A December 2011 podcast from Nationwide Children's Hospital in Columbus, Ohio, explores how an inhibitor of a protein called NF-kappa B has been beneficial in a mouse model of Duchenne muscular dystrophy (DMD) and is now being developed as a potential DMD treatment.
The podcast is part of a Nationwide Children's series called "This Month in Muscular Dystrophy."
Denis Guttridge, an associate professor at Ohio State University Medical Center and Nationwide Children's whose research focuses on the NF-kappa B family of proteins, is interviewed in this 17-minute podcast by Kevin Flanigan, a neurologist and researcher at Nationwide. Both Guttridge and Flanigan have received MDA research support.
NF-kappa B is involved in the inflammatory response in the immune system. It's found in immune system cells called macrophages, which play a key role in muscle damage in DMD.
Guttridge's group has found that, in addition to being found in macrophages, NF-kappa B is activated in muscle fibers in DMD mice and in boys with DMD, and that it appears to be interfering with the ability of the fibers to regenerate.
Guttridge and colleagues are working on a compound called NBD, a peptide (protein fragment) that can block NF-kappa B, at least in mice.
Early experiments showed DMD mice given NBD showed lessening of muscle inflammation and improved muscle degeneration.
Guttridge and colleagues are planning to test NBD in DMD dogs, which are closer to humans in size and in disease symptoms than mice are. If all goes well, they hope to develop it into a drug that can be tested in human trials in DMD.
"We're going to be doing a lot of testing in the next year or two to make sure the drug is safe," Guttridge said, emphasizing that the U.S. Food and Drug Administration requires a high level of safety demonstration before a compound can move from the laboratory to patients.
Editor's note: This story was amended 12/19/11 to reflect the fact that the podcast series at Nationwide is called "This Month in Muscular Dystrophy."