title: Developing cellular therapies for retinal degenerative diseases.
abstract: Biomedical advances in vision research have been greatly facilitated by the clinical accessibility of the visual system, its ease of experimental manipulation, and its ability to be functionally monitored in real time with noninvasive imaging techniques at the level of single cells and with quantitative end-point measures. A recent example is the development of stem cell-based therapies for degenerative eye diseases including AMD. Two phase I clinical trials using embryonic stem cell-derived RPE are already underway and several others using both pluripotent and multipotent adult stem cells are in earlier stages of development. These clinical trials will use a variety of cell types, including embryonic or induced pluripotent stem cell-derived RPE, bone marrow- or umbilical cord-derived mesenchymal stem cells, fetal neural or retinal progenitor cells, and adult RPE stem cells-derived RPE. Although quite distinct, these approaches, share common principles, concerns and issues across the clinical development pipeline. These considerations were a central part of the discussions at a recent National Eye Institute meeting on the development of cellular therapies for retinal degenerative disease. At this meeting, emphasis was placed on the general value of identifying and sharing information in the so-called "precompetitive space." The utility of this behavior was described in terms of how it could allow us to remove road blocks in the clinical development pipeline, and more efficiently and economically move stem cell-based therapies for retinal degenerative diseases toward the clinic. Many of the ocular stem cell approaches we discuss are also being used more broadly, for nonocular conditions and therefore the model we develop here, using the precompetitive space, should benefit the entire scientific community.
authors.affiliation: Unit on Ocular and Stem Cell Translational Research, National Eye Institute, National Institutes of Health, Bethesda, Maryland.
authors.forename: Kapil
authors.initials: K
authors.lastname: Bharti
authors.forename: Mahendra
authors.initials: M
authors.lastname: Rao
authors.forename: Sara Chandros
authors.initials: SC
authors.lastname: Hull
authors.forename: David
authors.initials: D
authors.lastname: Stroncek
authors.forename: Brian P
authors.initials: BP
authors.lastname: Brooks
authors.forename: Ellen
authors.initials: E
authors.lastname: Feigal
authors.forename: Jan C
authors.initials: JC
authors.lastname: van Meurs
authors.forename: Christene A
authors.initials: CA
authors.lastname: Huang
authors.forename: Sheldon S
authors.initials: SS
authors.lastname: Miller
mesh_terms: D000818:Animals; D060846:Cell Engineering; D064987:Cell- and Tissue-Based Therapy; D002986:Clinical Trials as Topic; D003226:Congresses as Topic; D003299:Cooperative Behavior; D004195:Disease Models, Animal; D053595:Embryonic Stem Cells / Q000637:transplantation; D006801:Humans; D054554:National Eye Institute (U.S.); D039904:Pluripotent Stem Cells / Q000637:transplantation; D044968:Regenerative Medicine; D012162:Retinal Degeneration / Q000628:therapy*; D033581:Stem Cell Transplantation / Q000379:methods*; D057170:Translational Research, Biomedical; D014481:United States
keywords: age-related macular degeneration; cell-based therapy; retinitis pigmentosa; stem cells