Idebenone: Clinical Potential Beyond Neurological Diseases

Idebenone, a short-chain analog of coenzyme Q10 with a hydroxydecyl side chain, is known to activate mitochondrial function by transferring electrons to the electron transport chain complex III, thereby promoting adenosine triphosphate production. Numerous clinical trials have demonstrated the effectiveness of idebenone in the treatment of neurological diseases. Interestingly, emerging evidence suggests that idebenone may also have beneficial effects beyond neurological conditions through disrupting mitochondrial membrane potential, inducing mitochondrial apoptosis, promoting mitophagy attenuating ferroptosis, reducing reactive oxygen species and lipid peroxidation, etc. This study aims to comprehensively review the clinical potential of idebenone in various fields, including cancers (such as breast cancer, melanoma, glioblastoma, neuroblastoma, hepatocellular carcinoma, prostatic carcinoma and pancreatic carcinoma), cardiovascular diseases (including atherosclerosis, hypertension, myocardial infarction and heart failure), diabetes mellitus, liver diseases, urogenital diseases, sepsis, and other diseases. The findings highlight the potential of idebenone as a promising therapeutic option for the prevention and management of these condition, which need to be validated in more clinical trials.