Title:Quality Control Analysis of Mesenchymal Stem/Stromal Cells During
Investigational New Drug Application for GvHD Administration in China
Volume: 18
Issue: 8
Author(s): Aitong Wang, Leisheng Zhang, Meng Zhao and Hao Yu*
Affiliation:
- National Engineering Research Center of Cell Products, AmCellGene Engineering Co., Ltd., Tianjin, 300457, China
- Tianjin Key Laboratory of Engineering Technologies for Cell Pharmaceutical, Tianjin, 300457, China
- The Postdoctoral
Research Station, School of Medicine, Nankai University, Tianjin, 300071, China
Keywords:
UC-MSCs, GvHD, QC, clinical trials, HSCT, stem cell transplantation.
Abstract: Graft-versus-host disease (GvHD), including the acute and chronic types (aGvHD, cGvHD),
arise as the dominating secondary disease in patients with unsatisfying consequences of allogeneic hematopoietic
stem cell transplantation (HSCT). Approximately half of GvHD patients were steroid-resistant,
with a two-year overall survival rate lower than 20%. Worse still, there are no standardized criteria for an
optimal second-line therapy for steroid-resistant aGVHD patients. Notably, pioneering investigators have
highlighted the ameliorative or therapeutic effects of human umbilical cord-derived mesenchymal
stem/stromal cells (hUC-MSCs) upon GvHD largely attributed to their unique hematopoietic-supporting
and immunomodulatory properties. Of note, quality control (QC) is the prerequisite to assure the safety
and quality of hUC-MSCs before investigational new drug (IND) applications and large-scale clinical
applications. Herein, we summarize the state-of-the-art updates upon IND-associated QC and clinical
trials of hUC-MSCs during allogeneic HSCT in China. Meanwhile, the supervisory policy and medical
ethics of current licensed MSC products for GvHD administration and the concomitant opportunities and
challenges have also been discussed.
