Fujii T, Nozaki F, Saito K, Hayashi A, Nishigaki Y, Murayama K, Tanaka M, Koga Y, Hiejima I, Kumada T. Efficacy of pyruvate therapy in patients with mitochondrial disease: a semi-quantitative clinical evaluation study. Mol Genet Metab. 2014 Jun;112(2):133-8. 

Tanaka M, Nishigaki Y, Fuku N, Ibi T, Sahashi K, Koga Y. Therapeutic potential of pyruvate therapy for mitochondrial diseases. Mitochondrion. 2007 Dec;7(6):399-401.

In 2007, Tanaka et al formulated the elegant and exciting theory that pyruvate may be helpful in mitochondrial diseases with an elevated lactate/pyruvate ratio by regenerating NAD+ and thus promoting energy production through glycolysis, by activating pyruvate dehydrogenase and by non-enzymatically removing hydrogen peroxide. In Fujii et al’s recent study, four severely affected patients with disorders of oxidative phosphorylation were treated with sodium pyruvate; three showed some improvement, and the effect was maintained for two years in one. The authors found that blood lactate and lactate/pyruvate ratios were not informative for the purpose of monitoring therapy.

The use of sodium pyruvate as a potential treatment for disorders of oxidative phosphorylation certainly merits further study, perhaps in a placebo-controlled trial with less severely affected patients, in whom treatments effect might be more apparent.

Posted by Alina Levtova, MD