title: [Anti-myostatin antibody therapy for myopathies].
abstract: Myostatin, a member of the muscle-specific transforming growth factor (TGF)-β family, negatively regulates skeletal muscle growth. It inhibits muscle stem cell proliferation and differentiation, and attenuates adult muscle fiber protein accretion, resulting in decreased skeletal muscle mass. Thus it has been considered to be a therapeutic target of myopathies including muscular dystrophy. Notably, administration of a blocking antibody against myostatin ameliorated the pathophysiology of dystrophin-deficient mdx mice. Although a clinical trial of an anti-myostatin antibody MYO-029 failed to achieve a significant outcome in patients with muscular dystrophies, various distinct approaches have been taken to establish anti-myostatin therapy including a myostatin decoy receptor ACE-031, a peptide drug derived from myostatin prodomain, small-molecule inhibitors against the myostatin receptor, a follistatin-derived peptibody inhibiting myostatin and myostatin siRNA with collagen-derived carrier particles. Clinical application of the anti-myostatin therapeutics for the treatment of patients with muscular dystrophy needs further evaluation of safety and specification of the target disease types among various muscular dystrophies.
authors.affiliation: Department of Neurology, Kawasaki Medical School.
authors.forename: Yoshihide
authors.initials: Y
authors.lastname: Sunada
mesh_terms: D000818:Animals; D000906:Antibodies / Q000627:therapeutic use; D006801:Humans; D051379:Mice; D009136:Muscular Dystrophies / Q000628:therapy*; D055435:Myostatin / Q000276:immunology*
chemical_list: D000906:Antibodies; D055435:Myostatin; C506829:Stamulumab