MDA awarded a grant totaling $310,893 to Howard Worman, professor of medicine and pathology, and cell biology at Columbia University Medical Center in New York, for research into treatments that target the underlying cellular disease process responsible for heart damage, or "cardiomyopathy," in people with Emery-Dreifuss muscular dystrophy (EDMD).
In previous research supported by MDA, Worman's group showed that inhibiting proteins in the MAP kinase signaling pathway improved heart function in a mouse model of EDMD.
"The 'first-generation' inhibitors that we used are not suitable for humans," Worman said. "In the current research, we will study drugs of the same classes that have already been given to humans to determine if they similarly improve heart function in these mice."
The group plans to administer the new inhibitors and then examine the mice using some of the same methods doctors use to examine the heart in humans, including echocardiography (a type of imaging study used to assess the heart's pumping ability). The investigators will extend their findings to other EDMD mouse models known to develop cardiomyopathy, and then ascertain whether administration of MAP kinase signaling inhibitors causes toxicity in these mice.
Positive results generated by Worman's research could pave the way for clinical trials of MAP kinase signaling inhibitors to treat humans with EDMD.
"MDA has supported our research on Emery-Dreifuss muscular dystrophy for more than 10 years," Worman said. "This most recent grant will allow us to translate these past 10 years of basic laboratory research into a treatment for human patients with muscular dystrophy."
Funding for this MDA grant began August 1, 2010.