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. 2025 May 9;37(3):321-327.
doi: 10.4103/tcmj.tcmj_249_24. eCollection 2025 Jul-Sep.

Allogeneic human umbilical cord blood for acute ischemic stroke: Phase I clinical trial

Raymond Y Lo  1   2   3 , Yuncin Luo  1   4 , Shu-Cin Chen  2 , Jen-Hung Wang  5 , Chen-Yu Ko  6 , Ying-Jie Chen  6 , Yu-Chin Su  6 , Tong-Young Lee  6 , Jonas C Wang  7 , Shinn-Zong Lin  8
Affiliations

Allogeneic human umbilical cord blood for acute ischemic stroke: Phase I clinical trial

Raymond Y Lo et al. Tzu Chi Med J. .

Abstract

Objectives: Transplantation of human umbilical cord blood cells (hUCB) may enhance neuroprotection, and thus, the intravenous (IV) infusion of hUCB in patients with acute ischemic stroke (AIS) is being tested for its safety and efficacy.

Materials and methods: We conducted a 12-month, open-label, and single-center, phase I trial of hUCB treatment in AIS patients at the age of 45-80 years, with magnetic resonance imaging evidence of acute infarction in the internal carotid artery supplied territory and the National Institute of Health Stroke Scale (NIHSS) score between 6 and 18. Eligible participants received a single-dose IV infusion of hUCB followed by the two doses of mannitol infusion within 9 days after the onset of stroke symptoms. The primary endpoint was the incidence of adverse events (AEs) and the secondary endpoints were the changes in NIHSS, Barthel index (BI), and Berg Balance Scale (BBS) scores.

Results: Six patients (Male: Female = 3: 3) were enrolled with a mean age at 65.8 years. A total of 40 AEs occurred in six participants during this study, which included nine serious adverse events. Only transient erythema multiforme and hematuria were probably and possibly related to hUCB infusion, respectively. The mean NIHSS score was 11.5 at baseline and it significantly improved at 1, 3, 6, 9, and 12 months after treatment (mean change from baseline: -4.0, -5.3, -6.8, -7.0, and -7.3). The mean BI score was 22.5 at baseline and it significantly increased at 3 and 6 months after treatment (mean change from baseline: 26.7 and 42.5, respectively). The BBS score increased numerically but did not reach statistical significance. The changes in cytokine levels and spleen size were unremarkable.

Conclusion: The IV hUCB was safe and well tolerated in AIS patients, and the preliminary efficacy results demonstrated its therapeutic potential, supporting the conduct of a randomized, placebo controlled, phase II clinical trial in future.

Keywords: Acute ischemic stroke; Adverse events; Human umbilical cord blood; National Institute of Health Stroke Scale; Phase I clinical trial.

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Conflict of interest statement

Dr. Raymond Y. Lo, an editorial board member at Tzu Chi Medical Journal, had no role in the peer review process of or decision to publish this article. The other authors declared no conflicts of interest in writing this paper. This study was supported by StemCyte through the clinical project SCTW001, which included the provision of drugs and research funding. StemCyte had no direct involvement in the design, conduct, or reporting of this research, and the Principal Investigator (PI) maintained full independence in all aspects of the study. Chen-Yu Ko, Ying-Chieh Chen, Yu-Chin Su, Tong-Young Lee, and Chia-Tsung Wang are employees of StemCyte. The authors declare no conflicts of interest related to this work.

Figures

Figure 1
Figure 1
(a) The National Institute of Health Stroke Scale (NIHSS) scores at multiple visits after acute ischemic stroke. (b) Change from baseline in NIHSS scores over time. BL: Baseline, NIHSS: National Institute of Health Stroke Scale (score: 0–42). *Statistically significant compared to baseline, defined as P < 0.05
Figure 2
Figure 2
Diffusion-weighted magnetic resonance imaging (MRIs) at baseline and T2-FLAIR MRIs at multiple visits during 12-month follow-ups. Subject no. 114-06 withdrew before visit 11
See this image and copyright information in PMC

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