Stem Cell Research & Therapy (May 2022)

Efficacy and safety of umbilical cord-derived mesenchymal stem cells in Chinese adults with type 2 diabetes: a single-center, double-blinded, randomized, placebo-controlled phase II trial

  • Li Zang,
  • Yijun Li,
  • Haojie Hao,
  • Jiejie Liu,
  • Yu Cheng,
  • Bing Li,
  • Yaqi Yin,
  • Qian Zhang,
  • Fei Gao,
  • Haibin Wang,
  • Shi Gu,
  • Jia Li,
  • Fengxiang Lin,
  • Yingfei Zhu,
  • Guanglei Tian,
  • Yulong Chen,
  • Weijun Gu,
  • Jin Du,
  • Kang Chen,
  • Qinghua Guo,
  • Guoqing Yang,
  • Yu Pei,
  • Wenhua Yan,
  • Xianling Wang,
  • Junhua Meng,
  • Saichun Zhang,
  • Jianming Ba,
  • Zhaohui Lyu,
  • Jingtao Dou,
  • Weidong Han,
  • Yiming Mu

DOI
https://doi.org/10.1186/s13287-022-02848-6
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 10

Abstract

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Abstract Background To determine the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs) in Chinese adults with type 2 diabetes mellitus (T2DM). Methods In this single-center, double-blinded, randomized, placebo-controlled phase II trial, 91 patients were randomly assigned to receive intravenous infusion of UC-MSCs (n = 45) or placebo (n = 46) three times with 4-week intervals and followed up for 48 weeks from October 2015 to December 2018. The primary endpoint was the percentage of patients with glycated hemoglobin (HbA1c) levels of < 7.0% and daily insulin reduction of ≥ 50% at 48 weeks. Additional endpoints were changes of metabolic control, islet β-cell function, insulin resistance, and safety. Results At 48 weeks, 20% of the patients in the UC-MSCs group and 4.55% in the placebo group reached the primary endpoint (p < 0.05, 95% confidence interval (CI) 2.25–28.66%). The percentage of insulin reduction of the UC-MSCs group was significantly higher than that of the placebo group (27.78% versus 15.62%, p < 0.05). The levels of HbA1c decreased 1.31% (9.02 ± 1.27% to 7.52 ± 1.07%, p < 0.01) in the UC-MSCs group, and only 0.63% in the placebo group (8.89 ± 1.11% to 8.19 ± 1.02%, p˃0.05; p = 0.0081 between both groups). The glucose infusion rate (GIR) increased significantly in the UC-MSCs group (from 3.12 to 4.76 mg/min/kg, p < 0.01), whereas no significant change was observed in the placebo group (from 3.26 to 3.60 mg/min/kg, p ˃ 0.05; p < 0.01 between both groups). There was no improvement in islet β-cell function in both groups. No major UC-MSCs transplantation-related adverse events occurred. Conclusions UC-MSCs transplantation could be a potential therapeutic approach for Chinese adults with T2DM. Trial registration This study was registered on ClinicalTrials.gov (identifier: NCT02302599).

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