A study on ensuring the quality and safety of pharmaceuticals and medical devices derived from the processing of allogeneic human somatic stem cells

• Hayakawa, Takao

  Pharmaceutical Research and Technology Institute, Kindai University

• Aoi, Takashi

  Department of iPS Cell Applications, Graduate School of Medicine, Kobe University・Center for Human Resource Development for Regenerative Medicine, Kobe University Hospital

• Umezawa, Akihiro

  Department of Reproductive Biology, National Research Institute for Child Health and Development, Japan

• Ozawa, Keiya

  Division of Genetic Therapeutics, The Advanced Clinical Research Center, The Institute of Medical Science, The University of Tokyo

• Sato, Yoji

  Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan

• Sawa, Yoshiki

  Division of Cardiovascular Surgery, Department of Surgery, Osaka University Graduate School of Medicine

• Matsuyama, Akifumi

  R&D Division of Regenerative Medicine, Foundation for Biomedical Research and Innovation, Japan

• Yamanaka, Shinya

  Center for iPS Cell Research and Application, Kyoto University

• Yamato, Masayuki

  Advanced Biomedical Science Center, Tokyo Women's Medical University

Abstract

As a series of endeavors to establish suitable measures for the sound development of regenerative medicine using human stem cell-based products, we studied scientific principles, concepts, and basic technical elements to ensure the quality and safety of therapeutic products derived from allogeneic 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. 0907-3, “Guideline on Ensuring the Quality and Safety of Pharmaceuticals and Medical Devices Derived from the Processing of Allogeneic 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 development of our study and the resulting guidance. For products derived from allogeneic somatic stem cells, major points to consider include 1) history, the source, and derivation of starting cells; 2) donor screening/testing and donor eligibility, especially in relation to the presence of adventitious agents, potential occurrence of donor-derived diseases, and immunocompatibility; 3) clinical records of a donor; 4) multipotency and self-replication ability of allogeneic human somatic stem cells; 5) cell banking; 6) potential presence of viruses in the final product; 7) extensive characterization of the cells at critical stage(s) of manufacture; 8) robustness of the manufacturing process; 9) quality consistency of the products such as the final products and critical intermediate(s) if any; and 10) 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 function. The ultimate goal of this guidance is to provide suitable medical opportunities as soon as possible to the patients with severe diseases that are difficult to treat with conventional modalities.

Journal

• Regenerative Therapy

  Regenerative Therapy 2 70-80, 2015-12

  Elsevier BV

Keywords

• Allogeneic human somatic stem cells
• Quality and safety of pharmaceuticals and medical devices
• Regenerative medicine
• Human stem cell-based products

Details

• CRID: 1050001335857571584
• NII Article ID: 120006471143
• ISSN: 23523204
• HANDLE: 2433/231220
• Text Lang: en
• Article Type: journal article
• Data Source:
  • IRDB
  • CiNii Articles
  • KAKEN