A soluble activin type IIB receptor improves function in a mouse model of amyotrophic lateral sclerosis

Exp Neurol. 2009 Jun;217(2):258-68. doi: 10.1016/j.expneurol.2009.02.017. Epub 2009 Mar 11.

Abstract

Amyotrophic lateral sclerosis (ALS) is a neurologic disease characterized by progressive weakness that results in death within a few years of onset by respiratory failure. Myostatin is a member of the TGF-beta superfamily that is predominantly expressed in muscle and acts as a negative regulator of muscle growth. Attenuating myostatin has previously been shown to produce increased muscle mass and strength in normal and disease animal models. In this study, a mouse model of ALS (SOD1(G93A) transgenic mice) was treated with a soluble activin receptor, type IIB (ActRIIB.mFc) which is a putative endogenous signaling receptor for myostatin in addition to other ligands of the TGF-beta superfamily. ActRIIB.mFc treatment produces a delay in the onset of weakness, an increase in body weight and grip strength, and an enlargement of muscle size whether initiated pre-symptomatically or after symptom onset. Treatment with ActRIIB.mFc did not increase survival or neuromuscular junction innervation in SOD1(G93A) transgenic mice. Pharmacologic treatment with ActRIIB.mFc was superior in all measurements to genetic deletion of myostatin in SOD1(G93A) transgenic mice. The improved function of SOD1(G93A) transgenic mice following treatment with ActRIIB.mFc is encouraging for the development of TGF-beta pathway inhibitors to increase muscle strength in patients with ALS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Activin Receptors, Type II / metabolism
  • Activin Receptors, Type II / therapeutic use*
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / metabolism
  • Amyotrophic Lateral Sclerosis / physiopathology
  • Animals
  • Body Weight / drug effects
  • Body Weight / physiology
  • CHO Cells
  • Cricetinae
  • Cricetulus
  • Disease Models, Animal
  • Female
  • Male
  • Mice
  • Mice, Transgenic
  • Muscle Strength / drug effects
  • Muscle Strength / physiology
  • Muscle Weakness / drug therapy*
  • Muscle Weakness / etiology
  • Muscle Weakness / physiopathology
  • Muscle, Skeletal / drug effects
  • Muscle, Skeletal / metabolism
  • Myostatin / antagonists & inhibitors*
  • Myostatin / metabolism
  • Recombinant Fusion Proteins / chemical synthesis
  • Recombinant Fusion Proteins / therapeutic use
  • Superoxide Dismutase / genetics
  • Superoxide Dismutase-1
  • Treatment Outcome

Substances

  • Mstn protein, mouse
  • Myostatin
  • Recombinant Fusion Proteins
  • Sod1 protein, mouse
  • Superoxide Dismutase
  • Superoxide Dismutase-1
  • Activin Receptors, Type II
  • activin receptor type II-B