TUCSON, Ariz., Aug. 24, 2011 ─
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 recommended by the Association’s Medical and Scientific Advisory Committees comprising the world’s top medical and scientific authorities was approved for funding by the MDA Board of Directors. The promising new initiatives are underway in 17 U.S. cities, the District of Columbia, as well as in Australia, Canada, Costa Rica, Italy, the Netherlands and the United Kingdom:
In Winston-Salem, NC, 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 up-regulate 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 Quebec City 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 (TDP-43 + FUS/TUS) 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 more rare forms of SMA and to search for genetic modifiers of the disease-causing genes.