MDA Awards Three-year, $900,000 Grant to Jerry Mendell, MD, of Nationwide Childrens Hospital

July 14, 2009

At a time when federal and private funds for biomedical research have become scarce, the Muscular Dystrophy Association reasserts its leadership in the fight against muscle diseases by announcing grants to innovative research projects throughout America and in Canada.

Federal support for the type of work we do has just about dried up, especially in the current economic climate. Simply put, without the support of MDA, this line of investigation could not be pursued by our lab, said Eric Schon, researcher at Columbia University Medical Center in New York.

MDA has committed over $2 billion to fund medical and scientific research, as well as clinical and client services, in its 55-year history.

These new research grants, just announced at the July MDA Board of Directors meeting, support research to be conducted from 2009 through 2011, totaling almost $5 million. MDA committed approximately $43million to new and ongoing research in 2008, said MDA Chairman of the Board R. Rodney Howell.

There are no cures yet for most muscle diseases, but the research MDA has funded has created the basis for progressive treatments of muscular dystrophy, amyotrophic lateral sclerosis (ALS), or Lou Gehrigs disease, and spinal muscular atrophy. It also served as the basis for a lifesaving therapy developed by Genzyme for a metabolic muscle disease called Pompe disease.

At Columbia University, Schon has been awarded a three-year $400,000 grant to continue research into diseases of the mitochondria caused by genetic mutations. Mitochondria are the powerhouse of the cell and, when they fail, can cause muscle weakness and pain, diabetes, blindness and heart disease, along with other debilitating and/or fatal conditions. Schons research may provide the key for treating manymuscle diseases.

MDA has awarded a three-year, $900,000 award to Jerry Mendell at Nationwide Childrens Hospital in Columbus, Ohio for a study to combat heart disease in muscular dystrophy. While advances in treatment of the devastating Duchenne muscular dystrophy (DMD) have prolonged boys lives, physicians are seeing more heart disease develop in young men living with the disease.

The longer patients with DMD live, the greater the risk of cardiac complications. In this study, we hope to improve cardiac care by establishing the best medication regimen and to look at the best time to start treatment to protect the patient from heart failure, Mendell said.

He added, It is clear that without the support of MDA this study and others like it could not be done. MDA is devoted to the treatment of Duchenne and the progress that has been made to this date is the result of the MDA commitment.

At the Ottawa Hospital Research Institute, in Ottawa, Ontario, Associate Scientific Director Rashmi Kothary will be able to continue his research seeking answers to the biological processes that cause SMA. MDA is awarding Kothary $300,000 for three years. Kothary is also affiliated with the University of Ottawa and like so many researchers, he is aware of and affected by the cutback in research funding.

The financial commitment from MDA is critical for the continued research in this area within my laboratory. It will contribute to the training of highly qualified personnel and trainees who will be the future scientists to tackle neuromuscular disease questions.

In San Diego, Hristelina Ilieva of the Ludwig Institute for Cancer Research has been awarded $180,000 over three years to study how defects in the blood-brain barrier contribute to amyotrophic lateral sclerosis (ALS), or Lou Gehrigs disease. Ilieva hopes to find a way to use genetic therapy to treat hereditary ALS.

It will also enable my final phase postdoctoral training; thereby providing me what I hope is a major career development opportunity that will form the basis for my own independent career, a career in which I can use both my MD and Ph.D. training in further development of therapies for ALS.

All projects seeking MDA funding are reviewed by leading experts on the Associations Medical Advisory Committee or Scientific Advisory Committee before being presented to the Board for approval. In all, 272 research applications were submitted to MDA for potential funding. Currently MDA supports 333 research teams around the globe.

About MDA
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education. The majority of contributions to MDA come from individual donors.

About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report ‘s 2018-19 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.