STANFORD GENETICIST RECEIVES $200K MDA RESEARCH GRANT TO DEVELOP NEW STEM CELL THERAPY FOR MUSCULAR DYSTROPHY

TUCSON, Ariz., Aug. 18, 2010 – Stanford University’s Michele Calos, Ph.D., professor of genetics, has been awarded a new $200,000 research grant from the Muscular Dystrophy Association to advance her lab’s pioneering work demonstrating that modified genes can be put into muscle using genetically engineered stem cells.  Ultimately, Calos aims to develop a therapy for Duchenne muscular dystrophy, a genetic disease that progressively weakens skeletal muscles. One out of every 3,500 boys is born with the genetic disease, which usually places patients in wheelchairs by their teens and can shorten lives by decades.

Michele Calos, Ph.D.

The work is at the cutting edge of a field that already is pushing the bounds of current technology.  “Our study involves genetic engineering, and it involves stem cells.  Both of these areas are relatively new and have considerable potential.  We will be trying novel approaches in an effort to provide clinical benefit for patients,” Calos explained.  “It is appropriate for patients and families to hope for progress,” she notes, “but also necessary to understand that we have a lot to learn and it will take some time to figure out the best way to apply the therapy.”
           
“Dr. Calos is refining an innovative technology for putting modified genes into cells,” said Louis M. Kunkel, Ph.D., chairman of the MDA Scientific Advisory Committee, and himself a pioneer in the area of muscular dystrophy research.  “Her work could ultimately enable researchers to treat Duchenne and Becker muscular dystrophies by successfully introducing a gene that produces the vital dystrophin protein that’s absent or malfunctioning in these diseases. It also could have widespread implications for treating other genetic diseases with modified genes.”

Calos is one of 38 research leaders who’ve been awarded multiyear grants from a new round of MDA grant funding, totaling just over $14 million.  MDA has invested almost $39 million in 2010 in neuromuscular disease research projects around the world. 

Calos said the MDA grant she’s receiving is vital to her work.  “There are limited resources available for the kind of cutting-edge gene therapy and stem cell therapy research that we pursue.  MDA funding is very important, because it allows us to pursue original ideas and develop proof-of-principle results.  I believe that pursuing more creative approaches and recruiting investigators that are relatively new to neuromuscular diseases can be critical to progress.”

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 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, 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 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 in helping to refine outcome measures for those clinical trials.

More than 1 million people in America are affected by neuromuscular diseases.

Northern California residents affected by any of the more than 43 muscle diseases covered by MDA’s program can receive excellent medical care at the MDA Clinic at Stanford University’s Department of Neurology; the Forbes Norris MDA/ALS Research Center at California Pacific Medical Center, San Francisco; the MDA Clinic at the University of California Medical Center at UC San Francisco Children’s Hospital; and the MDA Clinic at the UC Davis, Department of Physical Medicine and Rehabilitation Clinic. 
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research, view MDA research B-roll.  The Association also provides comprehensive health care and support services, advocacy and education.  For more information on MDA Research and programs go to: http://www.mda.org/.