Families of Boys with DMD Donate $100,000 to Research New Therapies

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Diagnosis, treatment and current research on heart failure in Duchenne muscular dystrophy

Three grassroots foundations started by the families of boys with Duchenne muscular dystrophy (DMD) have come together to award a $100,000 research grant to the Kennedy Krieger Institute’s Center for Genetic Muscle Disorders to study potential therapies.

The Kennedy Krieger Institute in Baltimore provides services for children with development concerns or injuries of the bran, spinal cord and musculoskeletal system. The institute is home to a team of researchers who are collaborating to understand how disorders develop, while also pioneering novel interventions and approaches to diagnosis.

The grant from the non-profits Ryan’s QuestMichael’s Cause and Pietro’s Fight will fund the scientific team’s efforts to study pre-existing drugs that have demonstrated therapeutic potential in cell cultures. These organizations believe global collaboration is necessary to advance potential therapies through pipeline development.

“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,” Kathryn Wagner, MD, PhD, director of the Center for Genetic Muscle Disorders, said in a press release. “Our team is deeply inspired by the grassroots efforts of families across the country who are raising critical dollars to fund research.”

Wagner and colleagues Gabsang Lee, PhD, and Congshan Sun, PhD, recently have explored the therapeutic potential of a series of pre-existing drugs, using human myoblasts (future muscle cells) derived from induced pluripotent stem cells (iPSCs), a type of stem cell that can be generated from mature living cells, from DMD patients.

Several of these screened drugs produced a reliable, positive therapeutic response in cell cultures. This funding will support the validation of these findings and the eventual advance into trial stage with mouse models of DMD.

Many of these drugs already have been approved by the U.S. Food and Drug Administration (FDA), so both research teams and families are hopeful that a promising discovery at the mouse model level could hasten the pace to human trials.

According to the National Center for Advancing Translational Sciences, drug repurposing stems from discoveries about the molecular basis of a disease. This opens unexplored opportunities to translate findings into new medicines. In addition, many agents approved for repurposing already have been tested in humans, guaranteeing their safety and pharmacological profiles.

New candidates developed under drug repurposing programs should be ready for trials faster than drugs developed from the start, and should be able to be integrated into healthcare sooner, too.

“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 DMD patient Ryan.