COLUMBIA, Mo., BIOLOGIST AWARDED $164K GRANT TO STUDY STEM CELLS IN MUSCLE REPAIR

TUCSON, Ariz., Aug. 18, 2010 — University of Missouri biologist Dawn Cornelison and her lab may be close to finding a piece of a very complex puzzle that ultimately could lead to a treatment or a cure for Duchenne muscular dystrophy (DMD).

That’s why she’s just been awarded a $163,576 grant from the Muscular Dystrophy Association to help her find a viable therapy for the rapidly progressive neuromuscular disease that affects one in 3,500 boys.

Cornelison and her lab study satellite cells, adult stem cells that the body uses to repair damaged muscles. This "small but mighty" cell population is responsible for normal muscle growth and repair, and is also the target of many developing therapies for DMD. When they detect damage to the muscle, these cells first divide rapidly to produce many more potential new muscle cells, then differentiate into functional muscle. Cornelison is building on the discovery made during her previous MDA-funded project that a single protein (used to sense factors in the tissue surrounding the satellite cell) is necessary for both steps in this process.

Dawn Cornelison

"What we've done is to show that we can separate out these two activities, and tie each one to a different small part of the molecule. The interesting thing is that both the part that is needed for proliferation and the part that are needed for differentiation are places where the cell can build complexes of many proteins; now we want to find out what exactly is being assembled at those two spots," Cornelison explained.

One goal of this research is to understand how satellite cells make decisions about what to do in the body during injury or disease based on what they are exposed to in the damaged muscle. This may help to overcome problems with cell therapies. In DMD, muscle cells are missing the crucial protein dystrophin. But, when researchers try to put healthy satellite cells containing dystrophin back into a damaged muscle, the results have not so far led to replacement with healthy muscle.
Cornelison, assistant professor of biological sciences at the University of Missouri, added, “The big question in our research is: How do these cells notice and respond to their environment?  We hope that once we understand this better, we can ask why aren’t these cells that we’ve given a good gene, behaving the way they would if they were there naturally instead of planted there therapeutically?”

Cornelison is one of 38 research leaders awarded, collectively, $14.1 million in new multiyear grants by MDA’s Board of Directors at its July meeting.  She has studied muscle regeneration for more than a decade.  To learn more about Cornelison and her research listen to her podcast or read transcript.

I have actually been supported by MDA my entire career. My graduate adviser had funding from MDA.  My postdoctoral adviser had funding from MDA, and when I was a postdoctoral fellow I got a grant to fund the last three years of my postdoc research under MDA’s Development Grant program.  When I started my independent position here at the University of Missouri about five years ago, I got a grant from MDA to follow up on some work I had successfully completed as a postdoc, which led to what we are working on now.  So, I’m very, very grateful to MDA.  The Association has been very generous in funding my research,” Cornelison explained.

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.
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.”
“Dr. Cornelison’s work is pivotal in helping us understand what happens in a transplanted cell’s environment.  This knowledge will be critical to turning cell and gene therapies into viable treatments, not only for people with DMD but for many other neuromuscular diseases,” said Louis M. Kunkel, Ph.D., chairman of the MDA Scientific Advisory Committee and a gene therapy pioneer.

Often credited for its leadership in building the field of neuromuscular disease research, MDA also has enhanced clinical care for people affected by muscle disorders, achieving important quality of life and longevity gains.  MDA has invested almost $39 million in 2010 in research worldwide and 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 Americans are affected by neuromuscular diseases.  Missouri residents affected by any of the more than 40 muscle diseases in MDA’s program can receive excellent medical care at MDA Clinics at: the University of Missouri Adult Neurology Department in Columbia; the St. John's Regional Medical Center in Joplin; the St. John's Regional Health Center in Springfield; and Washington University School of Medicine in St. Louis.  Individuals living with ALS are encouraged to visit the MDA/ALS Center at Washington University School of Medicine in St. Louis.

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research view video of MDA research at http://www.mda.org/research/gaag/2010/b-roll.html.  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.
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.