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Trial Suggests Safety and Efficacy of Endothelial Progenitor Cell Therapy in Chronic Kidney Disease

Review of “Safety and Efficacy of Intra-Renal Arterial Autologous CD34+ Cell Transfusion in Patients with Chronic Kidney Disease: A Randomized, Open-Label, Controlled Phase II Clinical Trial” from STEM CELLS Translational Medicine by Stuart P. Atkinson

Previous preclinical research from the laboratories of Hon‐Kan Yip (Kaohsiung Chang Gung Memorial Hospital and Chang Gung University College of Medicine, Kaohsiung, Taiwan) demonstrated that peripheral blood-derived CD34-positive endothelial progenitor cell (EPC) therapy preserved residual renal function in rats with chronic kidney disease by enhancing angiogenesis and blood flow and suppressing oxidative stress, inflammation, and fibrosis [1]. Chronic kidney disease remains a significant and growing public health problem worldwide [2-5] and can lead to the development of end‐stage renal disease [2, 4].

A subsequent phase I clinical trial using autologous CD34-positive EPCs to treat human patients with stage III and IV chronic kidney disease provided proof of safety [6], and now, the team returns with a new study describing the results of their phase II clinical trial that aimed to prove the efficacy of this approach in preserving renal function and improving clinical outcomes in chronic kidney disease patients.

Reporting in STEM CELLS Translational Medicine [7], Yang et al. undertook a randomized, open‐label, controlled phase II clinical trial to investigate the safety, efficacy, and outcomes of intrarenal artery infusion of autologous peripheral‐blood‐derived CD34-positive EPCs for patients with chronic kidney disease (stage III or IV).

  • 52 patients were randomly allocated into a treatment group and a control group
  • Primary endpoints included safety and change of creatinine level/creatinine clearance, while the secondary endpoints included 12‐month combined unfavorable clinical outcomes, improvement in proteinuria, and CD34-positive EPC‐related adverse events
  • CD34-positive EPCs were isolated by leukapheresis from the peripheral blood of patients following subcutaneous administration of granulocyte‐colony stimulating factor
  • All patients were discharged after autologous CD34-positive EPC cell therapy with no significant problems noted
  • One‐year combined unfavorable clinical outcomes (dialysis or death) were significantly lower in the CD34-positive EPC-treated patients when compared to the control group
  • However, circulating creatinine level, the ratio of urine protein to urine creatinine, and creatinine clearance showed no difference between the CD34-positive EPC-treated patients when compared to the control group at 12 months

Overall, this first phase II clinical trial designed to address the therapeutic impact of CD34-positive EPCs on patients with chronic kidney disease at stage III and IV suggests safety and efficacy. The authors do note that the small sample size, the lack of long‐term follow‐up, and the lack of a true double‐blinded design represent some of the limitations of this study.

For more on how CD34-positive EPCs may represent the way forward for the treatment of chronic kidney disease, stay tuned to the Stem Cells Portal!


  1. Huang TH, Chen YT, Sung PH, et al., Peripheral blood-derived endothelial progenitor cell therapy prevented deterioration of chronic kidney disease in rats. American Journal of Translational Research 2015;7:804-24.
  2. Hsu C-C, Hwang S-J, Wen C-P, et al., High Prevalence and Low Awareness of CKD in Taiwan: A Study on the Relationship Between Serum Creatinine and Awareness From a Nationally Representative Survey. American Journal of Kidney Diseases 2006;48:727-738.
  3. Jayatilake N, Mendis S, Maheepala P, et al., Chronic kidney disease of uncertain aetiology: prevalence and causative factors in a developing country. BMC Nephrology 2013;14:180.
  4. Kuo H-W, Tsai S-S, Tiao M-M, et al., Epidemiological Features of CKD in Taiwan. American Journal of Kidney Diseases 2007;49:46-55.
  5. Lunyera J, Mohottige D, Isenburg MV, et al., CKD of Uncertain Etiology: A Systematic Review. Clinical Journal of the American Society of Nephrology 2016;11:379.
  6. Lee MS, Lee FY, Chen YL, et al., Investigated the safety of intra-renal arterial transfusion of autologous CD34+ cells and time courses of creatinine levels, endothelial dysfunction biomarkers and micro-RNAs in chronic kidney disease patients-phase I clinical trial. Oncotarget 2017;8:17750-17762.
  7. Yang C-C, Sung P-H, Cheng B-C, et al., Safety and efficacy of intrarenal arterial autologous CD34+ cell transfusion in patients with chronic kidney disease: A randomized, open-label, controlled phase II clinical trial. STEM CELLS Translational Medicine 2020;9:827-838.