Featured Research

from universities, journals, and other organizations

New Potential Treatment For Muscular Dystrophy Appears To Be Safe

Date:
March 13, 2008
Source:
Blackwell Publishing Ltd.
Summary:
Myostatin, a protein that blocks muscle growth, has shown promising results as a potential therapeutic target for treating muscular dystrophy in animal studies, where its inhibition led to increased muscle mass and strength. A new study, the first to evaluate a myostatin inhibitor in patients, assessed its safety in adults with muscular dystrophy and found that it was well-tolerated.

Myostatin, a protein that blocks muscle growth, has shown promising results as a potential therapeutic target for treating muscular dystrophy in animal studies, where its inhibition led to increased muscle mass and strength. A new study, the first to evaluate a myostatin inhibitor in patients, assessed its safety in adults with muscular dystrophy and found that it was well-tolerated. 

Related Articles


Muscular dystrophy is an inherited disorder involving progressive muscle weakness and wasting. Although there has been progress in understanding the pathology of this disease, no drug treatments that increase muscle strength have been found. In addition, very few trials have been conducted for muscular dystrophy that begins in adulthood, and none of these involved novel drugs.

A double-blind, randomized study of 116 patients with muscular dystrophy was conducted by researchers from 10 centers in the United States and United Kingdom. Patients with multiple different types of muscular dystrophy were divided into four groups given sequentially higher doses of a myostatin inhibitor called MYO-029 produced by Wyeth Pharmaceuticals. Each group was randomized to receive the test drug or a placebo in a 3:1 ratio. The drug or placebo was administered intravenously every two weeks for six months, after which the patients were followed for three months. Although the purpose of the study was to test for safety, muscle strength and mass were also assessed.

The results showed that safety assessments, including vital signs, laboratory tests and physical examination showed no significant differences between treatment and placebo groups. There were no side effects to skeletal, smooth or cardiac muscle, and the most significant side effects related to the treatment were hypersensitivity skin reactions (such as hives).

No increase in muscle strength or improvement in function was seen during the nine months of the study, although muscle mass did increase in some of the patients. Because the sample sizes in the different dosage groups were small, differences between the groups did not reach statistical significance. “However, the consistency of the response to treatment in the various measures of effects on muscle tissue suggests that MYO-029 reached its intended target, producing a modest degree of muscle fiber hypertrophy and increased muscle mass in some treated subjects,” noted Kathryn R. Wagner of The Johns Hopkins University School of Medicine and her coauthors. They add that larger studies over longer periods of time would be necessary to properly evaluate the efficacy of this new treatment.

“This trial supports the hypothesis that systemic administration of myostatin inhibitors provides an adequate safety margin for clinical studies, and these inhibitors should be evaluated for stimulating muscle growth in muscular dystrophy,” the authors conclude. They note that additional muscle inhibitors are in development and clinical trials for other muscle-wasting conditions, and further evaluation of more powerful myostatin inhibitors for muscle disorders should be considered.

Journal reference: “A Phase I/II Trial of MYO-029 in Adult Subjects with Muscular Dystrophy,” Kathryn R. Wagner, James L. Fleckenstein, Anthony A. Amato, Richard J. Barohn, Katharine Bushby, Diana M. Escolar, Kevin M. Flanigan, Alan Pestronk, Rabi Tawil, Gil I. Wolfe, Michelle Eagle, Julaine M. Florence, Wendy M. King, Shree Pandya, Volker Straub, Paul Juneau, Kathleen Meyers, Cristina Csimma, Tracey Araujo, Robert Allen, Stephanie A. Parsons, John M. Wozney, Edward R. La Vallie, Jerry R. Mendell, Annals of Neurology, March 11, 2008.


Story Source:

The above story is based on materials provided by Blackwell Publishing Ltd.. Note: Materials may be edited for content and length.


Cite This Page:

Blackwell Publishing Ltd.. "New Potential Treatment For Muscular Dystrophy Appears To Be Safe." ScienceDaily. ScienceDaily, 13 March 2008. <www.sciencedaily.com/releases/2008/03/080311142307.htm>.
Blackwell Publishing Ltd.. (2008, March 13). New Potential Treatment For Muscular Dystrophy Appears To Be Safe. ScienceDaily. Retrieved November 28, 2014 from www.sciencedaily.com/releases/2008/03/080311142307.htm
Blackwell Publishing Ltd.. "New Potential Treatment For Muscular Dystrophy Appears To Be Safe." ScienceDaily. www.sciencedaily.com/releases/2008/03/080311142307.htm (accessed November 28, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Friday, November 28, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Rural India's Low-Cost Sanitary Pad Revolution

Rural India's Low-Cost Sanitary Pad Revolution

AFP (Nov. 28, 2014) — One man hopes his invention -– a machine that produces cheap sanitary pads –- will help empower Indian women. Duration: 01:51 Video provided by AFP
Powered by NewsLook.com
Research on Bats Could Help Develop Drugs Against Ebola

Research on Bats Could Help Develop Drugs Against Ebola

AFP (Nov. 28, 2014) — In Africa's only biosafety level 4 laboratory, scientists have been carrying out experiments on bats to understand how virus like Ebola are being transmitted, and how some of them resist to it. Duration: 01:18 Video provided by AFP
Powered by NewsLook.com
Ebola Leaves Orphans Alone in Sierra Leone

Ebola Leaves Orphans Alone in Sierra Leone

AFP (Nov. 27, 2014) — The Ebola epidemic sweeping Sierra Leone is having a profound effect on the country's children, many of whom have been left without any family members to support them. Duration: 01:02 Video provided by AFP
Powered by NewsLook.com
Experimental Ebola Vaccine Shows Promise In Human Trial

Experimental Ebola Vaccine Shows Promise In Human Trial

Newsy (Nov. 27, 2014) — A recent test of a prototype Ebola vaccine generated an immune response to the disease in subjects. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
 
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:  

Breaking News:

Strange & Offbeat Stories

 

Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:  

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile iPhone Android Web
Follow Facebook Twitter Google+
Subscribe RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins