Stem cells in clinical trials for treatment of retinal degeneration

Abstract

Introduction: After decades of basic science research involving the testing of regenerative strategies in animal models of retinal degenerative diseases, a number of clinical trials are now underway, with additional trials set to begin shortly. These efforts will evaluate the safety and preliminary efficacy of cell-based products in the eyes of patients with a number of retinal conditions, notably including age-related macular degeneration, retinitis pigmentosa and Stargardt’s disease.

Areas covered: This review considers the scientific work and early trials with fetal cells and tissues that set the stage for the current clinical investigatory work, as well the trials themselves, specifically those either now completed, underway or close to initiation. The cells of interest include retinal pigment epithelial cells derived from embryonic stem or induced pluripotent stem cells, undifferentiated neural or retinal progenitors or cells from the vascular/bone marrow compartment or umbilical cord tissue.

Expert opinion: Degenerative diseases of the retina represent a popular target for emerging cell-based therapeutics and initial data from early stage clinical trials suggest that short-term safety objectives can be met in at least some cases. The question of efficacy will require additional time and testing to be adequately resolved.

Keywords:

• age-related macular degeneration
• cell-based
• intravitreal
• photoreceptor
• progenitor
• retinal pigment epithelial
• retinitis pigmentosa
• Stargardt
• subretinal
• transplantation

Acknowledgements

The author would like to thank S Menges for assistance with preparation of the manuscript.

Declaration of interest

H Klassen receives ongoing support from the California Institute of Regenerative Medicine (CIRM) and the Polly and Michael Smith Foundation. H Kassen has Intellectual Property related to his co-founding of the startup company, jCyte. The sponsors had no role in manuscript preparation. The author has intellectual property as well as an equity interest in jCyte, Inc., a company that may potentially benefit from the type of results described. He also serves on the company’s Board. The terms of this arrangement have been reviewed and approved by the University of California, Irvine in accordance with its conflict of interest policies. The author consulted for ReNeuron and StemCells, Inc. in the past. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.

Notes

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