A regenerative medicine project at Wake Forest University is one of 40 initiatives sharing in $13.7 million in new Muscular Dystrophy Association funding for research on neuromuscular diseases.
Martin Childers, a professor at the Institute for Regenerative Medicine in the Wake Forest School of Medicine, has been awarded $480,000 over three years to research heart diseases associated with two muscular dystrophy diseases caused by mutations in the dystrophin gene. Duchenne muscular dystrophy, or DMD, is caused by a lack of dystrophin protein in skeletal and cardiac muscles. Becker muscular dystrophy, or BMD, is caused by partially functional dystrophin protein.
Many DMD patients die as young adults from heart failure due to heart damage caused by the genetic mutation, Childers said in a statement. Childers’s team will use two new technologies to address the disease. First they’ll create heart cells from the skin cells of DMD patients, a method called cellular reprogramming. Then they will use a drug discovery platform to screen compounds in individual cells.
“By marrying these two incredible technologies, this project will allow, for the first time, the testing of new drugs directly on the heart cells of an individual patient without any risk to the patient,” Childers said.
If the research is successful, it could become yet another technology to spin out of Wake Forest as a new company. Tengion (NASDAQ:TNGN), which is developing a tissue-generating platform technology, was formed in 2005 based on the research of Dr. Tony Atala, director of the Institute for Regenerative Medicine.
Childers’s project is one of 13 targeting DMD supported by the MDA in this round of grants. The MDA has also awarded grants to nine projects researching amyotrophic lateral sclerosis, or ALS, as well as projects researching spinal muscular atrophy, facioscapulohumeral muscular dystrophy and the link between diabetes and Friedreich’s ataxia.
