Families of Boys with Duchenne Muscular Dystrophy Band Together to Fund Research
BALTIMORE, MD – (April 12, 2017) Three grassroots foundations founded by the families of young boys diagnosed with Duchenne muscular dystrophy (DMD) have jointly awarded a $100,000 grant to Kennedy Krieger Institute's Center for Genetic Muscle Disorders for research into novel drug therapies. The funding from Ryan's Quest, of New Jersey, along with Michael's Cause and Pietro's Fight, both of New York, will enable the scientific team to study existing drugs that have shown early therapeutic promise in cell cultures. "We remain dedicated to helping identify existing FDA-approved drugs that can be repurposed to help treat Duchenne muscular dystrophy and ultimately expedite potential therapies for patients," said David Schultz, co-founder of Ryan's Quest and father of 11-year-old Ryan, who has DMD. Researcher Dr. Kathryn Wagner, and her colleagues Gabsang Lee and Congshan Sun, recently screened existing drugs using human myoblasts derived from induced pluripotent stem cells (iPSCs) from individuals living with DMD. The iPSCs are a type of stem cell that can be generated from mature living cells. Several drugs reliably produced a positive therapeutic response in the cells in culture. With this funding, the findings will be validated and trialed in the mouse model for DMD. As many of these drugs are already FDA approved, researchers and families are hopeful that a promising discovery in the mouse model could rapidly translate to clinical trials. "We are so grateful to these families, who in addition to raising three wonderful boys, Ryan, Michael and Pietro, are also working hard with us to develop novel treatments to combat DMD," said Dr. Kathryn Wagner, Director of the Center for Genetic Muscle Disorders at Kennedy Krieger Institute. "Our team is deeply inspired by the grassroots efforts of families across the country who are raising critical dollars to fund research." About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder that causes a loss of motor, pulmonary, and cardiac function, and premature death. It is one of the most common pediatric genetic disorders, affecting every one in 5,000 live male births and some females. DMD is caused by the body's inability to create dystrophin, a large protein found in muscle cells. Children with DMD usually lose their ability to walk around the age of 12 and succumb to the disease in their early to mid-twenties. There is no FDA approved treatment or cure. Ryan's Quest, Michael's Cause and Pietro's Fight are non-profits founded by families of boys and young men with Duchenne muscular dystrophy who believe that their global collaboration is paramount to see potential therapies through the drug pipeline. About the Kennedy Krieger Institute Internationally recognized for improving the lives of children and adolescents with disorders and injuries of the brain, spinal cord and musculoskeletal system, the Kennedy Krieger Institute in Baltimore, MD, serves more than 20,000 individuals each year through inpatient and outpatient clinics, home and community services and school-based programs. Kennedy Krieger provides a wide range of services for children with developmental concerns mild to severe, and is home to a team of investigators who are contributing to the understanding of how disorders develop while pioneering new interventions and earlier diagnosis. For more information on the Kennedy Krieger Institute, visit www.kennedykrieger.org.