RP stem cell therapy appears safe

An ongoing phase 1/2 trial shows that an investigational therapy for retinitis pigmentosa (RP) demonstrated a favorable safety and tolerability profile.

The regenerative medicine company jCyte and the Sue & Bill Gross Stem Cell Research Center at the University of California, Irvine are investigating the use of allogeneic human retinal progenitor cells to rescue sick and dying retinal photoreceptor cells in the diseased retina, either through secretion of growth factors or regeneration of new rod and cones.

Because the eye is immune-privileged, transplanted progenitor cells are not recognized as foreign and generate no immune response. As a result, patients are not immunosuppressed, as is often done in traditional organ transplantation, making the procedure relatively simple to complete in an office setting.

Funded by the California Institute for Regenerative Medicine, the trial has undergone 4 reviews by the Data Safety Monitoring Board, all of which were deemed successful. The first patient enrolled in the study was followed for 1 year. Interim safety results from this patient and 8 subsequent trial participants are described as “encouraging.”