Therapeutic Application of Human Tonsil-Derived Mesenchymal Stem Cells for Peripheral Nerve Regeneration

adverse events (AE). Efficacy measures included changes in the “scale for the assessment and rating of ataxia (SARA)” score and sensory organization test (SOT) score. Results: All subjects completed the study visits. There was no acute or delayed AE that was related to the Stemchymal® infusion during the follow-up period. For efficacy, in the patients with SCA3 (n = 6), SARA scores decreased in one, remained unchanged in 3 and increased in 2 patients after 1-year follow-up. In the patient with MSA-C, a marked increase of SARA score was observed. All SCA3 subjects showed early improvement in their balance performance (6/ 6, 100%) after Stemchymal® treatment. Conclusions: Our data suggest safety in all patients and some short-term therapeutic effects of intravenous administration of Stemchymal® in patients with SCA3. To further investigate the therapeutic efficacy of Stemchymal® treatment for SCA3, a double-blind, placebo-controlled, multiple doses, phase II trial is in progress (NCT02540655). Assessments of disease progression by rating systems (ex. SARA) and quantitative systems will be performed (Figure 1).