Stem cells in clinical trials for treatment of retinal degeneration

Expert Opin Biol Ther. 2016;16(1):7-14. doi: 10.1517/14712598.2016.1093110. Epub 2015 Sep 28.


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

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Humans
  • Induced Pluripotent Stem Cells / transplantation
  • Macular Degeneration / congenital
  • Macular Degeneration / therapy
  • Retina / pathology
  • Retina / physiopathology
  • Retinal Degeneration / surgery*
  • Retinitis Pigmentosa / therapy
  • Stargardt Disease
  • Stem Cell Transplantation* / methods
  • Stem Cell Transplantation* / trends