Chemistry, manufacturing, and controls regulatory considerations for human pluripotent stem cell-derived cellular products
10.16438/j.0513-4870.2020-0171
- VernacularTitle:关于人多能干细胞来源细胞治疗产品药学评价的思考
- Author:
Jia-qi LU
1
Author Information
1. Center for Drug Evaluation, National Medical Products Administration, Beijing 100022, China
- Publication Type:Research Article
- Keywords:
human pluripotent stem cell;
cellular product;
manufacturing process;
quality study;
pharmaceutical evaluation
- From:
Acta Pharmaceutica Sinica
2020;55(10):2478-2485
- CountryChina
- Language:Chinese
-
Abstract:
In recent years, the number of clinical trials of stem cell products has increased, and the research and development technology and evaluation system have developed rapidly. Human pluripotent stem cell (hPSC)-derived cellular products are in the phase I/II stage of clinical trials. Related products include hPSC-derived neurons, retinal pigment epithelial cells, pancreatic beta cells, etc. They are generally used for the repair and replacement of functional cells related to degenerative diseases and genetic diseases via local transplantation. So far, no similar products have been officially approved on market. As hPSC possesses multi-directional differentiation potential and the ability to form teratoma in vivo, compared with other stem cell products, hPSC-derived cellular products have relatively higher risk of tumorigenicity, longer differentiation induction cycle, more complex production process, together with the rapidly updating quality characterization methods, which pose challenges to the scientific evaluation of their human applications. Based on the problems in the recent review and communication of clinical trial applications of stem cell products, and with reference to the relevant technical guidelines, this paper proposes the chemistry, manufacturing, and controls review considerations on the manufacturing process and quality study of hPSC-derived cellular products. We hope to improve the communications between developers and regulators.
