Research shows promise for potential new gene therapy strategy for muscle-wastin

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Research shows promise for potential new gene therapy strategy for

muscle-wasting diseases

http://www.eurekalert.org/pub_releases/2008-03/nch-rsp031008.php

Investigators in The Research Institute at Nationwide Children's

Hospital have identified the role of a protein that could potentially

lead to new clinical treatments to combat musculoskeletal diseases,

including Duchenne muscular dystrophy (DMD).

Results of these studies appear in the March 11, 2008 issue of the

Proceedings of the National Academy of Sciences.

These studies, led by Kaspar, PhD, a principal investigator in

the Center for Gene Therapy at The Research Institute and an

assistant professor of Pediatrics at The Ohio State University, focus

on a protein called follistatin (FS). Using a single injection, gene-

delivery strategy involving FS, investigators treated the hind leg

muscles of mice. Results showed increased muscle size and strength,

quadruple that of mice treated with proteins other than FS. The

muscle enhancements were shown to be well-tolerated for more than two

years.

According to Dr. Kaspar, increased muscle mass and strength were also

evident when this strategy was tested using a model of DMD. Apart

from the injected hind leg muscles, strengthening effects were also

shown in the triceps. In addition, fibrosis, abnormal formation of

scar tissue and a hallmark of muscular dystrophy, was decreased in FS-

treated animals.

" We believe this new FS strategy may be more powerful than other

strategies due to its additional effects, including its ability to

reduce inflammation, " said Dr. Kaspar.

The strategy showed no negative effects on the heart or reproductive

ability of either males or females. The results were also replicated

in older animals, suggesting that this strategy could be useful in

developing clinical treatments for older DMD patients.

" This research provides evidence of multiple potential treatment

applications for muscle diseases including, but not limited to,

muscular dystrophy, " said Jerry Mendell, MD, director of the Center

for Gene Therapy at The Research Institute, a co-author on the study,

and professor of Pediatrics in Neurology and Pathology at The Ohio

State University. " These results offer promise for treatment of

potentially any muscle-wasting disease, including muscle weakness due

to other illnesses, aging, and inflammatory diseases such as

polymyositis. Our next step is to pursue clinical trials. "

The Research Institute at Nationwide Children's Hospital has a patent

pending on the FS technique due to the major role it may play for

muscular dystrophy treatment and other muscle-wasting diseases.