Senior scientist Julie Saba, at Children’s Hospital Oakland Research Institute in Oakland, Calif., was awarded an MDA research grant totaling $392,467 over a period of three years. The funds will help support Saba’s research into enhancing muscle regeneration and muscle stem cell functions as a new strategy for treating Duchenne (DMD) and Becker (BMD) muscular dystrophies.
In previous work, Saba and colleagues determined that metabolism of a lipid (fat-like substance) called sphingosine-1-phosphate, or S1P, is important in maintaining normal muscle development. S1P stimulates cell signals that promote muscle cell survival and activate muscle stem cells.
The team has found that when S1P levels in mice are decreased using drugs or genetic approaches, a corresponding decrease in muscle stem cell activation and muscle regeneration after injury occurs. The investigators also have determined that S1P signaling and metabolism are activated during muscle injury but may be deficient in muscles affected by MD, thereby contributing to poor muscle regeneration.
“In contrast,” Saba said, “when we use a food-derived small molecule that causes accumulation of S1P, we observe improved muscle regeneration and stem cell functions in a mouse model of MD.”
Such findings suggest that stimulating S1P signaling may improve muscle regeneration and strength in people with MD.
In her new work, Saba will study the effects of modulating S1P as a therapeutic strategy designed to work by activating peoples’ own stem cells to improve muscle regeneration.
“We hope that in the future we may also be able to leverage similar strategies of S1P modulation to improve muscle regeneration in combination with cell therapy approaches that are currently showing limited success,” Saba said.
Funding for this MDA grant began February 1, 2012.