Embryonic stem-cell-derived retinal pigment epithelial cells for macular degeneration – Authors' reply

Induced Pluripotent Stem Cells: Reprogramming Platforms and Applications in Cell Replacement Therapy

“…Allogeneic transplant of human ESCs has been utilized to treat spinal cord injury in 2010, 138 dry AMD in 2016, 139,140 and type I diabetes mellitus in 2014. 141,142 In these trials, the engraftment was highly efficient, and the treated individuals did not show signs of oncogenesis.…”

Human Pluripotent Stem Cells: Applications and Challenges in Neurological Diseases

“…Thus, the FDA has granted the permission to Advanced Cell Technology’s for clinical trials using hESC-derived RPE (MA09-hRPE cells) for Stargardt macular dystrophy (SMD) and dry age-related macular degeneration (AMD; studies registered with , numbers NCT01345006 and NCT01344993). A preliminary report regarding the safety and tolerability of this trial in one patient with AMD and the other with SMD showed no signs of hyperproliferation, tumorigenicity, ectopic tissue formation, or immune rejection of the hESC-derived RPE cells 4 months after transplantation (Schwartz et al, 2012). …”

“…The recent findings are supportive of a future hPSC-based therapy for neurological diseases. Long term engraftment of hPSC-derived cells in several CNS disease models demonstrated in vivo survival and function of these cells together with improvement up to complete restoration of the deficits resembling the symptoms observed in human neurological diseases (Harper et al, 2004; Keirstead et al, 2005; Deshpande et al, 2006; Gamm et al, 2007; Corti et al, 2009, 2010; Francis et al, 2009; Lu et al, 2009; Kriks et al, 2011; Schwartz et al, 2012). Also, hPSCs offer the advantage to provide an inexhaustible supply of differentiated cell types compared to the other cells that have been used in clinic until now (mesenchymal stem cells, fetal, and adult stem cells).…”