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MDA funds creation of Limb-Girdle Muscular Dystrophy clinical research network to speed therapy development

$700,000 investment will support seven medical centers that specialize in LGMD research and clinical care

New York, July 27, 2018 — The Muscular Dystrophy Association today announces the award of an MDA clinical research network grant to Vice Chair of Research and Associate Professor of Neurology Nicholas Johnson, at Virginia Commonwealth University in Richmond, to establish the Limb-Girdle Muscular Dystrophy Clinical Research Network. The new grant, totaling more than $700,000 over two years, will facilitate the development of tools and infrastructure needed to efficiently and effectively conduct clinical trials and accelerate treatments for limb-girdle muscular dystrophy (LGMD).

Therapy development in LGMD can significantly benefit from rich natural history data to describe the clinical course of the different forms of LGMD, as well as from validated clinical outcome assessments which can be used to determine whether a drug or intervention has provided treatment benefit.

Through the coordinated activities and enhanced communication among this new network of LGMD clinics, Johnson and colleagues aim to standardize approaches and develop the clinical outcome assessments to be used in future clinical trials.

“MDA is pleased to support the development and work of the LGMD Clinical Research Network, which will put in place critical infrastructure for limb-girdle muscular dystrophy research and therapy development,” said MDA Scientific Program Officer Lianna Orlando, Ph.D. “The network is well-suited to address current challenges facing LGMD researchers and clinicians, and its work will improve clinical trial readiness in this group of diseases.”

An initial step in the development of the network infrastructure is to conduct a longitudinal study of candidate clinical outcome assessments and develop disease-specific patient reported outcomes. Investigators will initially focus on four different genetic LGMD subtypes and will collect DNA, genetic diagnosis and clinical information to help characterize the natural course of the disease.

“We believe that the absence of tools to measure disease progression is a major barrier to conducting drug trials in this underserved population,” Johnson said. “This is critically important given the exciting progress in gene therapy.

“We are focused on developing these tools, with a particular focus on those assessments that may be used across different LGMD types, and which assessments are unique to specific subtypes,” Johnson added. “Progress in these areas is best suited for a research network that is geographically distributed and includes investigators with varied expertise in clinical and laboratory methods.”

The network comprises seven medical centers with significant expertise in LGMD research and clinical care, spanning across the United States. Centers are:

• California: University of California-Irvine — Tahseen Mozaffar, M.D., site investigator
• Colorado: University of Colorado in Aurora — Matt Wicklund, M.D., site investigator
• Iowa: University of Iowa in Iowa City — Kathy Mathews, M.D., site investigator
• Massachusetts: Brigham and Women’s Hospital in Boston — Anthony Amato, M.D., site investigator
• Kansas: University of Kansas Medical Center in Kansas City — Jeff Statland, M.D., site investigator
• Missouri: Washington University in St. Louis — Conrad Weihl, M.D., Ph.D., site investigator
• Virginia: Virginia Commonwealth University in Richmond — Nicholas Johnson, M.D., principal investigator

MDA has funded more than $59 million in LGMD research since 1950, and including this most recent award, currently is funding 10 active LGMD grants, with a total funding commitment of more than $2.8 million. 

The new grant was approved by MDA’s Board of Directors after careful deliberations and analysis by MDA advisors and research staff. Currently, MDA is funding 177 research projects around the world, with another 28 pending.

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, Public Relations and Communications
Muscular Dystrophy Association
646-992-2908 c: 917.538.5895
mfiance@mdausa.org