Treatment to improve degenerating muscle gains strength

Nov 11, 2009

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 strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in - directly to the quadriceps of non-human primates results in long-term 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 ," said the study's corresponding author, Brian 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 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 ."

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 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.

Source: Nationwide Children's Hospital (news : web)

Explore further: Ebola mistakes should serve a lesson says WHO

add to favorites email to friend print save as pdf

Related Stories

'Mighty mice' made mightier

Aug 29, 2007

The Johns Hopkins scientist who first showed that the absence of the protein myostatin leads to oversized muscles in mice and men has now found a second protein, follistatin, whose overproduction in mice lacking ...

Scientist clears hurdles for muscular dystrophy therapy

Oct 29, 2008

Approximately 250,000 people in the United States have some form of muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common type of the disease, predominantly affecting males. Boys with DMD will lose the ...

Recommended for you

Ebola mistakes should serve a lesson says WHO

16 hours ago

The World Health Organization's chief admitted on Sunday that the UN agency had been caught napping on Ebola, saying it should serve as a lesson to avoid similar mistakes in future.

British Ebola nurse discharged from hospital

23 hours ago

A British nurse who contracted Ebola while working as a volunteer in Sierra Leone said she was "happy to be alive" as she was discharged from hospital on Saturday having made a full recovery.

Tide turning in Ebola fight after hard lessons

Jan 24, 2015

A top U.N. official in the fight against Ebola greeted just three patients at one treatment center he visited this week in Sierra Leone. Families in Liberia are no longer required to cremate the remains of ...

Just five Ebola cases left in Liberia: UN

Jan 24, 2015

The United Nations said on Saturday Liberia was dealing with just five remaining cases of Ebola, in the clearest sign yet that the country is nearing the end of the outbreak.

User comments : 0

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.