Researchers at the Johns Hopkins Kimmel Cancer Center have identified a potential new strategy for treating group 3 medulloblastoma, a highly aggressive and difficult-to-treat form of childhood brain cancer. In experiments with mice, the team found that disrupting the way tumor cells metabolize fructose can enhance the immune system's ability to fight the disease, according to a study published in Acta Neuropathologica Communications.
Medulloblastoma is the most common malignant brain tumor in children, and group 3 subtype has the poorest prognosis, with a five-year survival rate of only about 50-60%. Current treatments, including surgery, radiation, and chemotherapy, often cause severe long-term side effects. The Johns Hopkins study offers a novel approach by targeting the unique metabolic dependencies of these cancer cells.
The research focused on the role of fructose metabolism in tumor growth. Cancer cells often rely on altered metabolic pathways to fuel their rapid proliferation, a phenomenon known as the Warburg effect. The team discovered that group 3 medulloblastoma cells express high levels of enzymes involved in fructose metabolism, such as ketohexokinase (KHK). By blocking KHK or reducing fructose availability, the researchers were able to slow tumor growth in mouse models.
Importantly, inhibiting fructose metabolism also appeared to boost the immune response against the tumors. The treatment increased the infiltration of T cells and other immune cells into the tumor microenvironment, suggesting that metabolic targeting could synergize with immunotherapies. This dual effect—directly starving cancer cells while activating the immune system—could lead to more effective and less toxic treatments for children with this devastating disease.
The findings come as other companies, such as CNS Pharmaceuticals Inc. (NASDAQ: CNSP), are also developing next-generation therapies for glioblastoma and other brain cancers, highlighting the growing interest in metabolic approaches to oncology. While the Johns Hopkins study is still at the preclinical stage, it opens the door to future clinical trials that could test fructose metabolism inhibitors in combination with existing treatments.
Further research is needed to understand how this approach can be translated to human patients, but the results provide a promising lead for a cancer that has seen few therapeutic advances in decades. The study was supported by the National Institutes of Health and other funding sources, underscoring the importance of continued investment in pediatric cancer research.
