Clinical Outcomes of Transplanted Modified Bone Marrow–Derived Mesenchymal Stem Cells in StrokeA Phase 1/2a Study
Aha Journals 2016;47:1817-1824. DOI: 10.1161/STROKEAHA.116.012995
Gary K. Steinberg, MD, PhD; Douglas Kondziolka, MD; Lawrence R. Wechsler, MD; L. Dade Lunsford, MD; Maria L. Coburn, BA; Julia B. Billigen, RN, BS; Anthony S. Kim, MD, MAS; Jeremiah N. Johnson, MD; Damien Bates, MD, PhD; Bill King, MS; Casey Case, PhD; Michael McGrogan, PhD; Ernest W. Yankee, PhD; Neil E. Schwartz, MD, PhD
Background and Purpose—Preclinical data suggest that cell-based therapies have the potential to improve stroke outcomes. Methods—Eighteen patients with stable, chronic stroke were enrolled in a 2-year, open-label, single-arm study to evaluate the safety and clinical outcomes of surgical transplantation of modified bone marrow–derived mesenchymal stem cells (SB623).
Results—All patients in the safety population (N=18) experienced at least 1 treatment-emergent adverse event. Six patients experienced 6 serious treatment-emergent adverse events; 2 were probably or definitely related to surgical procedure; none were related to cell treatment. All serious treatment-emergent adverse events resolved without sequelae. There were no dose-limiting toxicities or deaths. Sixteen patients completed 12 months of follow-up at the time of this analysis. Significant improvement from baseline (mean) was reported for: (1) European Stroke Scale: mean increase 6.88 (95% confidence interval, 3.5–10.3; P<0.001), (2) National Institutes of Health Stroke Scale: mean decrease 2.00 (95% confidence interval, −2.7 to −1.3; P<0.001), (3) Fugl-Meyer total score: mean increase 19.20 (95% confidence interval, 11.4–27.0; P<0.001), and (4) Fugl-Meyer motor function total score: mean increase 11.40 (95% confidence interval, 4.6– 18.2; P<0.001). No changes were observed in modified Rankin Scale. The area of magnetic resonance T2 fluid-attenuated inversion recovery signal change in the ipsilateral cortex 1 week after implantation significantly correlated with clinical improvement at 12 months (P<0.001 for European Stroke Scale).
In this interim report, SB623 cells were safe and associated with improvement in clinical outcome end points at 12 months.