Long-Term Safety and Function of RPE from Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration (original) (raw)

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Casey Eye Institute, Oregon Health and Science University

, Portland, Oregon

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Advanced Cell Technology, Worcester

, Massachusetts,

USA

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Casey Eye Institute, Oregon Health and Science University

, Portland, Oregon

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Casey Eye Institute, Oregon Health and Science University

, Portland, Oregon

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Casey Eye Institute, Oregon Health and Science University

, Portland, Oregon

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Advanced Cell Technology, Worcester

, Massachusetts,

USA

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Advanced Cell Technology, Worcester

, Massachusetts,

USA

Correspondence: Robert Lanza, M.D., Advanced Cell Technology, Worcester, MA 01605; Telephone: 508-756-1212 (ext. 655); e-mail: rlanza@advancedcell.com

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Casey Eye Institute, Oregon Health and Science University

, Portland, Oregon

Correspondence: Raymond Lund, Ph.D., Casey Eye Institute, Oregon Health and Science University, Portland, OR 97239, USA. Telephone: 503-494-2256; e-mail: lundr@ohsu.edu

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Bin Lu, Christopher Malcuit, Shaomei Wang, Sergej Girman, Peter Francis, Linda Lemieux, Robert Lanza, Raymond Lund, Long-Term Safety and Function of RPE from Human Embryonic Stem Cells in Preclinical Models of Macular Degeneration, Stem Cells, Volume 27, Issue 9, September 2009, Pages 2126–2135, https://doi.org/10.1002/stem.149
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Abstract

Assessments of safety and efficacy are crucial before human ESC (hESC) therapies can move into the clinic. Two important early potential hESC applications are the use of retinal pigment epithelium (RPE) for the treatment of age-related macular degeneration and Stargardt disease, an untreatable form of macular dystrophy that leads to early-onset blindness. Here we show long-term functional rescue using hESC-derived RPE in both the RCS rat and Elov14 mouse, which are animal models of retinal degeneration and Stargardt, respectively. Good Manufacturing Practice-compliant hESC-RPE survived subretinal transplantation in RCS rats for prolonged periods (>220 days). The cells sustained visual function and photoreceptor integrity in a dose-dependent fashion without teratoma formation or untoward pathological reactions. Near-normal functional measurements were recorded at >60 days survival in RCS rats. To further address safety concerns, a Good Laboratory Practice-compliant study was carried out in the NIH III immune-deficient mouse model. Long-term data (spanning the life of the animals) showed no gross or microscopic evidence of teratoma/tumor formation after subretinal hESC-RPE transplantation. These results suggest that hESCs could serve as a potentially safe and inexhaustible source of RPE for the efficacious treatment of a range of retinal degenerative diseases.

Disclosure of potential conflicts of interest is found at the end of this article.

Copyright © 2009 AlphaMed Press

This article is published and distributed under the terms of the Oxford University Press, Standard Journals Publication Model (https://academic.oup.com/journals/pages/open\_access/funder\_policies/chorus/standard\_publication\_model)

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