Stem Cell Clinical Trials for Stargardt Disease and Dry AMD Set to Begin

Advanced Cell Technology (ACT) has received authorization from the FDA to begin the first-ever human study of a retinal degenerative disease treatment derived from human stem cells. The Phase I/II clinical trial will evaluate the treatment in people with Stargardt disease, a juvenile form of macular degeneration that causes progressive, devastating vision loss.

“ACT’s clinical trial is a critical, groundbreaking milestone in the development of stem cell therapies for retinal diseases,” says Stephen Rose, Ph.D. “Stem cells offer enormous potential for saving and restoring vision, and we are delighted to see ACT’s treatment directed toward Stargardt disease, a vision-robbing condition in children and young adults for which there are currently no treatments or cures. In the long-run, stem cells offer significant potential for restoring vision in people with the most advanced retinal disease.

ACT has also received FDA authorization to launch a Phase I/II clinical trial of the same treatment for people with dry age-related macular degeneration. The trial will enroll a total of 12 participants. Potential dry AMD trial sites include Stanford and the University of California, Los Angeles.

The Foundation Fighting Blindness has funded many preclinical studies of human embryonic stem cells for retinal degenerative diseases that helped make ACT’s clinical trial possible. Dr. Ray Lund, a stem cell research expert who has served as a consultant to ACT, has received Foundation funding since the 1980s for his innovative cell transplantation and stem cell research. He says that results of ACT’s preclinical studies of their stem cell treatment were phenomenal, and he is optimistic about their potential to save vision in people.

ACT’s treatment involves the transformation of human embryonic stem cells into retinal pigment epithelial cells (RPE). RPE cells degenerate in several retinal conditions including Stargardt disease and age-related macular degeneration. RPE cells provide essential supportive functions for photoreceptors, the cells that provide vision. By placing healthy RPE cells in the retina, researchers believe they can save photoreceptors and slow or halt vision loss from a variety of retinal degenerative conditions.

ACT is planning to extend the clinical study of their treatment to age-related macular degeneration. They are also seeking authorization to begin clinical trials in Europe.

“Recent progress in the advancement of stem cell therapies has been outstanding,” says William T. Schmidt, chief executive officer, Foundation Fighting Blindness. “We invest in a variety of stem cell projects, including those that use embryonic stem cells, because they offer one of the most powerful and immediate opportunities to stop blindness.”

The Foundation funds approximately $2 million annually in cell- and stem-cell-based research.