Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to improve cardiac care and believe in the promising work Dr. Guttridge and his team at Ohio State are doing.
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Almost all Duchenne patients develop heart problems. In order to treat the disease, new drugs will have to work by fixing both skeletal and heart muscles. In contrast to Duchenne skeletal muscles, very little is known about a dystrophic heart.
Dr. Guttridge is focusing on a protein called NF-kappaB (NF-kB), a signaling pathway which he has shown to contribute to the degeneration of skeletal muscles. Dr. Guttridge now wants to understand how the NF-kB pathway contributes to the degeneration of heart muscle in Duchenne. By using a compound that inhibits NF-kB called NBD, which is capable of rescuing a dysfunctional heart, NF-kB’s role in cardiomyopathy in Duchenne is reduced. Dr. Guttridge explains what this grant will provide:
“Thanks to the generosity of PPMD, who has a long history in pursuing cardiac interventions, we will have the resources to pursue our study of NF-kB – specifically how NF-kB is capable of causing Duchenne hearts to fail. Obtaining this information will provide additional insight into how NF-kB functions in Duchenne, and we believe potential ways to target this molecule to improve the benefits of future Duchenne therapies.”
Abby Bronson, Senior Vice President of Research Strategy for PPMD, explains the importance of this project:
“In almost every incidence of Duchenne that we see, cardiac issues are a significant problem for patients. What Dr. Guttridge and his team are doing is anticipating the challenges future therapies may face in treating these issues of the heart. To end Duchenne, we will have to outsmart the disease progression. Dr. Guttridge is doing just that, by understanding the role NF-kB plays in heart failure. We are so grateful to the work being done at institutes like The Ohio State University.”
Heart issues don't just affect some people with Duchenne; they affect all people with Duchenne. Dilated cardiomyopathy and heart failure remain the leading cause of death in Duchenne. Just as there are variations in onset of skeletal muscle difficulties, there are variations in the onset of cardiac muscle difficulties. Eventually, nearly 100% of all people with Duchenne develop dilated cardiomyopathy and heart failure.
For the past several years, PPMD has heavily invested in the pursuit of optimal care, interventions, and research pertaining to the management and prevention of cardiomyopathy. To learn more about PPMD’s extensive research portfolio, please visit ParentProjectmd.org.