Parent Project Muscular Dystrophy Grants $175,000 for Biomarkers, Non-Ambulatory Endpoints in Duchenne Muscular Dystrophy
Dr. Craig McDonald of UC Davis to Receive Supplemental Funds
HACKENSACK, N.J., Jan. 18, 2013 /PRNewswire-USNewswire/ -- 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.
About Duchenne muscular dystrophy
Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.
SOURCE Parent Project Muscular Dystrophy