The Muscular Dystrophy Association (MDA) is financing 13 new research programs aimed at developing treatments for amyotrophic lateral sclerosis (ALS) and other life-threatening diseases. Toward that goal, the organization recently announced it would distribute $3.5 million in new development grants.
For the summer round of grants, 244 applicants submitted project ideas. Winners promised to approach different angles of the disease, including better understanding genes, mechanisms, and causes of the disease and exploring new therapy development aimed at future clinical trials.
Among the winners are researchers from the Children’s Hospital in Pittsburgh, the Baylor College of Medicine in Houston, Boston Children’s Hospital in Massachusetts, the University of Nevada School of Medicine in Reno, the University of California, Davis, and the University of Michigan Medical School in Ann Arbor.
The grants will finance research into new therapies to suppress disease mechanisms for ALS caused by a mutation in the FUS gene, and to advance molecular diagnostics for neuromuscular disease patients. Additional projects include testing the effectiveness of a U.S. Food and Drug Administration (FDA) approved therapy in the heart and muscle function of a mouse model for Duchenne muscular dystrophy, and new approaches to gene therapies for facioscapulohumeral muscular dystrophy (FSHD).
“We have seen unprecedented progress in neuromuscular disease research in the past few years, with six new drugs — four with direct ties to MDA research dollars — having been approved by the FDA to treat diseases in our program,” Grace Pavlath, PhD, senior vice president and scientific director at MDA, said in a press release.
“With the addition of this latest round of grants, we’ve begun funding some incredibly exciting research projects that we hope will keep the momentum going and result in more treatment options for our community,” she added.
With the new projects, MDA’s research spending totaled $13.7 million in 2017 alone.
Since its founding in 1950, the MDA has funded over $1 billion in neuromuscular disease research. This has led to four therapies being approved in just the last two years: Keveyis (dichlorphenamide) in August 2015 as a therapy for hyperkalemic and hypokalemic periodic paralysis, a disease characterized by muscle weakness or paralysis due to excessive or defective potassium levels; Exondys (eteplirsen) in September 2016 to treat the most common childhood form of muscular dystrophy; Spinraza (nusinersen) in December 2016 as a therapy for spinal muscular atrophy (SMA); and Emflaza (deflazacort) in February 2017 for Duchenne muscular dystrophy.
Currently, the MDA is funding over 150 different research projects worldwide, with a combined investment of $45.6 million raised with the help of the MDA’s donors and supporters.
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