Background: This is a first-in-human, open-label, single-arm, dose-escalation study (NCT04097652) using UMC119-06 from Meridigen Biotech Co., Ltd., a mesenchymal stem cell derived from human umbilical cord, aimed at treating acute ischemic stroke (AIS). The study includes two dosage cohorts, with doses of 1×10^6 cells/kg and 5×10^6 cells/kg. The primary objective is to assess the safety, tolerability, and maximum feasible dose of UMC119-06 in AIS patients, while the secondary objective is to evaluate long-term safety and clinical efficacy after a single administration.
Method: The study enrolled six adult AIS patients, with safety assessments conducted across both dosage cohorts.
Result: UMC119-06 demonstrated positive outcomes through improvements in the modified Rankin Scale (mRS), the National Institutes of Health Stroke Scale (NIHSS), the Barthel Index (BI), and brain MRI infarct volume. The favorable improvements indicating improvements in functional outcomes among AIS patients treated with UMC119-06. Additionally, biomarkers such as IL-6, TNF-α, VEGF, and HGF showed changes indicating the potential impact of UMC119-06. In terms of safety, UMC119-06 demonstrated a well-tolerated safety profile. None of the AEs were classified as possibly, probably, or definitely related to UMC119-06.
Conclusion: UMC119-06 demonstrated good safety and potential efficacy in this Phase I study, but larger-scale, controlled studies are necessary to confirm its potential as a treatment option for AIS.