RegeneRx, CNMC explore treatment for degenerative muscle disease

BETHESDA, Md.—RegeneRx Biophar-maceuticals reported in early August that it had begun a collaboration with the Division of Cardiology at Children's National Medical Center (CNMC) in Washington, D.C., to study thymosin beta 4 (TB4). The goal is to explore TB4's ability to treat degenerative muscle diseases that often result in cardiomyopathy—a progressive deterioration and weakening of the heart.

 

More than 84,000 patients with degenerative muscular dystrophy diseases exist in the United States currently, with about 50,000 of them having cardiomyopathy. Cardiomyopathy can lead to arrhythmia, heart failure and sudden death, and for most patients, transplantation of a new heart is the only viable treatment. RegeneRx hopes to change that situation with TB4.

 

Dr. Christopher Spurney, assistant professor of pediatrics at CNMC, is leading the project, which will study the effects of TB4 in non-human models that have a disease similar to human Duchenne muscular dystrophy and go on to develop cardiomyopathy.

 

"This collaboration resulted originally from an article published in Nature last November showing that our proprietary product was effective in preventing functional and pathological damage to the myocardium in mouse studies," notes J.J. Finkelstein, president and chief executive officer of RegeneRx. "That gained a lot of attention, and for some time already we had discussed with CNMC how we might collaborate since (we are) both in the metro D.C. area. This seemed like the logical project for us to pursue together."

 

The study published in Nature involved a series of 60 mice at the University of Texas Southwestern Medical Center, in which myocardial infarction was induced. A third of the mice were given a placebo, another third received intracardiac administration of TB4 and the remainder received TB4 through a tail vein. In the two active groups that received TB4, there was statistically significant reduction of scarring of the heart and statistically significant increase in heart function, Finkelstein says.

 

"This is a very exciting study as it may relate to previously published work showing TB4's ability to protect and repair the heart after an acute myocardial infarction," Spurney says. "We look forward to testing it in our models to see if we can produce similar results. If TB4 is shown to be protective, we would hope to move into human clinical trials."