Funded projects cover DMD, ALS, spinal muscular atrophy, facioscapulohumeral muscular dystrophy, and Friedreich ataxia.
The Muscular Dystrophy Association (MDA) is giving out a total of $13.7 million to 40 new research initiatives targeting nearly two dozen progressive neuromuscular diseases. Among these are 13 initiatives targeting Duchenne muscular dystrophy (DMD), nine projects focused on amyotrophic lateral sclerosis (ALS), as well as efforts on spinal muscular atrophy (SMA), facioscapulohumeral muscular dystrophy (FSHD), and the link between diabetes and Friedreich ataxia (FA).
This latest round of peer-reviewed grants recommended by the association’s medical and scientific advisory committees has been approved for funding by the MDA board of directors. The programs are under way 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 to screen thousands of experimental compounds and FDA-approved drugs. 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 is investigating whether a combining licofelone with 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 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 regards to FA, a project in Philadelphia will use MDA money to determine the exact mechanisms by which diabetes occurs in FA. This may also shed insights into the causes of insulin resistance in people with type 2 diabetes.
Other initiatives by MDA-funded investigators include the Minneapolis-based effort to identify inhibitors of the DUX4 gene implicated in FSHD and to test promising compounds in a transgenic mouse model for that disease as well as a Miami-based program to find novel genes that cause more rare forms of SMA and to search for genetic modifiers of the disease-causing genes.