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

First Year FY2013
Center Regenerative medicine for spinal cord injury and stroke using neural precursor cells of iPS cell origin
Director of the center Hideyuki Okano
(Professor, School of Medicine, Keio University)
Subsidiary institution National Hospital Organization Osaka National Hospital, Dainippon Sumitomo Pharma Co., Ltd.
Summary The objective at this center is the early implementation of regenerative medicine to treat CNS diseases, using stocks of clinical-grade iPS cells for regenerative medicine produced at a core center for iPS cell research.
There are as yet no effective therapies for spinal cord injury or cerebral infarction, but there have been high hopes for regenerative medicine using stem cells. Research at this center will build up stocks of neural precursor cells of iPS cell origin for use in regenerative medicine and will establish methods for safety screening and procedures against post-transplantation neoplastic transformation by using these cells to analyze tumorigenicity. Clinical research will be undertaken on subacute spinal cord injury, implementing first-in-man studies within four years and following the MHLW's Guidelines on clinical research using human stem cells. The center will also use stocks of neural precursor cells of iPS cell origin for use in regenerative medicine to develop stem cell transplantation methods to treat chronic spinal cord injury and subacute and chronic cerebral infarction.
First Year FY2013
Center Development of cell replacement therapy using iPS cell-derived neural cells against Parkinson's disease and stroke
Director of the center Jun Takahashi
(Deputy Director and Professor, Center for iPS Cell Research and Application, Kyoto University)
Subsidiary institution RIKEN
Summary The aim of this center is to start cell replacement therapy using iPS cells against Parkinson’s disease and stroke, which urgently need a new treatment.
In Parkinson’s disease, previous clinical studies have demonstrated that the fetal cell transplantation improves Parkinsonian symptoms, but this method has not been widely adopted because of the issues with the quantity and quality of the donor cells. Therefore, we use iPS cells to overcome these issues. The first stage aims at the autologous transplantation to treat sporadic (non-familial) Parkinson’s disease patients, because it minimizes the possibilities of immune response and pathogenic infection. The second stage aims at allogenic transplantation using HLA-matched iPS cells derived from healthy persons. Taking advantage of these experiences, in the third stage, we will try to find a method to reconstruct the cortico-spinal tract of stroke patients suffering from motor paralysis.
First Year FY2013
Center Research and development center for clinical application of complex tissue formation technologies to restore visual function
Director of the center Masayo Takahashi
(Project Leader, Center for Developmental Biology, RIKEN)
Subsidiary institution Foundation for Biomedical Research and Innovation
Summary Research at this center is aimed at developing regenerative medicine for intractable retinal diseases such as retinitis pigmentosa, using the transplantation of photoreceptor cells from human pluripotent stem cell origin (human ES/iPS cells). Intractable retinal diseases are serious progressive diseases that result in vision loss. There are high expectations for treatment using photoreceptor cell transplants. Research to date at this center has developed technologies for 3D retina formation and technologies to produce neural retina sheets including highly pure and highly dense photoreceptor cells from human ES/iPS cells. In the current research, human ES/iPS cells will be used and various projects will be implemented to allow clinical applications in humans as soon as possible. The projects will include technologies to modify neural retina sheets for preclinical research, proof of concept through animal transplants, preparation and quality control methods for clinical-grade neural retina sheets for transplantation, functional evaluation in medium-sized animals, detailed analyses of safety and graft rejection in clinical studies, and focused and objective functional analysis in humans as well as evaluation of the methodology.
First Year FY2013
Center Center for the development of myocardial regenerative treatments using iPS cells
Director of the center Yoshiki Sawa
(Professor, Graduate School of Medicine, Osaka University)
Summary The objective of research at this center is to establish therapeutic methods using myocardial cell sheets of iPS cell origin to treat severe heart failure. There is a shortage of donors for heart transplants and problems with durability and device lag with cardiac assist devices; substitute treatments are hardly widespread. With the goal of developing myocardial regeneration as a complete cure for severe heart failure, this research will develop methods for the efficient differentiation and mass culture of myocardial cells from GMP-grade HLA-homozygous iPS cells produced at a core center for iPS cell research, and will demonstrate efficacy and safety. Then the research will establish therapeutic methods using myocardial cell sheets of iPS cell origin, will conduct first-in-man safety studies within 3 years of the start of research to treat ischemic cardiomyopathy using the myocardial cell sheets of iPS cell origin, and then proceed to clinical trials. The ultimate goal is to develop myocardial grafts of iPS cell origin and pacemaker cells of iPS cell origin, and broaden the indications to pediatric heart failure and dilated cardiomyopathy.
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
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  • JST Yamanaka iPS Cell Project
  • Development of Systems and Technologies for Advanced Measurement and Analysis