Researchers at the National Eye Institute (NEI) are launching a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy (GA), the advanced dry form of age-related macular degeneration (AMD).
Under the phase I/IIa clinical trial protocol 12 patients with advanced-stage GA will receive the iPSC-derived RPE implant in one of their eyes and be closely monitored for a period of at least one year to confirm safety.
A concern with any stem cell-based therapy is its oncogenic potential: the ability for cells to multiply uncontrollably and form tumors. In animal models, the researchers genetically analyzed the iPSC-derived RPE cells and found no mutations linked to potential tumor growth.
The use of an individual’s autologous (own) blood cells is expected to minimize the risk of the body rejecting the implant. Should early safety be confirmed, later study phases will include more patients to assess the efficacy of the implant to prevent blindness and restore vision in patients with GA.
1. Sharma R, Khristov V, Rising A, Jha BS, Dejene R, Hotaling N, Li Y, Stoddard J, Stankewicz C, Wan Q, Zhang C, Campos MM, Miyagishima KJ, McGaughey D, Vilasmil R, Mattapallil M, Stanzel B, Qian H, Wong W, Chase L, Charles S, McGill T, Miller S, Maminishikis A, Amaral J, Bharti K. Patient-specific clinical-grade iPS cell-derived retinal pigment epithelium patch rescues retinal degeneration in rodent and pig eyes. Jan 16, 2019 Science Translational Medicine. DOI: 10.1126/scitranslmed.aat5580