Government regulators have given the go-ahead to a second study that will for the first time carefully test a treatment created using human embryonic stem cells in people, according to the company sponsoring the experiment.
The Food and Drug Administration approved a request by Advanced Cell Technology Inc. of Marlborough, Mass., to inject cells created from human embryonic stem cells into the eyes of 12 patients suffering from advanced cases of a disease that causes blindness. Stargardt’s Macular Dystrophy, which is one of the most common forms of juvenile macular blindness, progressively destroys vision, usually beginning in childhood. It is currently incurable.
The study will involve injecting 50,000 to 200,000 cells known as retinal pigmented epithelial cells in the hopes that they will replace those ravaged by the disease. Studies in rats found the cells helped prevent further vision loss and even restored some sight. While the study is designed primarily to test the safety of the treatment, researchers will looks for signs of improvement in the patients’ vision. The company hopes the approach will work for many conditions, including the leading cause of blindness among the elderly.
“I think this marks the beginning of a new era for stem cell research,” Robert Lanza, the company’s chief scientific officer, wrote in an email. “After a decade of intense controversy, the field is finally ready to prove itself—and to actually start helping patients suffering from a range of horrific diseases. It also shows the new readiness of the FDA to work with researchers to move exciting new stem cell therapies out of the laboratory and into the clinic.”