MitoQ is an antioxidant. Its active ingredient is ubiquinone, as found in coenzyme Q10 and idebenone. However, the ubiquinone in MitoQ is attached to a positively charged, lipophilic molecule called TPP (triphenyl phosphonium), which allows it to selectively accumulate in mitochondria. This makes it more potent than untargeted ubiquinone analogs at protecting cells in culture from oxidative stress
Oxidative stress is believed to play a major role in ALS pathogenesis1. Unfortunately, trials of antioxidants have thus far failed to show any benefits in patients with ALS2 3. One explanation for these failures is that the antioxidants did not reach the optimal place. Since mitochondria are both the main producer and main target of oxidative stress, mitochondrial-focused antioxidant therapy might be more successful. Orally administered MitoQ can alter markers of both oxidative stress and mitochondrial dysfunction in cell culture4 and animal model of ALS (11).
“This study supports the role of mitochondrial dysfunction in the development and progression of ALS, which may allow for the development of more mitochondria-targeted therapies to fight this disease,” concluded study’s co-author and Director of the Center, Dr. Rafael Radi. “We also found that MitoQ has beneficial effects in the murine model of ALS, which will likely lead to clinical trials using MitoQ with ALS patients and hopefully lead to extend the survival and improve the quality of life of ALS patients.”
2015 - An Overview of Potential Targets for Treating Amyotrophic Lateral Sclerosis and Huntington’s Disease
MitoQ is a mitochondrial antioxidant that contains the antioxidant Quinone linked to a lipophilic triphenylphosphonium cation. Researchers showed that MitoQ prolonged life span and the pathology of SOD-knockout Drosophila melanogaster, the enzyme involved in ALS. In addition, the compound has been shown to exert neuroprotective effects in SODG93A mice, slowing functional decline, decreasing oxidative damage and disease progression, and increasing survival.
Furthermore, other studies showed that MitoQ preincubation prevented the cell death observed in cultures of motor neurons + SOD-mutant astrocytes alone.
MitoQ has a promising mechanism, positive preclinical data from two different ALS models, and appears reasonably safe and inexpensive, especially at doses of 10 mg daily. Available anecdotal data are insufficient to determine how helpful this might be in PALS. A small open-label pilot trial with validated ALS diagnoses and outcomes appears warranted.
Kaufmann P, Thompson J, Buchsbaum R, Shefner J, Krivickas L, Katz J, et al.Phase II trial of coq10 for ALS finds insufficient evidence to justify phase III. Ann Neurol. 2009;66:234–44. Crossref, Web of Science ®, Google Scholar ↩︎
Graf M, Ecker D, Horowski R, Kramer B, Riederer P, Gerlach M, et al.High dose vitamin E therapy in amyotrophic lateral sclerosis as add-on therapy to riluzole: results of a placebo controlled double-blind study. J Neural Transm. 2005;112:646–60. Crossref, Web of Science ®, Google Scholar ↩︎