Unique Course Trains Young Physicians and Researchers in Muscle Disorders and Therapies

Every year, more than 60 young trainees come to Nationwide Children’s Hospital for the annual Myology Course, an intensive, week-long introduction to muscle biology, disease and therapeutics.

Hosted by Nationwide Children’s and The Ohio State University, it is now in its 8^th year, and is unique in North America, according to Kevin Flanigan, MD, director of Nationwide Children’s Neuromusmuscular Program, and director of the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s.

Dr. Flanigan founded the course in 2011 to offer trainees an intensive introduction to neuromuscular disorders, and from year to year, about 80% of the content doesn’t change, covering much the same education in the basics of muscle health and neuromuscular disease.

The other 20%?

“The rest of the content reflects the leapfrogging advances of therapeutics for neuromuscular disorders,” Dr. Flanigan says.

A lot of advances are homegrown, says Jill Rafael Fortney, PhD, professor of Physiology and Cell Biology at OSU and course co-director. “For the past decade, about 25 labs from Nationwide Children’s and Ohio State have worked together as one center, the Center for Muscle Health and Neuromuscular Disorders, to study muscle diseases, and between our institutions we have moved more neuromuscular gene therapies to clinical trials than anyone else in the world.”

The course reflects the wide expertise of Nationwide Children’s and OSU faculty. Roughly half of the trainees each year are MDs in neurology or neuromuscular training programs, and the other half are pre- or postdoctoral PhD trainees, who work in basic science labs.

“The basic science training is what makes our five-day course truly unique among myology workshops,” Dr. Flanigan says.  All of the trainees attend morning lectures addressing basic muscle biology, clinical syndromes, and therapeutics, but then split up in the afternoon. PhD trainees choose elective workshops to learn basic wet lab techniques, while clinical trainees hear lectures on clinical care and clinical research, with visits from informative patients and families. They regroup for keynote presentations each evening.

The course has a growing national and international reputation, and has been filled each of the eight years. This year’s class came from institutions from Boston to California, as well as from France and Brazil.

Among this year’s highlighted speakers were Bertrand Fontaine, MD, PhD, director of the Institute of Myology in Paris, speaking on disorders of muscle channels, and Perry Shieh, MD, PhD, from UCLA, speaking on the very promising results for a new gene therapy for X-linked myotubular myopathy.

“Each year we are fortunate to have such prominent speakers in the field, which I think reflects the value our peers see in the course,” Dr. Flanigan says. “This year we had new faculty from Yale, as well as returning faculty from the University of Colorado, Columbia University, and the National Institutes of Health.”

Lee Sweeney, PhD, professor of pharmacology and therapeutics at the University of Florida College of Medicine, and director of its Myology Institute, has taught in the course every year since it began, giving a lecture on mechanisms of muscle contraction, and how genetic defects can result in disease.

Dr. Sweeney has also sent a dozen trainees to the course over the years.

“I think the myology course gives the most comprehensive background on the basics of muscle diseases of any course in the country,” Dr. Sweeney says.

And, providing the latest in gene therapy is a natural fit, he adds. “They’ve pioneered a lot in that area… No one has a more coherent and unified focus on developing gene therapies than Nationwide Children’s does.”

“From the point of view from our field, we have to train more neuromuscular specialists who are real experts and more PhD scientists who really understand the translational aspects, who view translation as a goal,” Dr. Flanigan says.

The course, which runs Aug. 22-28 in 2020, is free to all attendees. Parent Project Muscular Dystrophy and the Muscular Dystrophy Association fund the lion’s share of the program.

For more information, visit: https://www.nationwidechildrens.org/research/areas-of-research/center-for-gene-therapy/myology-course.