TY - JOUR
T1 - A study on ensuring the quality and safety of pharmaceuticals and medical devices derived from the processing of autologous human somatic stem cells
AU - Hayakawa, Takao
AU - Aoi, Takashi
AU - Umezawa, Akihiro
AU - Ozawa, Keiya
AU - Sato, Yoji
AU - Sawa, Yoshiki
AU - Matsuyama, Akifumi
AU - Yamanaka, Shinya
AU - Yamato, Masayuki
N1 - Funding Information:
The authors would like to thank Dr. Kazuaki Kakehi (Kindai University, Japan), Dr. Hiroyuki Moriyama (Kindai University, Japan), and Dr. Satoshi Yasuda (National Institute of Health Sciences, Japan) for technical support. This work was supported by Research Grants H23-IYAKU-SHITEI-022 H25-JITSUYOKA(SAISEI)-IPPAN-008 and H26-IYAKUB-IPPAN-018 from the Japanese Ministry of Health, Labour and Welfare , and Research Grants 15mk0104009h0102 , 15bk0104014h0003 from Japan Agency for Medical Research and Development (AMED) .
Funding Information:
The authors would like to thank Dr. Kazuaki Kakehi (Kindai University, Japan), Dr. Hiroyuki Moriyama (Kindai University, Japan), and Dr. Satoshi Yasuda (National Institute of Health Sciences, Japan) for technical support. This work was supported by Research Grants H23-IYAKU-SHITEI-022 H25-JITSUYOKA(SAISEI)-IPPAN-008 and H26-IYAKUB-IPPAN-018 from the Japanese Ministry of Health, Labour and Welfare, and Research Grants 15mk0104009h0102, 15bk0104014h0003 from Japan Agency for Medical Research and Development (AMED).
Publisher Copyright:
© 2015 The Japanese Society for Regenerative Medicine
PY - 2015/12
Y1 - 2015/12
N2 - To make sure that novel human cell-based products contribute to human health care, it is essential that, based on sound science at present, suitable measures be taken by the manufacturers and regulatory authorities on applying these products to the treatment of patients by taking into account specificity of starting cell lines, the manufacturing process, products, administration procedures, diseases in question, and patient population. As part of such an endeavor, we studied scientific principles, concepts, and basic technical elements to ensure the quality and safety of therapeutic products derived from autologous human somatic stem cells, taking into consideration scientific and technological advances, ethics, regulatory rationale, and international trends in human stem cell-derived products. This led to the development of the Japanese official Notification No. 0907-2, “Guideline on Ensuring the Quality and Safety of Pharmaceuticals and Medical Devices Derived from the Processing of Autologous Human Somatic Stem Cells,” issued by Pharmaceuticals and Food Safety Bureau, Ministry of Health, Labour and Welfare of Japan on September 7, 2012. The present paper describes the background information and the development of our study and the resulting guidance. For products derived from autologous somatic stem cells, major points to consider include 1) multipotency and self-replication ability of autologous human somatic stem cells and differences in cell characteristics of the final products from those of the starting cells; 2) a donor's infectious status; 3) the risk of proliferation/reactivation of viruses during the manufacturing processes; 4) robust process control to minimize unevenness of “custom-made” products; 5) a limited amount of samples for quality evaluation of products; and 6) robust application and function of the final products in a cell environment different from where the original cells were localized and were performing their natural endogenous functions. The ultimate goal of this guidance is to provide suitable medical opportunities as soon as possible to the patients.
AB - To make sure that novel human cell-based products contribute to human health care, it is essential that, based on sound science at present, suitable measures be taken by the manufacturers and regulatory authorities on applying these products to the treatment of patients by taking into account specificity of starting cell lines, the manufacturing process, products, administration procedures, diseases in question, and patient population. As part of such an endeavor, we studied scientific principles, concepts, and basic technical elements to ensure the quality and safety of therapeutic products derived from autologous human somatic stem cells, taking into consideration scientific and technological advances, ethics, regulatory rationale, and international trends in human stem cell-derived products. This led to the development of the Japanese official Notification No. 0907-2, “Guideline on Ensuring the Quality and Safety of Pharmaceuticals and Medical Devices Derived from the Processing of Autologous Human Somatic Stem Cells,” issued by Pharmaceuticals and Food Safety Bureau, Ministry of Health, Labour and Welfare of Japan on September 7, 2012. The present paper describes the background information and the development of our study and the resulting guidance. For products derived from autologous somatic stem cells, major points to consider include 1) multipotency and self-replication ability of autologous human somatic stem cells and differences in cell characteristics of the final products from those of the starting cells; 2) a donor's infectious status; 3) the risk of proliferation/reactivation of viruses during the manufacturing processes; 4) robust process control to minimize unevenness of “custom-made” products; 5) a limited amount of samples for quality evaluation of products; and 6) robust application and function of the final products in a cell environment different from where the original cells were localized and were performing their natural endogenous functions. The ultimate goal of this guidance is to provide suitable medical opportunities as soon as possible to the patients.
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U2 - 10.1016/j.reth.2015.05.002
DO - 10.1016/j.reth.2015.05.002
M3 - Article
AN - SCOPUS:85045291571
VL - 2
SP - 57
EP - 69
JO - Regenerative Therapy
JF - Regenerative Therapy
SN - 2352-3204
ER -