Thirteen Participants Treated in ACT's Stem Cell Clinical Trial

Results from the first-ever clinical trials of a retinal disease treatment derived from stem cells continue to be encouraging. Advanced Cell Technology (ACT), developer of the therapy, recently reported that 13 people — nine with Stargardt disease and four with dry age-related macular degeneration (AMD) — have had no safety issues after receiving transplanted cells. In addition, modest vision improvements for the first participants treated more than a year ago have been sustained.

According to ACT, the transplanted cells — retinal pigment epithelial (RPE) cells developed from human embryonic stem cells — appear to be engrafting into the retinas of some of the participants. Engraftment is an indicator that the transplanted cells have the potential to function like native cells.

RPE cells provide essential nutrition and waste removal for photoreceptors, the cells that provide vision. The dysfunction of RPE cells in dry AMD and Stargardt disease eventually leads to loss of photoreceptors.

The first nine participants in ACT’s trials each received transplants of 50,000 cells, and the next four received 100,000 cells. The researchers plan to transplant as many as 200,000 cells for the treated retina of each patient during their three Phase I/II clinical trials. Two studies — one for dry AMD, the other for Stargardt disease — are taking place in the United States. A trial for Stargardt disease is also underway in the United Kingdom.

ACT’s researchers plan to treat people with less advanced disease in later stages of their human studies. They believe the treatment may provide greater vision improvement in these individuals.

The Foundation Fighting Blindness funded laboratory research that made ACT’s human studies possible.