reliance on fructose for energy production.
"Tumor cells have a unique metabolism that differs from normal cells," said Dr. Barbara Slusher, director of the Johns Hopkins Drug Discovery program and co-author of the study. "By targeting fructose metabolism, we may be able to starve the cancer cells while leaving healthy cells unaffected." The researchers used a compound that inhibits the enzyme ketohexokinase, which is involved in fructose breakdown. In mouse models, the treatment reduced tumor growth and increased infiltration of cancer-fighting immune cells.
The findings come as other companies explore metabolic approaches to brain cancer. CNS Pharmaceuticals Inc. (NASDAQ: CNSP) is developing treatments for glioblastoma, another aggressive brain tumor. While the Johns Hopkins research is still in preclinical stages, it offers hope for a new strategy against medulloblastoma. The team plans to further investigate the combination of metabolic inhibitors with existing immunotherapies.
"This could be a game-changer for children with this devastating disease," said Dr. Eric Raabe, a pediatric oncologist at Johns Hopkins and study co-author. "We are excited about the potential to translate these findings into clinical trials." The study was supported by the National Institutes of Health and the Maryland Department of Health's Cigarette Restitution Fund.
For more information on the research, visit the Kimmel Cancer Center. The full study is available in Acta Neuropathologica Communications.


