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

Cells ◽  
2020 ◽  
Vol 9 (12) ◽  
pp. 2657
Author(s):  
Emma Rybalka ◽  
Cara Timpani ◽  
Danielle Debruin ◽  
Ryan Bagaric ◽  
Dean Campelj ◽  
...  

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.

Author(s):  
Emma Rybalka ◽  
Cara Timpani ◽  
Danielle Debruin ◽  
Ryan Bagaric ◽  
Dean Campelj ◽  
...  

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 outcomes 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 has failed to translate into clinical benefit to patients has 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.


Neurology ◽  
1978 ◽  
Vol 28 (12) ◽  
pp. 1224-1224 ◽  
Author(s):  
M. W. Haymond ◽  
K. E. Strobel ◽  
D. C. DeVivo

2019 ◽  
Vol 10 (3) ◽  
pp. 557-573 ◽  
Author(s):  
Peng Zhang ◽  
Jian He ◽  
Fei Wang ◽  
Jing Gong ◽  
Lu Wang ◽  
...  

2015 ◽  
Vol 136 (2) ◽  
pp. 351-362 ◽  
Author(s):  
Jana Zschüntzsch ◽  
Yaxin Zhang ◽  
Florian Klinker ◽  
Gregor Makosch ◽  
Lars Klinge ◽  
...  

Cell ◽  
1997 ◽  
Vol 90 (4) ◽  
pp. 717-727 ◽  
Author(s):  
Anne E Deconinck ◽  
Jill A Rafael ◽  
Judith A Skinner ◽  
Susan C Brown ◽  
Allyson C Potter ◽  
...  

2006 ◽  
Vol 15 (6) ◽  
pp. 989-998 ◽  
Author(s):  
Chun Guo ◽  
Michael Willem ◽  
Alexander Werner ◽  
Gennadij Raivich ◽  
Michael Emerson ◽  
...  

Biomedicines ◽  
2021 ◽  
Vol 9 (12) ◽  
pp. 1911
Author(s):  
Andrea Farini

Duchenne muscular dystrophy (DMD) is the most common, lethal, muscle-wasting disease of childhood [...]


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