Participants in the First Stem Cell Clinical Trials for Retinal Disease Show Improved Vision

January 24, 2012

In the first-ever clinical trials of a stem cell treatment for retinal disease, two women – one with dry age-related macular degeneration (AMD), the other with Stargardt disease – demonstrated improved vision, including the ability to read more lines on an eye chart with their treated eyes.

Most important, the treatment is improving their independence and ability to perform daily activities. The 78-year-old woman with dry AMD says that she has begun making her own breakfast and shopping by herself. The Stargardt patient, a graphic designer in her fifties, reports doing more chores around the house and riding her bike again. She also notes improvements in her color perception and ability to adapt to dark settings.

Researchers from the Jules Stein Eye Institute at the University of California, Los Angeles, conducting the two Phase I/II clinical trials — one for each disease — are also pleased with the safety results for the first two patients. No adverse events have occurred, and overall safety has been demonstrated. Four-month results of the study were reported January 23, 2012, in the journalLancet.

The treatment is being developed by Advanced Cell Technology (ACT), a biotechnology company. The Foundation Fighting Blindness funded earlier stem cell lab studies, including the innovative work of Dr. Ray Lund, which made ACT’s clinical trials possible.

“This is a promising report. We are very pleased to learn that the treatment has been safe thus far — demonstrating safety is paramount in these early human studies — and we are delighted that both patients are showing vision improvement,” says Dr. Stephen Rose, chief researcher officer, Foundation Fighting Blindness. “While the trials are still at an early stage, the results are good news for more than 10 million people in the U.S., and millions more around the world, affected by retinal degenerative diseases.

“We at the Foundation have been excited about the potential of stem cells for saving and restoring vision, and ACT’s results show that we are on the right track. We look forward to more results from this trial and future Foundation-funded clinical trials for treatments derived from stem cells.”

During these early stages, ACT’s treatment involves the transplantation of 50,000 retinal pigment epithelial (RPE) cells transplanted into each eye. The RPE cells, derived from human embryonic stem cells using a procedure that wouldn’t destroy the embryos, play a critical role in supporting photoreceptors, the cells that provide vision. In dry AMD and Stargardt disease, RPE cells degenerate, which leads to the dysfunction and death of photoreceptors. ACT will transplant as many as 200,000 RPE cells in each patient’s retina as the Phase I/II trials progress. Twelve people will be enrolled in each of the two trials.

Researchers note that the cells transplanted into the Stargardt disease patient appeared to integrate in the proper location of the retina and take on normal RPE structure and organization. The anatomical results for the dry AMD patient were not as favorable; the RPE cells did not integrate. But the researchers were encouraged by the vision improvement they appeared to provide.

Dr. Rose notes, “We need to keep in mind that these results are for the first two people with a retinal disease ever to be treated with a therapy derived from stem cells. They are very short-term results, so there is a lot more to learn. With that said, the news is very encouraging and exciting.”

These results have been covered extensively in the media, including this report by BBC News.