Researchers at the National Eye Institute (NEI) will launch a clinical trial to test the safety of a new patient-specific stem cell-based therapy to treat geographic atrophy - the advanced ‘dry’ form of age-related macular degeneration (AMD) - offering promise for potential future treatments.
“The protocol, which prevented blindness in animal models, is the first clinical trial in the US to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC),” said Dr Kapil Bharti, NEI senior investigator. Under the phase I/IIa clinical trial protocol, 12 patients with advanced-stage geographic atrophy 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 ability for cells to multiply uncontrollably and form tumours, said researchers. But prior analysis of the animal models found no mutations linked to potential tumour growth and the use of an individual’s own blood cells is expected to minimise the risk of the body rejecting the implant.
If early safety is confirmed, later study phases will include more patients in order to assess the efficacy of the implant in preventing blindness and restoring vision in patients with geographic atrophy.
