The Muscular Dystrophy Association announced today the awarding of 70 new grants totaling over $17 million toward neuromuscular disease research. The newly funded projects aim to advance research discoveries and new therapy development in multiple areas including amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy (LGMD) spinal muscular atrophy (SMA), myasthenia gravis MG), and myotonic dystrophy (DM), and many more.
This round of grant funding reinforces Muscular Dystrophy Association's commitment to the progress of neuromuscular disease research and builds on the more than $1 billion the organization has already invested in research to uncover new treatments and cures for neuromuscular diseases since its inception. The organization's model of funding research across many neuromuscular diseases means findings from one disease often enable progress in others, maximizing the speed at which progress can be made. Each grant impacts neuromuscular disease research in a different way, from better understanding the underlying mechanisms of a particular disease to uncovering therapeutic targets to building clinical research infrastructure that will expedite clinical trials to providing early career support to promising young researchers.
"The Muscular Dystrophy Association has consistently been a leader in advancing treatments for individuals with neuromuscular disease. Patients are now living longer, more independent lives, due in large part to the more than $1 billion we have invested in research over the past 70+ years," said Donald S. Wood, PhD, President and CEO, Muscular Dystrophy Association. "We are pleased to invest an additional $17 million during this grant cycle to continue to fund the research that will lead to increasing advancements in neuromuscular disease treatments and cures."
"The Muscular Dystrophy Association continues to fund the most innovative research that will lead to cures for a range of neuromuscular diseases," says Sharon Hesterlee, PhD, Chief Research Officer, Muscular Dystrophy Association. "We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable."
The following are highlights from the 2022 Muscular Dystrophy Association Research Grant awards:
- Jeffrey Statland (University of Kansas Medical Center), Nicholas Johnson (Virginia Commonwealth University), and Rabi Tawil and Charles Thornton (University of Rochester) were jointly awarded the Clinical Research Network Grant of $1,517,502 to support their clinical trial network for facioscapulohumeral dystrophy, limb-girdle muscular dystrophy, and myotonic dystrophy. Muscular Dystrophy Association will support the consolidation of project management and oversite from 15 core centers in the United States under a single umbrella of the MD Clinical Trial Research Network, aimed towards clinical trial readiness for these diseases.
- Michael Shy (University of Iowa) was awarded the Research Infrastructure Grant of $439,250 to support the Inherited Neuropathy Consortium (INC) to develop the infrastructure necessary to evaluate therapies for patients with Charcot-Marie-Tooth disease.
- Forum Kamdar (Regents of the University of Minnesota) was awarded a Clinical Research Network Grant of $198,000 towards his project to combine data from large muscular dystrophy and advanced heart failure centers in order to assess cardiac care of patients and address gaps in current cardiac care of patients.
- Muscular Dystrophy Association is funding pivotal research to support the development of genetic therapies in Duchenne muscular dystrophy, mitochondrial diseases and LAMA2 muscular dystrophy. The projects focused on gene therapies are: "Utrophin Genome Editing for Duchenne's Muscular Dystrophy (DMD) Therapy" by Dr. Tejvir Khurana at the University of Pennsylvania; "Developing Gene Therapy Approaches for mtDNA Deletions" by Dr. Carlo Moraes at the University of Miami; and "Linker-based gene therapy of LAMA2-related muscular dystrophy using AAVMYO" by Dr. Markus Ruegg at the University of Basel.
- Muscular Dystrophy Association is also funding stem cell-based therapies to further develop treatments for muscular dystrophies: "Mobilizing Muscle Stem Cells to Treat DMD" by Dr. Michael Rudnicki at the Ottawa Hospital Research Institute.
- In addition, Muscular Dystrophy Association is funding grants to enhance understanding of disease mechanisms for ALS, Emery-Dreifuss muscular dystrophy, and congenital myopathies via funding the following projects: "Investigating a link between ER stress and nucleocytoplasmic transport in ALS" by Dr. Chang Geon Chung at the Johns Hopkins University; Assessing the role of Fibro-Adipogenic Progenitors in EDMD" by Dr. Chiara Mozzetta at the CNT Institute of Molecular Biology and Pathology; and "Calcium regulation of ER stress and protein degradation in congenital myopathy" by Dr. Amy Hanna at the University of Queensland.
- Recognizing the importance of genetic diagnoses in the era of genetic medicines, Muscular Dystrophy Association is also funding technologies with the potential to improve diagnostic yield and provide better information towards treatment and clinical trial options for patients and families affected by neuromuscular diseases. The following project is its commitment to improving the diagnostic process for neuromuscular diseases: "Long-read genome sequencing to diagnose neuromuscular disorders" by Dr. Greg Cooper at HudsonAlpha Institute for Biotechnology.
- Muscular Dystrophy Association will also fund several alternative therapeutic strategies to treat DMD using mirabegron (FDA approved B3AR agonist currently used for treating patients with hyperactive bladders), cannabidiol (CBD) and CSF1R inhibitors (a class of drug that takes on the formation of cancer tumors). These projects will explore the value of repurposing FDA approved compounds and their potential benefit on DMD: "Mirabegron in Treating Muscle Dystrophy" by Dr. Xuhui at the University of California at San Francisco; "Cannabidiol for the treatment of Duchenne Muscular Dystrophy" by Dr. George Rodney at the Baylor College of Medicine; and 'CSF1R inhibitors as therapeutics for Duchenne Muscular Dystrophy," by Dr. Fabio Rossi at the University of British Columbia.
