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September 15, 2010

Rochester leads international effort to improve muscular dystrophy treatment

$11 million in funding supports 5-year study to compare treatments

A large international study aimed at improving the care of muscular dystrophy patients worldwide is being launched by physicians, physical therapists, and researchers at the Medical Center.

Neurologist Robert “Berch” Griggs is heading the study of treatments for Duchenne muscular dystrophy, the most common form of the disease that affects children. The condition, which affects boys almost exclusively, progresses rapidly. Boys’ symptoms start when they are toddlers; untreated, they end up in a wheelchair before they become teenagers. With today’s best treatments, the disease, which affects about 28,000 boys and young men in the United States, is often fatal by the time a patient reaches his 20s or early 30s.

Griggs, a professor of neurology, medicine, pediatrics, pathology, and laboratory medicine and a member of the Center for Human Experimental Therapeutics, calls the current treatment landscape for the disease “chaotic.” Recently he has identified 29 different treatment regimens in use by doctors around the world.

The new study is designed to eliminate the chaos. With at least $11 million in funding from the National Institute of Neurological Disorders and Stroke, Griggs and co-leader Kate Bushby, Action Research Professor of Neuromuscular Genetics at Newcastle University in the United Kingdom, and investigators at 41 other institutions around the world will study the three treatments most commonly used today. The study will include 300 boys ages 4 through 7 throughout North America and Europe. Recruiting for the study will begin in the summer of 2011. Study funding is slated to reach close to $15 million.

In the study, patients will be randomly assigned to receive one of three steroid treatments: a) prednisone every day; b) prednisone every day for 10 days, alternating with 10-day periods without the drug; c) daily use of a steroid known as deflazacort, which is approved for use in Europe but not the United States. Neither the patients nor the researchers will be aware of the patients’ treatment assignments until the conclusion of the trial.

Children will be evaluated every six months for three to five years. Researchers will measure the breathing capacity of the patients, which is often an indicator of how long a patient will live; they will ask patients and parents how satisfied they are with the treatment; and they will measure how long it takes patients to stand up after lying down, which is an indicator of how long a patient will be able to walk.

The study brings together two worldwide groups of physicians who specialize in the study of muscle disorders. The Muscle Study Group, with nearly three dozen sites in the United States, Canada, and Europe, is based in Rochester and headed by Griggs. The TREAT-NMD study group is a consortium of dozens of institutions across Europe and elsewhere that work together to study neuromuscular disorders.

Bushby and Griggs are leading the clinical aspects of the new study. Michael McDermott, a professor of biostatistics and neurology, and Rabi Tawil, a professor of neurology, are coleaders of biostatistical analysis and data management.

The team from Rochester also includes Barbara Herr, an assistant professor of neurology, manager of the U.S. portion of the project; associate project manager Christine Annis; Shree Pandya, lead U.S. physical therapist; Mary Brown, Heart Research Program manager; William Martens, data manager; and Emma Ciafaloni, an associate professor of neurology, who will lead the study of patients in Rochester.

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