Stem cell clinical trials: toward cell-based therapy for retinal degenerations

Abstract

Stem cell-based therapeutic strategies are central to the emerging field of regenerative medicine. The past two decades have seen many spectacular results in animal models and at this juncture the clinical translation of much of this work is in progress. It should be evident that the technical and regulatory challenges facing the development of novel cell-based therapeutics are not insignificant, yet a number of early milestones have already been achieved. While it is too soon to predict the eventual outcome of these efforts, early indications could be characterized as cautiously optimistic. Certainly, hopes have been raised that something efficacious will emerge within the next several years. In particular, diseases of the CNS represent an area of vast unmet medical need, particularly in light of aging demographics in much of the world. It is therefore not surprising that strategies aimed at the treatment of CNS conditions have received much of the attention. Of these, retinal degenerative diseases have proven to be disproportionately popular among potential disease targets. Currently, there are clinical trials underway in which a range of different stem cell types are being transplanted to the diseased retina of patients with a variety of retinal disease phenotypes, including the atrophic form of age-related macular degeneration (AMD), as well as a number of other degenerative retinal conditions.