Ryan's Quest and Michael's Cause request proposals for top clinical development projects with an emphasis on inclusion of older patients
HAMILTON, N.J. and STATEN ISLAND, N.Y., Aug. 21, 2018 /PRNewswire-iReach/ -- Several gene therapies for Duchenne muscular dystrophy have advanced to clinical trials in recent months, offering great hope to the patient community. However, progress has been mired by manufacturing and safety setbacks, which resulted in clinical holds for two of the companies. Sadly, older patients are excluded from the initial series of gene therapy clinical trials, in part because they generally have more advanced disease which may trigger a more rigorous immune reaction to the viral capsid, the therapeutic gene, or manufacturing contaminants.
This request for proposals will identify top projects that aim to overcome gene therapy safety and manufacturing challenges that are particularly problematic for older patients but also exist in the younger patient population. The foundations are also interested in supporting clinical trials outside of gene therapies that include older patients.
Scientists and clinicians are encouraged to reach out to Robert Capolongo and David Schultz using the two emails below to initiate the application process. Applicants will be required to submit a one-page letter-of-intent and selected projects will be invited to submit a full proposal. Funding is currently available and inquiries are encouraged immediately, although there is no formal deadline. Full details will be provided by email.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a neuromuscular disorder that occurs in approximately 1:3600 males and some females which leads to iterative cycles of muscle degeneration and regeneration resulting in inflammation, fibrosis, progressive decline of motor, respiratory, and cardiac function, and premature death. To date, there are no satisfactory therapies and Duchenne remains a severe unmet need.
About the Foundations
Michael's Cause was founded in 2011 after their son Michael was diagnosed with Duchenne Muscular Dystrophy. Ryan's Quest was founded in 2008 after their son Ryan was diagnosed. The Foundations raise awareness of Duchenne and direct all funding to the best medical and scientific research to find better treatments and a future cure for this disease. The Foundations continue to collaborate with Pietro's Fight, Powers Promise, and many others to support this and other drug development projects. To date, Michael's Cause and Ryan's Quest have directed over $3.9 million dollars towards research.
Contact Information for RFP and Grant Inquiries
President, Michael's Cause
President, Ryan's Quest
$100,000 grant to Kennedy Krieger will make study of novel drug treatments possible
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.