MDA AWARDS $302K TO UT RESEARCHER DEVELOPING FATIGUE-RESISTANT MUSCLE AS PATHWAY TO TREAT DUCHENNE MUSCULAR DYSTROPHY

TUCSON, Ariz., Aug. 18, 2010 — One of Dr. Vihang Narkar’s life joys is “making fundamental biomedical discoveries."  It’s a reason why he’s a researcher at The University of Texas Health Science Center at Houston’s Institute of Molecular Medicine, and he’s convinced that boosting fatigue-resistant aerobic muscle could be a potential therapeutic pathway for treating Duchenne muscular dystrophy (DMD).

Noting that Duchenne is a progressive neuromuscular disorder caused by a defective gene that fails to produce the structural protein known as dystrophin, Dr. Narkar explains that while fixing or replacing a defective gene is a difficult proposition, it’s critical to explore how “fundamental muscle remodeling pathways can be used to bypass the genetic defect and combat pathology.”

Dr. Narkar will be able to explore this exciting DMD research direction with the help of a new, three-year Muscular Dystrophy Association grant of $302,326.

Dr. Vihang Narkar

“What we have on our hands is a genetic pathway which can make a muscle resistant to fatigue," Narkar said.  “We have found that over-expression of an orphan estrogen-related nuclear receptor in healthy mouse skeletal muscle activates genes that encode a highly aerobic and fatigue-resistant muscle." Now, his laboratory is investigating whether the nuclear receptor pathway can be activated to engineer the aerobic “muscle of choice” in DMD to reverse the dystrophic pathology.

Dr. Narkar is one of 38 research leaders receiving new multiyear awards from MDA. His three-year project is part of the $14.1 million in research funding approved during the Association’s July Board meeting. This is his first grant through MDA. To learn more about Dr. Narkar about his current project, listen to his recent MDA Q&A podcast.

Narkar called the grant “vital’’ for the next wave of experiments.

“MDA is a substantial source of research support," he said, adding that “a lot of exciting science comes out of labs funded by the MDA which has prompted further studies. MDA plays an important role in advancing treatments for muscle diseases." 

“Dr. Narkar’s research is innovative and important," said R. Rodney Howell, M.D., chairman of the MDA Board of Directors. “We’re delighted that he’s joining the MDA family of investigators who are collectively focused on speeding treatments to help children and adults affected by progressive muscle diseases."

Howell added, “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 toward 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, comprised of 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 has invested almost $39 million in 2010 in 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 1 million people in America are affected by neuromuscular diseases.

In the greater Houston area, individuals affected by any of the more than 40 muscle diseases in MDA's program can receive excellent medical care at the MDA Neuromuscular Clinic at Methodist Neurological Institute, and the MDA Clinic at Texas Children’s Hospital.

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.