June 2023
Volume 64, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2023
Induced pluripotent stem cell derived retinal organoid sheets for regenerative therapy in patients with end-stage retinitis pigmentosa
Author Affiliations & Notes
  • Michiko Mandai
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
    retinal regeneration, RIKEN BDR, Kobe, Hyogo, Japan
  • Yasuhiko Hirami
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Sunao Sugita
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Akiko Maeda
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • tadao Maeda
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Midori Yamamoto
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Hirofumi Uyama
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Satoshi Yokota
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Masashi Fujihara
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Masataka Igeta
    Biostatictics, Hyogo Ika Daigaku, Nishinomiya, Hyogo, Japan
  • Takashi Daimon
    Biostatictics, Hyogo Ika Daigaku, Nishinomiya, Hyogo, Japan
  • Kanako Fujita
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Tomoko Ito
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Masayo Takahashi
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Yasuo Kurimoto
    Kobe City Eye Hospital, Kobe, Hyogo, Japan
  • Footnotes
    Commercial Relationships   Michiko Mandai Sumitomo Pharma Co. Ltd., Code F (Financial Support); Yasuhiko Hirami Sumitomo Pharma Co. Ltd., Code F (Financial Support); Sunao Sugita Vision Care Inc., Code E (Employment); Akiko Maeda None; tadao Maeda Sumitomo Pharma Co. Ltd., Code F (Financial Support); Midori Yamamoto Vision Care Inc., Code E (Employment); Hirofumi Uyama None; Satoshi Yokota None; Masashi Fujihara None; Masataka Igeta None; Takashi Daimon None; Kanako Fujita Sumitomo Pharma Co. Ltd., Code F (Financial Support); Tomoko Ito Sumitomo Pharma Co. Ltd., Code F (Financial Support); Masayo Takahashi Vision Care Inc., Code E (Employment); Yasuo Kurimoto Sumitomo Pharma Co. Ltd., Code F (Financial Support)
  • Footnotes
    Support  AMED Japan Grant JP19bk0104082, Sumitomo Pharma Co., Ltd.
Investigative Ophthalmology & Visual Science June 2023, Vol.64, 4628. doi:
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      Michiko Mandai, Yasuhiko Hirami, Sunao Sugita, Akiko Maeda, tadao Maeda, Midori Yamamoto, Hirofumi Uyama, Satoshi Yokota, Masashi Fujihara, Masataka Igeta, Takashi Daimon, Kanako Fujita, Tomoko Ito, Masayo Takahashi, Yasuo Kurimoto; Induced pluripotent stem cell derived retinal organoid sheets for regenerative therapy in patients with end-stage retinitis pigmentosa. Invest. Ophthalmol. Vis. Sci. 2023;64(8):4628.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Retinitis pigmentosa is a hereditary retinal degenerative disease characterized by night blindness and progressive loss of visual field due to the degeneration of the retinal rod photoreceptor cells. Induced pluripotent stem cells (iPSC)-derived retinal organoid sheet showed a potential function after transplantation in animal models of retinal degereration. In this clinical study, allogenic iPSC-retinal organoid sheets were transplanted in the eye of 2 patients with end-stage rtinitis pigmentosa to study the safety and survival of the graft,

Methods : The primary endpoint was the survival and safety of the transplanted retinal organoid sheets in the first year after transplantation, and the secondary endpoints were the safety of the retinal sheet transplantation procedure and visual function evaluation. The first patient was a 67-year-old woman with a corrected visual acuity of hand motion who recieved three retinal organoid sheets near the macula of the right eye; the second patient was a 42-year-old man with a corrected visual acuity of 0.05 who recived three retinal organoid sheets near the macula of the right eye. The patients were followed-up for one year.

Results : The grafts survived in a stable condition in both patients through the first year after transplantation with the increased retinal thickness at the transplant site. No serious adverse events were observed in either case. The first case showed a partial improvement in visual function tests including full-field stimulus testing and Table Test.

Conclusions : Allogeneic iPSC-derived retinal organoid sheet transplantation is a safe and practical therapeutic approach. The treatment efficacy in terms of visual function should be further investigated.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.

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