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Stem cells in clinical trials for treatment of retinal degeneration

Henry Klassen1

  • 1a University of California, Gavin Herbert Eye Institute & Stem Cell Research Center , Sue & Bill Gross Hall, Room 2006, 845 Health Sciences Road, Irvine, CA 92697-1705, USA +1 94 98 24 77 50 ; +1 94 98 24 96 26 ; hklassen@uci.edu.

Expert Opinion on Biological Therapy
|September 29, 2015
PubMed
Abstract

Insights

Cell-based therapies show promise for retinal degenerative diseases like age-related macular degeneration. Early clinical trials indicate short-term safety, but efficacy requires further investigation.

Area of Science:

  • Regenerative medicine
  • Ophthalmology
  • Stem cell therapy

Background:

  • Decades of basic science research in animal models of retinal degenerative diseases.
  • Numerous clinical trials are now underway or commencing shortly.
  • Focus on conditions including age-related macular degeneration, retinitis pigmentosa, and Stargardt's disease.

Purpose of the Study:

  • To review scientific work and early trials with fetal cells and tissues.
  • To examine current clinical investigatory work and ongoing trials.
  • To assess the safety and preliminary efficacy of cell-based products for retinal conditions.

Main Methods:

  • Review of preclinical studies and early clinical trials.
  • Inclusion of trials involving fetal cells and tissues.
  • Analysis of cell types such as retinal pigment epithelial cells, neural progenitors, and stem cells.

Main Results:

  • Degenerative retinal diseases are a key target for cell-based therapeutics.
  • Initial data from early-stage clinical trials suggest short-term safety can be achieved.
  • Efficacy of these therapies remains to be fully determined.

Conclusions:

  • Cell-based therapeutics offer a promising avenue for treating retinal degenerative diseases.
  • Early clinical trial data support the short-term safety of these approaches.
  • Long-term efficacy requires further extensive research and clinical evaluation.
Keywords:
Stargardtage-related macular degenerationcell-basedintravitrealphotoreceptorprogenitorretinal pigment epithelialretinitis pigmentosasubretinaltransplantation

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