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演題詳細

Symposium

基礎-臨床統合シンポジウム:iPS細胞の基礎研究から臨床応用まで
Integrated Symposium of Basic and Clinical Neuroscience:Inducued pluripotent stem (iPS) cells: from basic research to clinical application

開催日 2014/9/11
時間 9:00 - 11:00
会場 Room A(Main Hall)
Chairperson(s) 髙橋 良輔 / Ryosuke Takahashi (京都大学大学院医学研究科・臨床神経学 / Department of Neurology, Kyoto University Graduate School of Medicine, Japan)
岡野 栄之

iPS細胞による網膜細胞治療
Retinal cell therapy using iPS cells

  • S1-A-1-4
  • 高橋 政代 / Masayo Takahashi:1 
  • 1:理化学研究所発生・再生科学総合研究センター / Center for Developmental Biology, RIKEN, Japan 

Age-related macular degeneration (AMD) causes severe visual impairment due to age-dependent disorder of retinal pigment epithelium (RPE).
We generated hiPSC-derived RPE (hiPSC-RPE) cell sheets optimized to meet clinical use requirements including quality, quantity, consistency, and safety. These cell sheets are generated as a monolayer of cells without any artificial scaffolds. They have the necessary quality, such as expression of typical RPE markers, tight junction formation, polarized secretion of growth factors and phagocytotic ability. It is easy to obtain enough amount of cells to cover the retinal lesion of less than 2 mm in diameter. The hiPSC-RPE consistently showed similar expression patterns of RPE signature genes even though made from various iPS cell lines. As for the safety, we evaluated tumorigenesity tests using immune deficient mice, karyotype.

Furthermore, autologous primate iPSC-RPE cell sheets showed no immune rejection or tumor formation after transplantation into the subretinal space of the cynomologus monkeys, while allogeneic transplantation showed immune rejection. These results suggest that autologous hiPSC-RPE cell sheets may serve as a useful form of graft for use in tissue replacement therapy for AMD.

We made Standard Operation Procedure (SOP) and Quality Control procedure for each step. In the clinical research, six patients with active wet type AMD after existing treatment such as anti-VEGF drug injection into the eye will be enrolled. The primary endpoint is safety of the treatment. We will follow the patients for more than three years. The efficacy, the secondary endpoint, will be examined one year after the surgery.

However those autologous cell therapy works, it is difficult to treat many people. To make the cell therapy as a standard treatment in the future, we should seek for the way to treat with allogeneic cells and also the automatical production system etc.

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