TUCSON, Ariz.—The Muscular Dystrophy Association today announced funding totaling $13.7 million for 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases. Among these are 13 new initiatives targeting Duchenne muscular dystrophy (DMD), nine new projects focused on ALS (amyotrophic lateral sclerosis or Lou Gehrig's disease), as well as efforts on spinal muscular atrophy (SMA), facioscapulohumeral muscular dystrophy (FSHD), and the link between diabetes and Friedreich's ataxia (FA).
These new projects are in addition to hundreds of other MDA-funded scientific investigations being advanced worldwide to find effective treatments for neuromuscular diseases, "Truly rapid progress is being made in the fight against muscular dystrophy, ALS and related diseases," said R. Rodney Howell, M.D., chairman of the MDA Board of Directors. "And MDA will not stop until these diseases are conquered."
This latest round of peer-reviewed grants were recommended by the Association's Medical and Scientific Advisory committees, comprising the world's top medical and scientific authorities, and were approved for funding by the MDA Board of Directors. The promising new initiatives are under way in 17 U.S. cities and the District of Columbia, as well as in Australia, Canada, Costa Rica, Italy, the Netherlands and the United Kingdom:
Baltimore, Md. (Grants 1, 2)
Boston, Mass. (Grants 1. 2, 3, 4, 5, 6)
District of Columbia
Houston, Texas (Grants 1, 2, 3, 4)
Iowa City, Iowa
La Jolla, Calif. (Grants 1, 2, 3)
Los Angeles, Calif.
New York, N.Y.
Palo Alto, Calif.
Philadelphia, Pa. (Grants 1, 2)
London, United Kingdom
Quebec City, Canada
San Pedro, Costa Rica
Victoria, Australia (Grants 1, 2)
In Winston-Salem, N.C., investigators are studying DMD-related heart disease by reprogramming skin cells to create heart cells in order to screen thousands of experimental compounds and drugs already approved by the Food and Drug Administration (FDA). Another MDA-funded team in Philadelphia is exploring a new way to upregulate utrophin, a muscle protein that may be able to stand in for the larger dystrophin protein that, when absent, causes DMD. Plus, an MDA-sponsored team in Victoria, Australia, is working to prevent extensive bone loss caused by corticosteroids, the only available treatment shown to slow the progression of DMD.
For ALS, a Houston-based team with MDA funding is investigating whether a combination drug treatment (Licofelone + Riluzole) works better than the first FDA-approved drug for ALS, riluzole. Another group of MDA-sponsored investigators in Montreal is looking for the earliest visible signs of ALS. Finally, a San Diego team supported by MDA is using next-generation gene sequencing technology to better understand what makes ALS-causing mutations in two genes (TDP43 + FUS) so crucial to the ALS disease process.
With tens of millions of Americans affected by type 2 diabetes and 30 percent of FA patients developing diabetes, a new MDA-funded project in Philadelphia to determine the exact mechanisms by which diabetes occurs in FA also could shed valuable insights into the causes of insulin resistance seen in people with type 2 diabetes.
Other exciting initiatives by MDA-funded investigators include the Minneapolis-based effort to identify inhibitors of the DUX4 gene implicated in FSHD, and to quickly test promising compounds in a transgenic mouse model for that disease; and a Miami-based effort to find novel genes that cause rare forms of SMA and to search for genetic modifiers of the disease-causing genes.
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.
In addition to funding more than 300 research projects worldwide, MDA maintains a national network of some 200 hospital-affiliated clinics; facilitates hundreds of support groups for families affected by neuromuscular diseases; and provides extraordinary local summer camp opportunities for thousands of youngsters fighting progressive muscle diseases. MDA is the first nonprofit to receive a Lifetime Achievement Award from the American Medical Association "for significant and lasting contributions to the health and welfare of humanity."