The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle

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Rybalka, Emma ORCID: 0000-0002-4854-0036, Timpani, Cara ORCID: 0000-0003-4567-4319, Debruin, Danielle A, Bagaric, RM ORCID: 0000-0002-2286-2870, Campelj, Dean G and Hayes, Alan ORCID: 0000-0003-1398-3694 (2020) The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle. Cells, 9 (12). ISSN 2073-4409

Abstract

Myostatin inhibition therapy has held much promise for the treatment of muscle wasting disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy (DMD). Following on from promising pre-clinical data in dystrophin-deficient mice and dogs, several clinical trials were initiated in DMD patients using different modality myostatin inhibition therapies. All failed to show modification of disease course as dictated by the primary and secondary outcome measures selected: the myostatin inhibition story, thus far, is a failed clinical story. These trials have recently been extensively reviewed and reasons why pre-clinical data collected in animal models have failed to translate into clinical benefit to patients have been purported. However, the biological mechanisms underlying translational failure need to be examined to ensure future myostatin inhibitor development endeavors do not meet with the same fate. Here, we explore the biology which could explain the failed translation of myostatin inhibitors in the treatment of DMD.

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Item type Article
URI https://vuir.vu.edu.au/id/eprint/42097
DOI https://doi.org/10.3390/cells9122657
Official URL https://www.mdpi.com/2073-4409/9/12/2657
Subjects Historical > FOR Classification > 0601 Biochemistry and Cell Biology
Current > Division/Research > Institute for Health and Sport
Current > Division/Research > College of Health and Biomedicine
Keywords myostatin, regulator of muscle stem “satellite” cell proliferation, cell differentiation, dystrophin protein expression, dystrophic muscles
Citations in Scopus 7 - View on Scopus
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