Researchers at MUSC Hollings Cancer Center are developing a promising new drug to combat aggressive head and neck cancers. The drug works by targeting cancer cells’ mitochondria – their energy source – by boosting levels of a fat molecule called C18-ceramide. C18-ceramide triggers the destruction of mitochondria and starves cancer cells. It also blocks fumarate, a key energy molecule, creating a dual attack on cancer metabolism while largely sparing healthy cells.

In mouse models and patient-derived tumors, the drug significantly slowed tumor growth and caused cancer cells to collapse energetically. Because reduced ceramide is a hallmark of many cancers, this approach could one day lead to a new class of targeted therapies that are safer and more effective than chemotherapy or radiation. Although still in preclinical stages, the researchers are optimistic about advancing the drug to clinical trials as a potential lifeline for patients with treatment-resistant cancers.