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Myostatin as a therapeutic target in Amyotrophic lateral sclerosis.

Authors :
Walsh FS
Rutkowski JL
Source :
Neurochemistry international [Neurochem Int] 2012 Nov; Vol. 61 (6), pp. 931-5. Date of Electronic Publication: 2012 Jul 26.
Publication Year :
2012

Abstract

Amyotrophic Lateral Sclerosis is a devastating neurological disease that is inevitably fatal after 3-5years duration. Treatment options are minimal and as such new therapeutic modalities are required. In this review, we discuss the role of the myostatin pathway as a modulator of skeletal muscle mass and therapeutic approaches using biological based therapies. Both monoclonal antibodies to myostatin and a soluble receptor decoy to its high affinity receptor have been used in clinical trials of neuromuscular diseases and while there have been efficacy signals with the latter approach there have also been safety issues. Our approach is to target the high affinity receptor-binding site on myostatin and to develop a next generation set of therapeutic reagents built on a novel protein scaffold. This is the natural single domain VNAR found in sharks which is extremely versatile and has the ability to develop products with superior properties compared to existing therapeutics.<br /> (Copyright © 2012 Elsevier Ltd. All rights reserved.)

Details

Language :
English
ISSN :
1872-9754
Volume :
61
Issue :
6
Database :
MEDLINE
Journal :
Neurochemistry international
Publication Type :
Academic Journal
Accession number :
22841860
Full Text :
https://doi.org/10.1016/j.neuint.2012.07.016