Treatment To Improve Degenerating Muscle Gains Strength

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Treatment To Improve Degenerating Muscle Gains Strength

http://www.medicalnewstoday.com/articles/170742.php

A study appearing in Science Translational Medicine puts scientists one step

closer to clinical trials to test a gene delivery strategy to improve muscle

mass and function in patients with certain degenerative muscle disorders.

Severe weakness of the quadriceps is a defining feature of several neuromuscular

disorders. Researchers at Nationwide Children's Hospital have shown that a gene

delivery strategy that produces follistatin - a naturally occurring protein that

inhibits myostatin, a growth factor expressed specifically in skeletal muscle -

directly to the quadriceps of non-human primates results in long-term gene

expression with muscle enhancing effects, including larger muscles with greater

strength.

Previously, Nationwide Children's researchers demonstrated follistatin's

therapeutic potential using rodent models. This more recent study produced

similar results in non-human primates, in a translational study to demonstrate

efficacy in safety in a species more closely related to humans. Non-human

primates that received the injection of the follistatin transgene experienced

pronounced and durable increases in muscle size and strength. Muscle growth

occurred for 12 weeks after treatment, after which time the growth rates

appeared to stabilize and were well tolerated, with no adverse events noted over

the course of the 15-month study.

" Our studies indicate that this relatively non-invasive approach could have

long-term effects, involve few risks and could potentially be effective in

various types of degenerative muscle disorders including multiple forms of

muscular dystrophy, " said the study's corresponding author, Kaspar, PhD,

principal investigator in the Center for Gene Therapy of The Research Institute

at Nationwide Children's Hospital.

Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at

Nationwide Children's added, " These findings serve as the basis for testing in

patients and give us confidence in moving forward with our translational program

of follistatin to enhance muscle mass. "

The research team has developed a plan with the Food and Drug Administration and

is currently in the process of performing the formal toxicology and

biodistribution studies to support initiating a human clinical trial.

The potential use of this strategy for muscle strengthening has important

implications for patients with muscle diseases including Duchenne muscular

dystrophy - the most common form of muscular dystrophy - as well as for the

planned first clinical trial for inclusion body myositis. It also may be

applicable to other forms of muscular dystrophy, such as facioscapulohumeral

muscular dystrophy, in which gene replacement or other types of gene

manipulation are not feasible because of the absence of a specific gene defect.

In addition to their roles at Nationwide Children's, Drs. Kaspar and Mendell

hold faculty posts at The Ohio State University College of Medicine.