Korean J Physiol Pharmacol.  2016 Jan;20(1):63-67. 10.4196/kjpp.2016.20.1.63.

Allogeneic clonal mesenchymal stem cell therapy for refractory graft-versus-host disease to standard treatment: a phase I study

  • 1Department of Internal Medicine, Inha University Hospital, Inha University School of Medicine, Incheon 22332, Korea.
  • 2Department of Internal Medicine, Incheon St. Mary's Hospital, The Catholic University of Korea, Incheon 21431, Korea.
  • 3Department of Internal Medicine, Seoul National University Hospital, Seoul National University School of Medicine, Seoul 03080, Korea.
  • 4Department of Internal Medicine, Asan Medical Center, University of Ulsan College of Medicine, Seoul 05505, Korea.
  • 5Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul 06591, Korea.
  • 6Translational Research Center, Inha University School of Medicine and SCM Lifescience Co., Ltd., Incheon 22332, Korea. sunuksong@inha.ac.kr


Severe graft-versus-host disease (GVHD) is an often lethal complication of allogeneic hematopoietic stem cell transplantation (HSCT). The safety of clinical-grade mesenchymal stem cells (MSCs) has been validated, but mixed results have been obtained due to heterogeneity of the MSCs. In this phase I study, the safety of bone marrow-derived homogeneous clonal MSCs (cMSCs) isolated by a new subfractionation culturing method was evaluated. cMSCs were produced in a GMP facility and intravenously administered to patients who had refractory GVHD to standard treatment resulting after allogeneic HSCT for hematologic malignancies. After administration of a single dose (1x10(6) cells/kg), 11 patients were evaluated for cMSC treatment safety and efficacy. During the trial, nine patients had 85 total adverse events and the rate of serious adverse events was 27.3% (3/11 patients). The only one adverse drug reaction related to cMSC administration was grade 2 myalgia in one patient. Treatment response was observed in four patients: one with acute GVHD (partial response) and three with chronic GVHD. The other chronic patients maintained stable disease during the observation period. This study demonstrates single cMSC infusion to have an acceptable safety profile and promising efficacy, suggesting that we can proceed with the next stage of the clinical trial.


Bone marrow; Graft-versus-host disease; Hematopoietic stem cell transplantation; Mesenchymal stem cells
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