Parent Project Muscular Dystrophy (PPMD) has awarded Dr. Craig McDonald of the University of California, Davis (UC Davis) $175,000 in supplemental funds to expand his ongoing study through the 20 CINRG (Cooperative International Neuromuscular Research Group) centers to better understand the progression of Duchenne muscular dystrophy (Duchenne) and determine the impact of the Duchenne standards of care established by the Centers for Disease Control (CDC). Related studies will focus on developing new endpoints in boys who are still walking and those who can no longer walk, and identifying blood markers that track the progression of the disease. The PPMD funding will allow Dr. McDonald to recruit 100 additional subjects into the study.
"Funding from PPMD will make a critical difference in the development of new outcome measures and the overall quality of the data we collect in this study," said McDonald who is a professor and chair of the UC Davis Department of Physical Medicine and Rehabilitation. "These studies have already impacted the development of endpoints for trials in Duchenne and the new funding will allow us to extend the research into younger boys and boys who have not yet started taking steroids."
This research is critical because companies developing therapies for Duchenne, the Food and Drug Administration, and academic investigators have all expressed a need to develop additional "endpoints," or ways to measure the effectiveness of experimental drugs in clinical trials. Although the six minute timed walk has been used in many clinical trials to date, new endpoints that can be used for wider ranges of ability so that more boys will be eligible to participate in drug trials could improve investigators' ability to recruit for clinical trials. For example, Dr. McDonald's group is developing an "upper extremity function test" that could be used in clinical trials for subjects who have lost the ability to walk.
In addition to developing new endpoints, the investigators will also be taking blood samples from participants to look for proteins in the blood or "biomarkers" that could indicate quickly whether or not a drug is working. The group has already identified several prospective biomarker candidates and now needs to test those markers in a larger group of participants.
"We are pleased to provide funding to enhance this project," said Pat Furlong , Founding President and CEO of Parent Project Muscular Dystrophy. "As the pipeline of new drugs for Duchenne continues to mature, we are committed to making sure that investigators and companies have all the tools they need to approve drugs efficiently."
This grant was made possible as a result of PPMD's recent holiday campaign which focused on raising funds for projects aimed at identifying biomarkers and genetic modifiers, which will help to unlock better and faster ways of advancing clinical trials.
Parent Project Muscular Dystrophy