MDA Awards Human Clinical Trial Grant for Myotonic Dystrophy Natural History Study
Funding to Nicholas Johnson will advance clinical trial preparedness
CHICAGO, August 30, 2017 – The Muscular Dystrophy Association (MDA) is pleased to announce the award of a human clinical trial grant to Nicholas Johnson, M.D., assistant professor of neurology, pediatrics and pathology at the University of Utah in Salt Lake City, to conduct a natural history study in congenital myotonic dystrophy (congenital DM1). The award, which totals $598,348 over three years, reflects MDA’s commitment to support research that can improve and accelerate the development of future clinical trials, with the ultimate goal being the development of treatments and cures for all the diseases in its program.
Congenital DM1 is the most severe form of myotonic dystrophy type 1, with onset of symptoms at birth that include weakness, breathing problems, feeding problems and clubfoot. During childhood, children often have intellectual impairment, fatigue, behavioral concerns and weakness. Currently, there are no available treatments.
Johnson will collect measurements in children with congenital DM1 of strength, cognition and quality of life, measured over the course of years, in order to determine how the disease progresses over time and why the symptoms of children with the disease differ from those of adults. The aim is to determine which of those measurements could best be used as meaningful clinical endpoints for future clinical trials in congenital DM1.
“MDA is excited to be able to support Dr. Johnson’s work to advance our understanding of how congenital DM1 progresses through childhood,” said MDA Scientific Program Officer Lianna Orlando, Ph.D. “Determining effective endpoint measures that reliably change over the course of the disease, and could potentially be used to indicate whether interventions or treatments are having any beneficial effects, is an important next step towards being able to conduct clinical trials in this patient population.”
Making it possible for kids with congenital DM1 to participate in clinical trials is a necessary step in finding effective therapies for the disease.
“Many of the symptoms experienced by children with DM1 are distinct from those experienced by adults with the disease,” Johnson explained. “With an improved understanding of these differences, there will be a lot of opportunities to identify new treatment targets.
“The long-term goal,” he noted, “is to reduce the severity of symptoms for children with this disease.”
MDA has funded more than $47 million in myotonic dystrophy research since 1950. Including a clinical research network grant announced in January 2017 to continue support of the Myotonic Dystrophy Clinical Research Network, three DM research grants announced in March, and a clinical research training fellowship to support studies on the molecular processes that drive DM, MDA currently is funding 10 active DM grants with a total funding commitment of $3.2 million.
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.
Roxan Triolo Olivas
MDA Vice President Public Relations and Community Programs