MDA awarded a research grant totaling $308,061 over a period of three years to Lawrence Steinman, Zimmerman Chair and professor of neurology, neurosciences and pediatrics at Stanford University School of Medicine in Stanford, Calif. The funds will help support Steinman’s work to inhibit immune system response to replacement of the missing dystrophin protein in Duchenne muscular dystrophy.
Dystrophin-specific immunity has been reported in people with DMD both before and after treatment with gene therapy.
Steinman and colleagues plan to “map the exact nature” of the immune response to dystrophin in boys with Duchenne MD. In addition, the investigators have invented a strategy designed to shut down the immune response to dystrophin, and plan “to test this approach in experimental gene therapy in mice with a DMD-like disease.”
Steinman said that most attempts to suppress unwanted immunity in DMD use “big-hammer drugs” with strong immune system suppression properties. His new approach is more selective, he noted, designed to “turn off the immune response specifically when it is unwanted,” leaving the rest of the immune system intact.”
“Ultimately, this could increase the success of gene therapy in DMD,” Steinman said, “by inhibiting an unwanted immune system response when the normal dystrophin molecule is produced.”
Funding for this MDA grant began February 1, 2012.