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. 2023 Dec 7;30(12):1585-1596.e6.
doi: 10.1016/j.stem.2023.11.004.

Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa

Yasuhiko Hirami  1 , Michiko Mandai  2 , Sunao Sugita  3 , Akiko Maeda  4 , Tadao Maeda  5 , Midori Yamamoto  5 , Hirofumi Uyama  6 , Satoshi Yokota  7 , Masashi Fujihara  7 , Masataka Igeta  8 , Takashi Daimon  8 , Kanako Fujita  9 , Tomoko Ito  9 , Naoki Shibatani  9 , Chikako Morinaga  10 , Tetsuya Hayama  11 , Aya Nakamura  12 , Kazuki Ueyama  12 , Keiichi Ono  12 , Hidetaka Ohara  13 , Masayo Fujiwara  11 , Suguru Yamasaki  11 , Kenji Watari  11 , Kiyoko Bando  13 , Keigo Kawabe  13 , Atsushi Ikeda  11 , Toru Kimura  14 , Atsushi Kuwahara  11 , Masayo Takahashi  15 , Yasuo Kurimoto  7
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Free article

Safety and stable survival of stem-cell-derived retinal organoid for 2 years in patients with retinitis pigmentosa

Yasuhiko Hirami et al. Cell Stem Cell. .
Free article

Abstract

Transplantation of induced pluripotent stem cell (iPSC)-derived retinal organoids into retinal disease animal models has yielded promising results, and several clinical trials on iPSC-derived retinal pigment epithelial cell transplantation have confirmed its safety. In this study, we performed allogeneic iPSC-derived retinal organoid sheet transplantation in two subjects with advanced retinitis pigmentosa (jRCTa050200027). The primary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year post-transplantation. The secondary endpoints were the safety of the transplantation procedure and visual function evaluation. The grafts survived in a stable condition for 2 years, and the retinal thickness increased at the transplant site without serious adverse events in both subjects. Changes in visual function were less progressive than those of the untreated eye during the follow-up. Allogeneic iPSC-derived retinal organoid sheet transplantation is a potential therapeutic approach, and the treatment's safety and efficacy for visual function should be investigated further.

Keywords: clinical study; full-field stimulus threshold testing; induced pluripotent stem cell; regenerative therapy; retinal organoid; retinitis pigmentosa; transplantation.

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Conflict of interest statement

Declaration of interests Y.H. reports that Kobe City Eye Hospital received a research grant from the Japan Agency for Medical Research and Development (AMED) (grant number: JP19bk0104082) and financial support from Sumitomo Pharma Co. Ltd. for this study. S.S., T.M., M.Y., and M.T. are employed by Vision Care, Inc. T.H., A.N., K.U., K.O., H.O., M. Fujiwara, S. Yamasaki, K.W., K.B., K.K., A.I., and A.K. are employed by Sumitomo Pharma Co. Ltd. T.K. is a board member of Sumitomo Pharma Co. Ltd. The authors are co-inventors on patent applications.

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