Problem Protein Isolated In Muscular Dystrophy

Main Category: Muscular Dystrophy / ALS
Article Date: 21 Aug 2007 - 1:00 PDT

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Scientists may have just taken an important step in the fight against muscular dystrophy. According to the Journal of Clinical Investigation, researchers at Ohio State University Medical Center have isolated a protein that could play a major role in crippling MD patients.* It is a finding that could someday lead to new ways to treat some forms of MD, and offer new hope for the one million Americans who have it.**

Having dealt with muscular dystrophy nearly his entire life, Brent Yetter decided to make a career of it. He works in a lab dedicated to fighting these types of diseases.

"It's personally fulfilling to know that every day you're coming in and helping a lot of kids and adults out there that are going through the same things you are," says Yetter.

Brent has Duchenne muscular dystrophy - the most common and severe form of the disease. And while he works at this lab at Columbus Children's Hospital, just five miles away, researchers in another lab may have isolated a protein causing some of Brent's problems.

Denis Guttridge, PhD, of Ohio State University Medical Center says the protein called NFkB has been known to cause inflammation in the muscles, but it may be doing much more than that.

"What we think is happening is that NFkB, when it's active, is actually putting the brakes on muscle so that it cannot regenerate," says Guttridge.

Normally, any time they're used, some muscle cells die. They quickly rebuild themselves to become stronger. But in patients like Brent, that doesn't happen, and it appears this protein is partially to blame. That lead Guttridge to ask the question…

"If our protein is not there, do we see a difference in the disease pathology? And the answer to that was 'yes,'" says Guttridge.

In other words, if NFkB is altered, or removed altogether, the muscle may be able to repair itself. That's the idea behind the next set of experiments that will hopefully lead to new drugs to help people with MD.

There are some 60 different types of muscular dystrophy,** but Duchenne is the most common. Doctors hope that by targeting this particular protein, it may someday help with other forms of the disease too.

*"Interplay of IKK/NFkB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy", Journal of Clinical Investigation, Volume 117 Number 4, April 2007 pp889-901
http://www.jci.org

**"Basic Information about MDA", Muscular Dystrophy Association, http://www.mdausa.org