Low levels of myostatin may impair muscular atrophy and dystrophy treatments

In a paper published in Nature Communications, researchers from University College London and colleagues showed that the myostatin (MSTN; GDF8) pathway is downregulated in muscle wasting or atrophying diseases, providing a potential explanation for why anti-myostatin therapies have been ineffective in clinical trials.

Skeletal muscle mass is partially controlled by the myostatin pathway. Previous research has associated mutations in the myostatin gene with muscle hypertrophy in animals and humans, leading companies to develop myostatin-targeting candidates to treat muscle wasting or atrophying diseases. According to BioCentury's BCIQ database, there are at least eight anti-myostatin therapies in clinical or preclinical development for muscular dystrophy or atrophy. However, the paper's authors write that clinical trials of myostatin inhibitors have failed to show significant improvements in muscle strength or physical function...