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Perspectives on Clinical Trials in Spinal Muscular Atrophy

Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah, swoboda{at}genetics.utah.edu

John T. Kissel, MD

Department of Neurology, Ohio State University School of Medicine, Columbus, Ohio

Thomas O. Crawford, MD

Departments of Neurology and Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland

Mark B. Bromberg, MD, PhD

Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah

Gyula Acsadi, MD, PhD

Departments of Neurology and Pediatrics, Wayne State University School of Medicine, Detroit, Michigan

Guy D'Anjou, MD

Division of Pediatric Neurology, Hôpital Sainte-Justine Montréal, Montréal, Québec, Canada

Kristin J. Krosschell, MA, PT

Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine, Northwestern University, Chicago, Illinois

Sandra P. Reyna, MD

Departments of Neurology and Pediatrics, University of Utah School of Medicine, Salt Lake City, Utah

Mary K. Schroth, MD

Department of Pediatrics, The University of Wisconsin School of Medicine, Madison, Wisconsin

Charles B. Scott, PhD

Louise R. Simard, PhD

Departments of Biochemistry and Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada

Spinal muscular atrophy is one of the most heterogeneous of the single-gene neuromuscular disorders. The broad spectrum of severity, with onset from the prenatal period to adulthood, presents unique challenges in the design and implementation of clinical trials. The clinical classification of subjects into severe (type 1), intermediate (type 2), and mild (type 3) subtypes has proved useful both in enhancing communication among clinicians internationally and in forging the collaborative development of outcome measures for clinical trials. Ideally, clinical trial design in spinal muscular atrophy must take into account the spinal muscular atrophy type, patient age, severity-of-affection status, nature of the therapeutic approach, timing of the proposed intervention relative to disease progression, and relative homogeneity of the cohort to be studied. Following is an overview of the challenges and opportunities, current and future therapeutic strategies, and progress to date in clinical trials in spinal muscular atrophy.

Key Words: spinal muscular atrophy • clinical trials • histone deacetylase inhibitors

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