Gboxin is a small molecule inhibitor that targets mitochondrial oxidative phosphorylation in certain cancer cells, particularly glioblastoma. It disrupts energy production by inhibiting the F1F0-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 an anticancer agent in preclinical studies. It has been investigated for its ability to cross the blood-brain barrier, making it a candidate for treating brain tumors. Current research focuses on optimizing its efficacy and understanding resistance mechanisms.