AMES, Iowa — Ryan’s Quest and Michael’s Cause, two nonprofit organizations, have provided a $150,000 grant to Iowa State University to study a combination of compounds that could be an effective therapy for children suffering from Duchenne muscular dystrophy.
Ryan's Quest, based in New Jersey, and Michael’s Cause, based in New York City, are dedicated to finding treatments and a cure for Duchenne muscular dystrophy. The organizations’ funding will support the research of Joshua Selsby, an associate professor of animal science.
Duchenne muscular dystrophy is the most common lethal genetic disorder diagnosed during childhood, affecting mostly boys and young men. It is a progressive muscle-wasting disorder that causes loss of motor, pulmonary and cardiac function and premature death.
Selsby, whose research is conducted with mice, has been studying the effects of different combinations of nutraceuticals — foods or dietary supplements with potential health or medical benefits — as therapies that decrease the severity of the disease. One key area of research is with quercetin, a compound among a group of plant pigments called flavonoids that give many fruits, flowers and vegetables their colors.
In initial research supported by the Duchenne Alliance, including Ryan’s Quest and Michael’s Cause, Selsby found that long-term enrichment of diets with quercetin temporarily improved the function of skeletal muscles of mice afflicted with Duchenne. His research, in collaboration with John Quindry of University of Montana, also found that quercetin may have a bigger, longer lasting impact on protecting heart muscle.
“We think quercetin effectively pushed the accelerator, so to speak, in delivering a therapeutic effect, but that dystrophic muscle didn’t have enough gas to keep the process moving,” said Selsby.
Based on those results, Ryan’s Quest and Michael’s Cause have provided the new funding so Selsby can investigate how to overcome this limitation, using another compound called nicotinamide riboside, a trace nutrient found in foods.
Recently published studies have shown promise using nicotinamide riboside in mouse models, raising the possibility that when used in combination with quercetin, it will provide needed “fuel” to keep a positive impact occurring and produce a greater result than either compound used by itself, Selsby said.
Selsby also will test quercetin and nicotinamide riboside in combination with a steroid and a blood pressure medication that many boys diagnosed with Duchenne are taking.
“This will be a comprehensive study done in combination with scientists at the University of Montana,” Selsby said. “For 12 months, we will monitor respiratory and cardiac function in mice with Duchenne treated with these combinations of compounds, followed by a biochemical and histological examination of skeletal and cardiac muscle.”
Selsby said the support for research from Ryan’s Quest and Michael’s Cause is helping to explore compounds with the potential for immediate impacts and application to patients suffering from Duchenne.
“Therapeutic home runs have been few and far between for Duchenne, so there's an urgen need for base hits that could have immediate impact,” Selsby said. “If what we’re discovering with these naturally occurring compounds can keep boys with Duchenne out of wheelchairs just a little while longer, it’ll be worth it.”
About Ryan’s Quest and Michael’s Cause
Ryan's Quest and Michael’s Cause are nonprofit organizations founded by families of boys with Duchenne muscular dystrophy. Both are dedicated to working together to fund promising biomedical research and clinical trials. Ryan’s Quest and Michael’s Cause are registered charities with 501(c)(3) status.