Gboxin

Gboxin is an experimental small molecule oxidative phosphorylation (OXPHOS) inhibitor that specifically targets glioblastoma (GBM). It functions by inhibiting mitochondrial ATP synthase, thereby disrupting the energy metabolism of cancer cells that are uniquely dependent on OXPHOS. The molecule's mechanism of action is dependent on its positive charge, which allows it to accumulate within the negatively charged mitochondrial matrix of tumor cells. While Gboxin shows potent anti-proliferative activity against GBM and small cell lung cancer (SCLC) cell lines in vitro, it is characterized by metabolic instability and weak anti-tumor effects in vivo. This has led to the development of more stable analogs, such as S-Gboxin and Y9, to improve therapeutic outcomes in various malignancies, including non-small cell lung cancer (NSCLC).