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Muscular Dystrophy Association announces 34 new research grants totaling $9.9 million

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Credit: Muscular Dystrophy Association

NEW YORK, August 23, 2018 - The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

"This latest round of grants is impressive in terms of total funding, number of grants awarded and the diverse impact these grants will have in furthering neuromuscular disease research," said MDA President & CEO Lynn O'Connor Vos. "With each grant cycle we are getting closer to unlocking the mysteries of so many of these neuromuscular diseases and identifying therapeutic targets that will lead to life-changing treatments, which is very exciting."

Of note in this latest round of funding are two grants awarded to set up clinical research networks for facioscapulohumeral muscular dystrophy (FSHD) and limb-girdle muscular dystrophy (LGMD), respectively. Additionally, a research infrastructure grant was awarded to fund the development of computational tools that can integrate and analyze complex amyotrophic lateral sclerosis (ALS) data sets.

This latest round of funding also includes 24 research grants awarded to established, independent investigators; five development grants awarded to investigators at the beginning of their careers and who are on the brink of becoming independent investigators; one clinical trial travel grant to help alleviate the financial burden on individuals and families traveling to participate in clinical research; and one clinical research training scholarship to a physician in the early stage of their medical career who is interested in pursuing a career in clinical neuromuscular research.

For more than six decades, MDA has funded basic, translational and clinical research as it works to unlock the mechanisms of neuromuscular disease and enable scientists and clinicians to develop safe and effective therapies capable of changing lives. The power of MDA's research program lies in its big-picture approach that leverages insights and information resulting from research in one disease area to inform and advance discoveries and breakthroughs in others. Many of the new awards were made to leading scientists who are conducting innovative research targeted to make an impact across multiple diseases in MDA's program.

The latest round of research grants was approved by MDA's Board of Directors following careful deliberations and analysis by MDA's Research Advisory Committee, through which approximately 50 leading clinicians and scientists in volunteer roles oversee the peer-review process.

Vital research and services dollars come from generous MDA partners and supporters who organize, and donate to, community fundraising programs including the International Association of Fire Fighters (IAFF), CITGO Petroleum Corporation, Harley-Davidson Motor Co., Acosta, Albertsons Companies, Casey's General Stores, Circle K, The Kroger Company, National Association of Letter Carriers and Dutch Bros. Coffee.

"This latest round of grants is a testament to the advances in technology and drug discovery that have taken place in the last few years," said MDA Senior Vice President and Scientific Director Grace Pavlath, Ph.D. "The opportunity to fund groundbreaking research has never been greater, and MDA remains committed to achieving our ultimate goal of providing treatments and cures for our community."

MDA is currently funding 177 different research projects around the world with a combined investment of $47.3 million.

Notable awards from the latest grant period:

Melissa Spencer, Ph.D., professor of Neurology at the University of California at Los Angeles, was awarded an MDA Research Grant to develop nanoparticles for the treatment of neuromuscular disorders, including Duchenne muscular dystrophy (DMD). This technique is an alternative approach to the delivery of therapies based on gene editing, gene replacement and gene silencing. Nanoparticle-based delivery has the potential to be more accurate and effective than the adeno-associated virus (AAV) method currently in use in gene therapy trials today.

Francis Sverdrup, Ph.D., associate professor of Biochemistry and Molecular Biology at St. Louis University, was awarded an MDA Research Grant to study drugs targeting DUX4 expression in FSHD. DUX4 protein in skeletal muscle is considered the leading cause of muscle degeneration in FSHD. Sverdrup's aim will be to continue work on recently identified compounds to find the most effective way to suppress DUX4, which may one day lead to a treatment for FSHD.

Carlo Rinaldi, Ph.D., associate professor and clinician scientist at the University of Oxford in England, was awarded an MDA Development Grant to study how variants in the gene of the androgen receptor (AR) protein cause spinal-bulbar muscular atrophy (SBMA) and investigate whether a novel antisense oligonucleotide therapy approach can target these toxic variants. The results of the study will provide a better understanding of SBMA disease mechanisms, as well as develop a new therapy potentially capable of treating the disease.

Ludo Van Den Bosch, Ph.D., principal investigator at VIB in Leuven, Belgium, was awarded an MDA Research Grant to study the role of histone deacetylase inhibitors in mouse models as a potential therapeutic approach for ALS. In ALS patients, the motor axon is the most vulnerable compartment determining survival of the nerve cell and it is thought that by inhibiting histone deacetylse 6 (HDAC6), the motor axon can be preserved. Previous MDA-supported work by Dr. Van Den Bosch has highlighted HDAC6 as a potential therapy for Charcot-Marie-Tooth disease (CMT), currently under development by industry.

Francesco Muntoni, M.D., chair of Pediatric Neurology in the ICH Developmental Neurosciences Programme at the UCL GOS Institute of Child Health in London, England, was awarded an MDA Research Grant to identify novel gene mutations in congenital muscular dystrophy (CMD) and in congenital myopathy (CMY) using advanced genetic techniques. Muntoni's research will aim to establish the gene-causing mutation in individuals with these conditions whose initial gene sequencing was not conclusive.

Samya Chakravorty, Ph.D., postdoctoral research fellow at Emory University, was awarded an MDA Development Grant to study the functional resolution of a subset of unsolved cases from MDA's LGMD genetic testing platform. The goal is to investigate these gene variants to see if these cases are the result of multi-gene pathogenicity -- whether instead of one genetic defect, patients have multiple defects that are causing their symptoms.

Michio Hirano, M.D., professor of Neurology at Columbia University Irving Medical Center, was awarded an MDA Research Grant to conduct an open-label expanded access trial of deoxynucleoside therapy for thymidine kinase 2 (TK2) deficiency, a type of mitochondrial DNA depletion syndrome (MDS). To date, 15 individuals have been treated with this therapy Hirano developed via compassionate-use mechanisms. With this current funding, a uniform prospective study on the safety and efficacy of this treatment in TK2 patients will be conducted, and the results will inform the design of the pivotal clinical trial necessary to obtain FDA approval.

Investing in clinical research infrastructure

In this latest round of grants, MDA has strategically committed funding to invest in clinical research tools and infrastructure that are crucial to better understanding disease pathways and accelerating treatments in ALS, FSHD and LGMD.

MDA awarded two clinical research network grants for FSHD and LGMD. Each grant will support a network of MDA Care Centers with expertise in each disease's research and clinical care and is targeted to facilitate the development of the tools and infrastructure needed to conduct clinical trials and accelerate treatments. Jeffrey Statland, Ph.D., principal investigator at the University of Kansas Medical School, will head up the FSHD network and Nicholas Johnson, Ph.D., associate professor of Neurology at Virginia Commonwealth University, will lead the LGMD network.

Additionally, MDA awarded a Research Infrastructure Grant to Jeffrey Rothstein, M.D., Ph.D., at Johns Hopkins University School of Medicine to fund the development of new data analytics approaches for the Answer ALS research project. These analytics tools will support the integration and analysis of more than 20 trillion data points generated by Answer ALS, with the goal of enhancing understanding of ALS disease mechanisms and advancing the discovery of targets for effective therapy.

2018 summer grants materials

MDA announces 34 new research grants totaling $9.9 million, by Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Director

MDA Grants at a Glance

MDA has funded more than $1 billion in neuromuscular disease research since 1950. In just the last three years, four drugs have been approved by the FDA that can trace their origins directly to MDA funding. These successes illustrate the vibrancy of MDA's research program and the welcoming of a new era in treating neuromuscular diseases.

About the Muscular Dystrophy Association

MDA is leading the fight to free individuals -- and the families who love them -- from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.

Contact

Mary Fiance
Director of Public Relations and Communications
646-992-2908
mfiance@mdausa.org

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