Allogeneic clonal mesenchymal stem cell therapy for refractory graft-versus-host disease to standard treatment: a phase I study.
10.4196/kjpp.2016.20.1.63
- Author:
Hyeon Gyu YI
1
;
Seung Ah YAHNG
;
Inho KIM
;
Je Hwan LEE
;
Chang Ki MIN
;
Jun Hyung KIM
;
Chul Soo KIM
;
Sun U SONG
Author Information
1. Department of Internal Medicine, Inha University Hospital, Inha University School of Medicine, Incheon 22332, Korea.
- Publication Type:Clinical Trial ; Original Article
- Keywords:
Bone marrow;
Graft-versus-host disease;
Hematopoietic stem cell transplantation;
Mesenchymal stem cells
- MeSH:
Bone Marrow;
Drug-Related Side Effects and Adverse Reactions;
Graft vs Host Disease*;
Hematologic Neoplasms;
Hematopoietic Stem Cell Transplantation;
Humans;
Mesenchymal Stromal Cells*;
Myalgia;
Population Characteristics
- From:The Korean Journal of Physiology and Pharmacology
2016;20(1):63-67
- CountryRepublic of Korea
- Language:English
-
Abstract:
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.
