Long-term administration of pirfenidone improves cardiac function in mdx mice

Muscle Nerve. 2006 Sep;34(3):327-34. doi: 10.1002/mus.20590.

Abstract

Duchenne muscular dystrophy, an X-linked recessive neuromuscular disorder due to lack of the protein dystrophin, manifests as progressive muscle degeneration and cardiomyopathy with increased fibrosis. The exact mechanisms involved in fibrosis are unknown, but a cytokine, transforming growth factor-beta (TGF-beta), is a likely mediator. This study tested whether the TGF-beta antagonist, pirfenidone, could reduce cardiac fibrosis. Eight-month-old mdx mice were treated for 7 months with 0.4%, 0.8%, and 1.2% pirfenidone in drinking water; untreated water was given to control mdx and C57 mice. Mice treated with 0.8% and 1.2% pirfendone had lowered cardiac TGF-beta mRNA and improved in vitro cardiac contractility (P < 0.05) to levels consistent with C57 mice, yet without a change in cardiac stiffness or fibrosis. These results show that the TGF-beta antagonist, pirfenidone, can improve cardiac function in mdx mice, potentially providing a new avenue for developing cardiac therapies for patients with Duchenne muscular dystrophy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Anti-Inflammatory Agents, Non-Steroidal / pharmacology*
  • Cardiomyopathies / drug therapy*
  • Cardiomyopathies / etiology
  • Cardiomyopathies / pathology
  • Disease Models, Animal
  • Fibrosis
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred mdx
  • Muscular Dystrophy, Animal / complications
  • Muscular Dystrophy, Animal / drug therapy*
  • Muscular Dystrophy, Animal / pathology
  • Muscular Dystrophy, Duchenne / complications
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Muscular Dystrophy, Duchenne / pathology
  • Myocardium / pathology
  • Pyridones / pharmacology*
  • RNA, Messenger / analysis
  • Transforming Growth Factor beta / genetics
  • Ventricular Dysfunction, Left / drug therapy
  • Ventricular Dysfunction, Left / etiology
  • Ventricular Dysfunction, Left / pathology

Substances

  • Anti-Inflammatory Agents, Non-Steroidal
  • Pyridones
  • RNA, Messenger
  • Transforming Growth Factor beta
  • pirfenidone