World’s First Clinical Research Using iPS Cells

Japan Initiates the World’s First Clinical Research Using iPS Cells

The scientific research institute RIKEN Center for Developmental Biology and the Foundation for Biomedical Research and Innovation applied in February for government permission to conduct clinical research. In July the institutes secured the approval of Health, Labor and Welfare Minister Norihisa Tamura. This week they will launch a colaboration for the world’s first clinical research using induced pluripotent stem cells (iPS cells).

At the base of this clinical research project are the experiments conducted by the group of Megumu K. Saito (Center of iPS Research and Application, Kyoto University) and Tatsutoshi Nakahata (CiRA Kyoto University), who established a highly-efficient method for differentiation of human iPS cells into the immune cells such as dendritic cells and macrophages.


Retinal Pigment Epithelium (RPE) cells derived from human iPS cells

In this research project, epithelium grown from iPS cells will be transplanted into patient’s eyes for regeneration of damaged retina. Age-related macular degeneration (AMD) is the most common cause of visual impairment in the elderly, and affects up to 1% of people over 50 years of age in Japan. AMD is characterized by progressive damage to the retinal pigment epithelium, a protective layer of cells located adjacent to the photoreceptors at the back of the eye.

It is a major concern that the tissue grown from iPS cells might develop into cancer after transplantation. Therefore, preceding studies have focused, among others, on cell morphology, gene expression, and tumorigenic processes in animal models. In addition, the eye was chosen for first clinical research because it has a lower risk of cancer, compared to other organs.

The paper “Robust and Highly-Efficient Differentiation of Functional Monocytic Cells from Human Pluripotent Stem Cells under Serum- and Feeder Cell-Free Conditions” was published in the U.S. scientific journal PLOS ONE .

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