Case ReportsAllogeneic Mesenchymal Stem Cell Therapy Outcomes for Three Patients with Spinal Muscular Atrophy Type 1Villanova, Marcello MD, PhD; Bach, John Robert MDAuthor Information From the Neuromuscular Rehabilitation Unit, Ospedale Privato Accreditato Nigrisoli, Bologna, Italy (MV); and Department of Physical Medicine and Rehabilitation, Rutgers New Jersey Medical School, University Hospital, Newark, New Jersey (JRB). All correspondence and requests for reprints should be addressed to: John R. Bach, MD, Department of Physical Medicine and Rehabilitation, Rutgers New Jersey Medical School, University Hospital B-403, 150 Bergen St, Newark, NJ 07103. Financial disclosure statements have been obtained, and no conflicts of interest have been reported by the authors or by any individuals in control of the content of this article. Supplemental digital content is available for this article. Direct URL citations appear in the printed text and are provided in the HTML and PDF versions of this article on the journal’s Web site (www.ajpmr.com). American Journal of Physical Medicine & Rehabilitation: May 2015 - Volume 94 - Issue 5 - p 410-415 doi: 10.1097/PHM.0000000000000309 Buy SDC Metrics Abstract No effective medical treatment has been documented for spinal muscular atrophy; however, cellular, molecular, and preclinical studies suggest that allogenic mesenchymal stem cells may play a role. Three children with spinal muscular atrophy type 1 underwent multiple intrathecal and intravenous infusions of mesenchymal stem cells. Their pretreatment, treatment, and posttreatment physical function were quantitated by the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders scale for two patients and documented by video for all three. Infant Test of Neuromuscular Disorders values were 3 before treatment, 10 and 16 during treatment, and 0 and 10 seven and twelve months after treatment was discontinued, respectively. No adverse effects have been noted for at least 44 and 49 mos from onset of treatment, respectively. These data represent the first objective, quantifiable improvements in physical function for any treatment of spinal muscular atrophy. Although the benefits were lost when the therapy was withdrawn, this may be an initial step in establishing mesenchymal stem cells as a safe and effective treatment of spinal muscular atrophy. Copyright © 2015 Wolters Kluwer Health, Inc. All rights reserved.