The Muscular Dystrophy Association has awarded 13 new research and development grants totaling $3.5 million to accelerate the development of treatments for life-threatening diseases, incuding muscular dystrophy and amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The latest awards bring the MDA 2017 grant total to $13.7 million.
According to the association, the goal is to make progress in numerous areas including: Elucidating the genes/mechanisms/causes of disease, screening for drug leads and further developing experimental drugs, preparing for clinical trials, supporting young investigators, and de-risking further industry investment in drug development for neuromuscular disease.
The money will be used to fund research projects aimed at “unlocking the causes and mechanisms, and developing therapies for, life-threatening diseases that can take away the ability to walk, run, talk, eat, and even breathe independently.”
The new research grants include:
- Identifying new drugs for ALS: Scientists at the Children’s Hospital in Pittsburghare working to identify drugs that can suppress disease mechanisms for ALS caused by a mutation in the FUSgene.
- Facilitating new gene discovery in neuromuscular diseases: Researchers at Baylor College of Medicine in Houston are working to shed light on new biological insights and advance molecular diagnostics for neuromuscular disease patients.
- Testing a potential therapy for Duchenne muscular dystrophy (DMD): Researchers at the University of Nevada School of Medicine in Reno are assessing the effects of an existing FDA-approved drug on the function of heart and skeletal muscles in a mouse model of DMD — because the drug already is FDA-approved, successful outcomes of this study could lead to rapid translation into a new class of treatments for DMD.
- Searching for drug targets in facioscapulohumeral muscular dystrophy (FSHD): Scientists at Boston Children’s Hospital in Massachusetts are using cutting-edge techniques and a novel approach to search for genetic modifiers of FSHD that could serve as concrete targets for therapy development.
- Optimizing dosing for a drug to treat mitochondrial disease: Researchers at the University of California, Davis, are working to optimize dosing in an FDA-approved drug called dimethyl fumarate, or DMF, in animal models — successful outcomes for this work could provide data to support an investigational new drug (IND) application to the FDA for a new therapy for mitochondrial myopathy.
- Testing a potential therapy for spinal-bulbar muscular atrophy (SBMA): Scientists at the University of Michigan Medical School in Ann Arbor are completing preclinical studies in a mouse model to establish the safety and efficacy of a new type of therapy to silence activity of the gene that is mutated in SBMA.
“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,” said MDA senior vice president and scientific director Grace Pavlath, Ph.D. “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.”
MDA has funded more than $1 billion in neuromuscular disease research since 1950. Currently, the organization is funding more than 150 different research projects around the world with a combined investment of $45.6 million.
(Source: Muscular Dystrophy Association)
Filed Under: Drug Discovery