Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD of the University of Florida in Gainesville to test the ability of magnetic resonance imaging (MRI) to detect changes in the muscles of those living with Duchenne muscular dystrophy (Duchenne) who participated in the Sarepta phase II trial of Eteplirsen.
"This supplemental funding from PPMD allowed us to very quickly take advantage of a trial in progress to see if MRI can detect changes in the amount and quality of muscle in response to an experimental treatment," said Dr. Vandenborne. ... Now PPMD has provided an additional $59,000 to Dr. Vandenborne to test the ability of MRI to detect changes in the muscle in response to a treatment – in this case Sarepta's Eteplirsen. The investigators have collected data from the active phase of the study and will continue collecting data as the original study participants stay on the drug in the extended access program. Results of the MRI analysis from the phase II Eteplirsen study will be submitted for publication as soon as the data is compiled.
"Developing accurate, reliable ways to detect when new drugs are working has become increasingly important, as more drugs enter clinical testing for Duchenne," said PPMD's Founding President and CEO Patricia Furlong . "This study is part of our ongoing effort to provide tools that will give each new experimental drug the very best chance for success."
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.
Humm, It is a five year study. So even if positive results showed with SPPT it could take years before published. It may be well to have a add on study for progression of SRPT for future testing for DMD treated patients. The current study adds all MD types.