MDA awarded a grant totaling $367,386 over three years to Masahiko Hoshijima, professor of medicine at the University of California, San Diego in La Jolla, Calif. The funds will help support Hoshijima's research into connections between cardiac and respiratory failure in muscle diseases such as Duchenne (DMD), Becker (BMD) and other muscular dystrophies (MDs).
Hoshijima plans to study how heart failure and respiratory failure are interlinked in MD. Weakness of the respiratory muscles, including the diaphragm and intercostal (part of the rib cage) muscles, leads to inadequate breathing called "hypoventilation" in many MDs. In contrast, these diseases affect the heart muscle directly.
Using the BIO14.6 hamster research model, Hoshijima and colleagues will administer gene therapy selective for muscle type at two different time points: early-stage treatment meant to prevent disease onset and progression, and later treatment meant to "rescue" animals after heart and respiratory system damage is well-developed.
One group of animals will receive heart treatment only; a second group will be treated with a therapy that targets skeletal and respiratory muscles and excludes heart treatment. The study team hopes data from these studies can clarify how heart failure and respiratory failure are independently as well as collaboratively contributing to the ultimate disability and death of the animal model of muscular dystrophy.
In further studies, the investigators plan to administer muscle-specific gene therapy to the rodent model in order to determine whether heart treatment can improve respiratory function and whether skeletal muscle-specific gene therapy can benefit systemic circulation.
Findings from Hoshijima's work may provide a better understanding of how better to treat human muscular dystrophies that affect more than one muscle type.
MDA funding, Hoshijima noted, is "vital to select rare-disease patient groups who can find hope in the potential for development of therapies tailored to their diseases."
Funding for this MDA grant began February 1, 2011.