Gboxin is a small molecule inhibitor that targets mitochondrial oxidative phosphorylation in certain cancer cells, particularly glioblastoma. It disrupts energy production by inhibiting the function of the F1Fo-ATP synthase complex, leading to reduced ATP levels and cancer cell death. Due to its selective toxicity toward tumor cells with specific metabolic dependencies, Gboxin shows potential as a targeted therapy in preclinical studies for brain cancers. It has demonstrated the ability to cross the blood-brain barrier, which enhances its relevance for treating central nervous system tumors. Current research focuses on optimizing its efficacy and understanding resistance mechanisms.