MDA AWARDS $364K TO MIAMI RESEARCHER ON PROMISING PATHWAY TO TREATMENT FOR MITOCHONDRIAL MYOPATHY

TUCSON, Ariz., Aug. 18, 2010 – When Dr. Carlos Moraes sees a challenge, he confronts it. For 20 years, he has doggedly pursued treatments for mitochondrial myopathy, with much of the work funded by the Muscular Dystrophy Association. Moraes, a professor at the University of Miami, Miller School of Medicine, recently found a way to successfully treat a mouse model for the progressive neuromuscular disease, and has just been approved for another MDA grant of $363,672 to accelerate progress toward a human treatment for mitochondrial myopathy.

Dr. Carlos Moraes

“We are working on finding alternative approaches to treatment,’’ said professor Moraes.  “The one related to this grant is the increase in mitochondrial biogenesis in the muscle cell. We found that by increasing the number of mitochondria we can improve the muscle disease. We have proven that in a mouse model of mitochondrial myopathy. That has made us very excited and motivated.’’

Moraes is one of 38 research leaders receiving new multi-year awards from MDA. His three-year study is part of the $14.1 million in new research funding approved during the Association’s July board meeting. This is Moraes’ sixth MDA research grant. Over the years, he has been awarded a total of $1.53 million. To learn more about Moraes and his current project, read his MDA Q&A transcript.

Moraes also has shown that his novel treatment approach for mitochondrial myopathy is yielding important insights into how to reduce muscle loss that is part of the normal aging process. “Some of the studies we’ve done increased the mitochondrial number in muscle of older animals to protect against the muscle loss in the aging process, called sarcopenia,’’ Moraes said. “You lose muscle when you get old. However, the increase in mitochondria biogenesis seems to decrease this loss during aging.  This improvement is very similar to what we observed in models of mitochondrial myopathies.’’

Moraes said MDA’s funding “is critical’’ and “extremely valuable’’ to the work he does in helping find a cure or treatment. “MDA has been a pioneer in funding research for muscle diseases,’’ Moraes said. “Without its funding, we would not be able to do this project.’’

“Dr. Moraes has opened a window to an exciting new avenue of potential therapy for mitochondrial myopathy,’’ added Howell, professor of pediatrics and chairman emeritus of the Department of Pediatrics at University of Miami Leonard M. Miller School of Medicine.  “His persistence and creativity are noteworthy, and it's exciting to think about the potential health benefits that could be achieved through his research.’’

According to R. Rodney Howell, M.D., chairman of the MDA Board of Directors, “generous Americans responding to the annual Jerry Lewis MDA Telethon, and to thousands of other special events benefiting the families served by MDA, deserve much of the credit for the rapid progress being made towards treatments for neuromuscular diseases.  It’s their strong belief in MDA’s capable stewardship of public funds that’s enabling so much to be accomplished – even in a sluggish economy.”

Research grant applications are peer-reviewed twice yearly by MDA’s Medical and Scientific Advisory Committees, comprising world-renowned experts in neuromuscular disease research.  The most promising of some 500 applications received each year are recommended for funding to the MDA Board of Directors.

Often credited for its leadership in building the field of neuromuscular disease research, MDA has simultaneously enhanced clinical care for individuals affected by muscle disorders, achieving important quality of life and longevity gains. The Association, which annually invests almost $39 million on research worldwide, is the first nonprofit to earn a Lifetime Achievement Award from the American Medical Association (“for significant and lasting contributions to the health and welfare of humanity”).

MDA-funded scientists have uncovered the genetic defects that cause several forms of muscular dystrophy; Charcot-Marie-Tooth disease (CMT); a form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease); childhood spinal muscular atrophy (SMA) and other neuromuscular conditions. Now entering a period of increasing numbers of clinical trials of potential therapeutics, the Association’s network of approximately 200 hospital-affiliated clinics is instrumental in identifying appropriate candidates for clinical trials; and to help refine outcome measures for those clinical trials.

More than one million people in America are affected by neuromuscular diseases.

Residents of South Florida who are affected by any of the 43 muscle diseases covered by MDA’s program can receive excellent medical care at the MDA Clinics at: Broward General Medical Center; Lee Memorial Hospital; and the University of Miami School of Medicine.

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. For more information on MDA research and programs, go to www.mda.org. 

For more information about these new grants, visit MDA's "Grants at a Glance," an online slideshow that showcases each grant with photos and detailed information.