Effective treatment of mitochondrial myopathy by nicotinamide riboside, a vitamin B3

EMBO Mol Med. 2014 Jun;6(6):721-31. doi: 10.1002/emmm.201403943.

Abstract

Nutrient availability is the major regulator of life and reproduction, and a complex cellular signaling network has evolved to adapt organisms to fasting. These sensor pathways monitor cellular energy metabolism, especially mitochondrial ATP production and NAD(+)/NADH ratio, as major signals for nutritional state. We hypothesized that these signals would be modified by mitochondrial respiratory chain disease, because of inefficient NADH utilization and ATP production. Oral administration of nicotinamide riboside (NR), a vitamin B3 and NAD(+) precursor, was previously shown to boost NAD(+) levels in mice and to induce mitochondrial biogenesis. Here, we treated mitochondrial myopathy mice with NR. This vitamin effectively delayed early- and late-stage disease progression, by robustly inducing mitochondrial biogenesis in skeletal muscle and brown adipose tissue, preventing mitochondrial ultrastructure abnormalities and mtDNA deletion formation. NR further stimulated mitochondrial unfolded protein response, suggesting its protective role in mitochondrial disease. These results indicate that NR and strategies boosting NAD(+) levels are a promising treatment strategy for mitochondrial myopathy.

Keywords: NAD+; mitochondrial myopathy; nicotinamide riboside; treatment; unfolded protein response.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adipose Tissue, Brown / drug effects
  • Adipose Tissue, Brown / metabolism
  • Adipose Tissue, Brown / pathology
  • Animals
  • Energy Metabolism / drug effects
  • Forkhead Box Protein O1
  • Forkhead Transcription Factors / metabolism
  • Lipid Metabolism / drug effects
  • Liver / drug effects
  • Liver / metabolism
  • Liver / pathology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mitochondria / drug effects*
  • Mitochondria / metabolism
  • Mitochondria / pathology
  • Mitochondrial Myopathies / drug therapy*
  • Mitochondrial Myopathies / metabolism
  • Mitochondrial Myopathies / pathology
  • Muscle, Skeletal / drug effects
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • NAD / metabolism
  • Niacinamide / analogs & derivatives*
  • Niacinamide / therapeutic use
  • Pyridinium Compounds
  • Sirtuin 1 / metabolism
  • Unfolded Protein Response / drug effects
  • Vitamin B Complex / therapeutic use*

Substances

  • Forkhead Box Protein O1
  • Forkhead Transcription Factors
  • Foxo1 protein, mouse
  • Pyridinium Compounds
  • nicotinamide-beta-riboside
  • NAD
  • Vitamin B Complex
  • Niacinamide
  • Sirt1 protein, mouse
  • Sirtuin 1