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JST Home > Research Center Network for Realization of Regenerative Medicine > Research Centers/Projects > Centers for Clinical Application Research on Specific Disease/Organ【Type A】

Centers for Clinical Application Research on Specific Disease/Organ
【Type B】

First Year FY2013
Center Center for development of mucosal regenerative therapies for inflammatory bowel diseases using cultured intestinal epithelial stem cells
Director of the center Mamoru Watanabe
(Professor, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University)
Subsidiary institution Keio University
Summary At this center, intestinal epithelial regenerative therapies for the treatment of refractory inflammatory bowel diseases (ulcerative colitis and Crohn’s disease), are to be developed by integrating unique techniques such as intestinal epithelial stem cell culture, their in vivo transplantation, and therapeutic gastrointestinal endoscopy. At present, the number of inflammatory bowel disease patients in Japan reaches up to over 170,000. The overall prevalence of these diseases, as well as the number of severely refractory patients, are both increasing rapidly and continuously. Regeneration of the mucosal epithelium is highly important for the successful treatment of these patients, and the development of therapeutic approaches that can promote rapid regeneration of the intestinal epithelium is critically required. Thus the aims of this center are directed to establish intestinal epithelial regenerative therapies for inflammatory bowel diseases:
  1. Development of a highly efficient intestinal epithelial stem cell culture method using endoscopic biopsy tissues.
  2. Establishment of a safe, and also effective, endoscopic intestinal epithelial stem cell transplantation techniques, by using animal models.
First Year FY2013
Center Center for development of innovative technologies for metabolic organs using induced pluripotent stem (iPS) cells
Director of the center Hideki Taniguchi
(Professor, Graduate School of Medicine, Yokohama City University)
Subsidiary institution National Center for Child Health and Development, Kuraray Co.,  Ajinomoto Co., Sekisui Medical Co.
Summary The aim of this center is to develop organ creation methods using human iPS cells, and thus to realize regenerative medical techniques for treating liver failure caused by refractory liver disease. Liver failure is a life-threatening disease, and the only life-saving procedure currently available is liver transplantation. However, there is clearly a worldwide shortage of donated organs, highlighting the urgent needs for developing new regeerative technology to grow human organs from iPS cells. At this center, an innovative three-dimensional culture technique, i.e. a method for creating liver organoids (liver buds), was established by mimicking the early organogenesis, making it possible to generate functional liver from human iPS cells by way of liver bud transplantation. The aims of the present research, by means of bulk production of human iPS-cell-derived liver buds for preclinical research, quality evaluation, development of transplantation techniques, achieving approval of the proof of concept, etc., are to establish manufacturing processes for clinical-grade transplantable human liver buds, and to achieve clinical application of these techniques to liver failure patients as soon as possible.
First Year FY2013
Center Center for development of cancer immunotherapy technology by regenerating natural killer T-cells (NKT cells)
Director of the center Haruhiko Koseki
(Group Director, Center for Integrative Medical Sciences, RIKEN)
Subsidiary institution Chiba University, Keio University, National Hospital Organization Clinical Research Center
Summary The aims of this center are to develop techniques for regenerating NKT cells using induced pluripotent stem (iPS) cells, and clinically apply iPS-cell-derived NKT cells for cancer therapy.
Because of strong anti-tumor activity of NKT cells, cancer therapy by using activated NKT cells has already been approved as Advanced Medicine in Japan and clinical trials have started. NKT-cell-targeted therapy, however, is restricted to a minority of patients, because often cancer patients suffer decrease in both the number and functional potency of NKT cells.
The present research is aimed to develop technology for induction and amplification of functionally competent NKT cells from iPS cells to supplement NKT cell pool in cancer patients. To date, we have shown proof of concepts by using model animals that iPS-cell-derived NKT cells elicit sufficient anti-tumor activity. During the technology-development phase of this project, spanning 3 years, we will establish standard protocols to generate iPS cells from human NKT cells and redifferentiate NKT-derived iPS cells into a sufficient number of NKT cells for clinical use. The technology-development phase will be followed by preclinical studies to verify the efficacy and safety of cell therapies using iPS-cell-derived NKT cells. After preclinical trials, we are planning to apply these cells to clinical tests targeting head and neck tumors and advanced lung cancer.
First Year FY2013
Center Center for development of regenerative therapies for cartilage diseases using induced pluripotent stem (iPS) -cell-derived chondrocytes
Director of the center Noriyuki Tsumaki
(Professor, Center for iPS Cell Research and Application, Kyoto University)
Subsidiary institution Osaka University, University of Tokyo
Summary The aims of this center are to induce differentiation of chondrocytes from iPS cell stocks, and to achieve regenerative medical treatments for articular cartilage injuries and congenital facial cartilage defects. Autologous chondrocyte transplantation is currently used to treat articular cartilage injuries and facial cartilage defects, but the number and quality of the transplanted cells are not entirely satisfactory, and this technique has not entered widespread use. In addition, for large-size cartilage preparation, it is necessary to develop new scaffolds. In the present research, methods for inducing differentiation of high-quality chondrocytes from iPS cell stocks are to be developed. The efficacy and safety of chondrocytes differentiated from iPS cells are to be verified; methods for lyophilized storage of these cells are to be developed; the efficacy of chondrocytes derived from iPS cells homozygous for human leukocyte antigen is to be confirmed; and preclinical studies are to be carried out using an animal model of cartilage defects. After carrying out clinical studies with localized articular cartilage defects, the next step will be to carry out clinical studies with more severe articular cartilage defects and congenital facial cartilage defects, with the objective of realizing iPS-cell-derived chondrocyte transplantation methods for treating cartilage diseases.
First Year FY2013
Center Center for development of next-generation pancreatic islet transplantation methods based on induced pluripotent stem (iPS) cell technology
Director of the center Atsushi Miyajima
(Professor,Institute of Molecular and Cellular Biosciences, The University of Tokyo)
Subsidiary institution National Center for Global Health and Medicine, Central Institute for Experimental Animals, Kaneka Corp.
Summary At this center, novel therapeutic methods for insulin-dependent diabetes are to be developed. Pancreatic islet transplantation is a radical treatment for insulin- dependent diabetes, but it has several major disadvantages such as a shortage of donors, and continuous treatment with immunosuppressive agents, resulting in serious adverse effects and high financial costs. During the technology-development phase, we aim to establish methods for efficient induction of functional islets from iPS cells and adapting them to large-scale culture. Furthermore, transplantation technologies using novel polymer membranes for the immune-encapsulaion of islets are to be developed in order to avoid treatment with immunosuppressive agents. In the final phase, the aims will be to establish facilities for production of clinical-grade pancreatic islets, to evaluate the safety and efficacy of pancreatic islet grafting, and to initiate clinical research as quickly as possible.
Research Center Network for Realization of Regenerative Medicine
Japan Agency for Medical Research and Development(AMED)
Regenerative Medicine Division,
Department of Strategy Promotion

1-7-1 Otemachi, Chiyoda-ku,Tokyo, 100-0004 Japan
Phone: +81-3-6870-2220
  • Brochure
  • iPS Trend
  • JST Yamanaka iPS Cell Project
  • Development of Systems and Technologies for Advanced Measurement and Analysis