A study on ensuring the quality and safety of pharmaceuticals and medical devices derived from the processing of autologous 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

• 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

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.

Journal

• Regenerative Therapy

  Regenerative Therapy 2 57-69, 2015-12

  Elsevier BV

Keywords

• Autologous human somatic stem cells
• Quality and safety of pharmaceuticals
• Human cell-based products

Details

• CRID: 1050564285810993792
• NII Article ID: 120006471144
• ISSN: 23523204
• HANDLE: 2433/231221
• Text Lang: en
• Article Type: journal article
• Data Source:
  • IRDB
  • CiNii Articles
  • KAKEN