PPMD awards $75,000 grant to University of California professor for Duchenne muscular dystrophy research

Dr. Carmen Bertoni to Receive $75,000 For Duchenne Muscular Dystrophy Research

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced today the latest recipient of the End Duchenne Grant Award Program. Carmen Bertoni, Ph.D., Assistant Professor of the Department of Neurology at the University of California - Los Angeles will receive a grant for $75,000, to be used to continue her work with high throughput screening (HTS) to identify chemical compounds that increase the efficiency of oligonucleotide-mediated (ssODN) gene correction in Duchenne muscular dystrophy ("Duchenne") and that can be advanced into a clinical application for the treatment of Duchenne.

The End Duchenne Grant Award Program was created by PPMD in partnership with the National Institutes of Health (NIH) in an effort to ensure continuation of promising Duchenne research and translation to human studies. The End Duchenne Grant Award Program is a bridge grant provided by PPMD to selected research projects that receive scores beyond the current funding paylines of the NIH Institutes and Centers supporting Duchenne research. The first grants were awarded earlier this year.

Explains Ms. Furlong, "With its research budget growing tighter each year, the NIH can fund only a fraction of the many promising applications it receives. Currently, only those grants which score in the top percentiles are able to be funded."

The End Duchenne Grant Award Program represents the first Duchenne specific bridging program and the first ongoing bridge funding program to be presented in the rare disease category. As a collaborative effort with the NIH, the selection strategy behind this award is very different than any other private research investment in Duchenne, yet the goal of this award remains consistent with all of PPMD's research initiatives: to advance promising research which will impact this generation affected by Duchenne.

The recipient of an End Duchenne Grant must be focused on translational research (the process of applying ideas, insights and discoveries generated through basic scientific inquiry to the treatment or prevention of disease or injury). The NIH will notify investigators whose translation grant applications score well, but not within the NIH funding levels, to submit their applications and score sheets to PPMD. H. Lee Sweeney, PPMD's Scientific Director and a member of the Grant's Review Board says, "PPMD's Scientific Review Board will re-review these applications seeking to identify those with the greatest potential to ultimately help all boys with Duchenne."

A researcher at the University of Pennsylvania himself, Dr. Sweeney went on to say that "Interest in the End Duchenne Grant Program is continuing to grow tremendously. Scientists have renewed faith in their projects because an organization like PPMD is showing their support by providing bridge funding. The quality and promise of the entries we are reviewing is impressive to say the least. UCLA received funding earlier in the year for the work that Dr. Melissa Spencer is doing. We are thrilled that Dr. Bertoni has also chosen Duchenne as the focus of her talents. If we can continue to help scientists like Dr. Bertoni maintain the momentum of these important avenues in treatment, so that they can receive funding from the NIH in the near future, then we will consider this grant program a huge success."

Dr. Bertoni's proposal will screen the Prestwick library, a collection of all of the FDA approved drugs to identify compounds that can significantly enhance the ssODN-mediated gene repair and that are already approved for use in humans, thus increasing the chances of moving this technology into a clinical scenario in a relatively short period of time.

Says Dr. Bertoni, "It is a tremendous honor to have the faith and support of the families fighting Duchenne. I believe my work will help speed up the approval process for getting critical technology to the trial stage and because of the financial support of PPMD, we can continue moving our work forward."

The End Duchenne Grant Award Program was designed to enable investigators to continue their projects and generate additional data for a successful re-application within a 12 to 24 month period, thus leveraging additional Duchenne-specific research dollars.

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.

An applicant seeking support from the End Duchenne Grant Award Program must be employed at a for-profit or non-profit organization or institution and have submitted and received a complete review of an application to the NIH directed toward translation of research into human clinical studies specific for Duchenne muscular dystrophy. The applicant must have the resources to conduct the proposed research project and the organization/institution must have appropriate grant administrative capacities for the handling and disbursing of research funds. For more information or to apply, please visit www.parentprojectmd.org/EndDuchenneGrant.