September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
Transplantation of Autologous induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium Cell Sheets for Exudative Age Related Macular Degeneration : A Pilot Clinical Study

Author Affiliations & Notes
  • Yasuo Kurimoto
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
    Ophthalmology, Kobe City Medical Ctr Gen Hosp, Kobe, Hyogo, Japan
  • Yasuhiko Hirami
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
    Ophthalmology, Kobe City Medical Ctr Gen Hosp, Kobe, Hyogo, Japan
  • Masashi Fujihara
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
    Ophthalmology, Kobe City Medical Ctr Gen Hosp, Kobe, Hyogo, Japan
  • Chikako Morinaga
    Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, Japan
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
  • Midori Yamamoto
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
    Ophthalmology, Kobe City Medical Ctr Gen Hosp, Kobe, Hyogo, Japan
  • Kanako Fujita
    Clinical Support Unit, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
  • Sunao Sugita
    Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, Japan
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
  • Michiko Mandai
    Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, Japan
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
  • Masayo Takahashi
    Laboratory for Retinal Regeneration, RIKEN Center for Developmental Biology, Kobe, Japan
    Ophthalmology, Institute for Biomedical Research and Innovation Hospital, Kobe, Japan
  • Footnotes
    Commercial Relationships   Yasuo Kurimoto, None; Yasuhiko Hirami, None; Masashi Fujihara, None; Chikako Morinaga, None; Midori Yamamoto, None; Kanako Fujita, None; Sunao Sugita, None; Michiko Mandai, None; Masayo Takahashi, None
  • Footnotes
    Support  AMED(JAPAN) Grant 15bk0104006h0003
Investigative Ophthalmology & Visual Science September 2016, Vol.57, No Pagination Specified. doi:
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      Yasuo Kurimoto, Yasuhiko Hirami, Masashi Fujihara, Chikako Morinaga, Midori Yamamoto, Kanako Fujita, Sunao Sugita, Michiko Mandai, Masayo Takahashi; Transplantation of Autologous induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium Cell Sheets for Exudative Age Related Macular Degeneration : A Pilot Clinical Study

      . Invest. Ophthalmol. Vis. Sci. 201657(12):.

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

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Abstract

Purpose : Recently developed induced pluripotent stem cell (iPSC) s are a promising donor source for regenerative medicine. We report the first-in-human case of iPSC therapy in which iPSC-derived retinal pigment epithelium (RPE) cells were transplanted to a patient with exudative age-related macular degeneration (AMD) .

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Methods : A patient with advanced exudative AMD in whom the current standard treatments had not been effective was recruited. A small piece of skin was collected from the patient’s arm and used to generate iPSCs. The iPSCs were differentiated into RPE sheets suitable for transplantation. After the assessments of safety and quality of the iPSC-derived RPE, the RPE sheet was subretinally transplanted following surgical removal of the neovascular tissue. The patient was monitored by systemic and ophthalmic examinations for a year after the surgery. The primary outcome to be assessed in this pilot study is the safety of this therapeutic protocol.


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Results : An autologous iPSC-derived RPE sheet was successfully transplanted. The submacularly transplanted RPE sheet survives well without any findings of immune rejection nor adverse proliferation, and any significant adverse events associated with the therapeutic protocol were not observed for a year. Retinal imaging examinations showed improvement of the pre-existed exudative change. Best-corrected visual acuity was maintained at 0.09 (=18/200) without additional anti-VEGF therapy and the score of VFQ (Visual Function Questionnaire)-25 improved from 40.7 to 58.3.

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Conclusions : The first-in human iPSC-based transplantation was carried out successfully in a patient with exudative AMD. The primary endpoint, safety of the therapeutic protocol, was attained after a year.


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This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.

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