14 Research Grants and 7 Development Awards for Fellowship Trainees Will Fund Critical Research Across Multiple Neuromuscular Diseases. MDA Collaboration Grants include AFM-Téléthon and Coalition to Cure Calpain 3.
Muscular Dystrophy Association Announces $5,000,000 for Research Grants to Accelerate Treatments
This grant cycle continues MDA's long-standing commitment to funding innovative research that has the potential to uncover new therapeutic targets and accelerate the development of treatments. The organization's investment in neuromuscular disease research exceeds $1 billion, marking significant progress in improving the lives of people living with these conditions.
"The Muscular Dystrophy Association is proud to continue driving critical research that brings hope and tangible progress to the neuromuscular community,” said Donald S. Wood, Ph.D., President and CEO, Muscular Dystrophy Association. "These projects reflect our dedication to the people we serve; we are eager to see how they will fuel clinical and scientific progress and, ultimately, lead to new treatments.”
MDA's strategic approach to funding research across a broad array of neuromuscular diseases ensures that findings in one disease area can often accelerate advancements in others. This innovative model maximizes the potential for breakthroughs that have led to life-changing treatments and care.
"We are excited to award these new grants to researchers whose work has the potential to bring us closer to disease-modifying treatments for neuromuscular diseases,” said Sharon Hesterlee, Ph.D., Chief Research Officer, Muscular Dystrophy Association. "These grants not only support established researchers but also nurture the next generation of scientists who will continue the fight against these debilitating diseases.”
2024 MDA Research Grant Highlights:
- James Dowling, M.D., Ph.D. (The Hospital for Sick Children) was awarded a grant to explore new ways to understand and prevent liver problems caused by gene therapy using AAV (adeno-associated virus). Dr. Dowling's team has developed a new method where mice are fed a diet similar to humans to help mimic liver issues experienced by patients in response to gene therapy. With this model, they can now study the liver damage caused by gene therapy and look for ways to prevent it.
- Jyoti Jaiswal, Ph.D (Children's Research Institute) was awarded a grant to study new treatment options for preventing muscle cell death and damage in Duchenne muscular dystrophy (DMD). This project will explore a therapy that targets lipids to help stabilize muscle cell membranes, reduce inflammation and muscle damage, and improve muscle function in DMD.
- Scot Wolfe, Ph.D. (University of Massachusetts Chan Medical School) was awarded a grant to study the in vivo correction of limb-girdle muscular dystrophy (LGMD) mutations. This project combines stem cell, nanoparticle, mouse model and genome editing technologies in translational studies to develop a corrective gene repair strategy for common mutations that cause LGMD 2G/R7. The technologies developed will provide a platform to correct other gene mutations that cause other forms of LGMD.
- Virginia Kimonis, M.D., M.R.C.P. (University of California Irvine) was awarded a research grant to investigate the use of new antisense oligonucleotides (ASOs) as a treatment for Valosin-containing protein (VCP) disease. The project will assess whether these ASOs, developed in collaboration with Ionis Pharmaceuticals, can effectively reduce VCP activity to normal levels in mice and stem cells with the eventual goal of clinical trials.
- Thomas Gaj, Ph.D. (The Board of Trustees of the University of Illinois Urbana-Champaign) was awarded a research grant to use a novel gene-editing technology to target and silence the harmful C9ORF72 RNAs responsible for the most prevalent genetic form of ALS. The project aims to deepen our understanding of the mechanisms underlying C9ORF72-related ALS and to develop an effective gene-targeted therapeutic for this genetic variant, accounting for nearly 40% of familial cases.
- Natalia Rodriguez-Muela, Ph.D. (German Center for Neurodegenerative Diseases) was awarded a research grant for her project to investigate the role of neural progenitor cells in causing SMA pathology. This project will help uncover issues during early stages of development that are connected to problems with motor neurons and muscle cells in SMA.
- Maria Pennuto, Ph.D. (University of Padua) was awarded a research grant to explore two potential therapeutic targets that may alleviate SBMA (Kennedy's disease) pathology. The project aims to mitigate the harmful effects of the disease by targeting two specific proteins that are elevated in the disease and studying their impact on muscles and motor neurons. The long-term goal is to develop a clinical approach that can deliver treatments targeting these two proteins to the muscles using nanoparticles.
Collaboration grants among foundations play a crucial role in advancing research and therapeutic discoveries.
- MDA and AFM-Téléthon are partnering to support two research grants:
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- Dr. Eran Hornstein, M.D., Ph.D. (Weizmann Institute of Science) will investigate the potential use of microRNAs as biomarkers for phenoconversion in pre-familial ALS.
- Dr. Michele Brischigliaro, Ph.D. (Miller School of Medicine of the University of Miami) will explore aberrant mitochondrial-mRNA folding as a key mediator in mitochondrial encephalomyopathies.
- MDA and Coalition to Cure Calpain 3 are collaborating to fund a research grant for Dr. Melissa Spencer, Ph.D. (University of California Los Angeles). Her project aims to develop optimized transgenes and capsids for treating LGMD2A/R1, focusing on improving the safety and effectiveness of gene therapy for patients.
The MDA Development Grants support promising early-career researchers, fostering the next generation of leaders in neuromuscular disease research. This prestigious program has given rise to many of the top researchers in the field. The following are highlights from the MDA 2024 Development Trainee Awards to support promising early career researchers:
- Adam Bittel, D.P.T., Ph.D. (Children's Research Institute): Defining the Protective Effects of Estrogen in FSHD
- Michele Brischigliaro, Ph.D. (Miller School of Medicine - University of Miami): Aberrant mt-mRNA folding as mediator of mitochondrial encephalomyopathies
- Bradley Hamilton, Ph.D. (Stanford University School of Medicine): Addressing immunotoxicities to AAV Gene Therapy for DMD and Other Disorders
- Felipe de Souza Leite, Ph.D. (Boston Children's Hospital): Notch Pathway modulation in Satellite Cells of two novel mdx models
- Subodh Mishra, Ph.D. (Research Foundation of SUNY - University at Albany:) The mechanism, synergies, and therapeutic horizon of quercetin in DM and C9-ALS
- Hichem Tasfaout, Ph.D. (University of Washington): Development of next-generation therapies for Duchenne muscular dystrophy
- Gianvito Masi, M.D. (Yale University): Investigating mechanistic drivers of MuSK autoimmunity
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About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For nearly 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, TikTok, LinkedIn, and YouTube.
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CONTACT: Mary Fiance, National Vice President, Strategic Communications
Muscular Dystrophy Association
