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Project Cure SMA is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.

Primary Objective
The primary objective of this Phase II trial is to assess the efficacy of oral valproic acid (VPA) in adults with SMA.

Inclusion Criteria

1. Confirmed diagnosis of 5q SMA through standard genetic testing
2. Ambulatory SMA 3 adults: all subjects must be able to walk independently for at least 30 feet without assistance (i.e., no canes, walkers)
3. Age 18 to 60 years at time of enrollment
4. Interest in participating and the ability to meet the study requirements
5. Women of child bearing age are required to be on birth control or abstain while participating in the study.

Exclusion Criteria

1. Non-ambulatory type 3 adults and all type 2 adults
2. Patients with co-existing medical conditions that preclude travel, testing or study medications
3. Patients who have participated in a treatment trial for SMA in the 3 months prior to this trial, or plan on enrolling in any other treatment trial during the duration of this trial.
4. Patients who are, in the investigator’s opinion, mentally or legally incapacitated from providing informed consent for the study, or are otherwise unable to meet study requirements or cooperate reliably with study procedures, especially strength testing.
5. Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day
6. Use of medications or supplements which interfere with VPA metabolism and increase the potential risks of the medications, or are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders within 3 months of study enrollment
7. Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.

Contact Information
John T. Kissel, MD, Professor of Neurology and Director, Division of Neuromuscular Disease at The Ohio State University Medical Center. If you are interested in participating and meet the above criteria, please contact Sharon Chelnick, Study Coordinator, at (614) 293-4973 or sharon.chelnick@osumc.edu.