Eye Disease Trial Could Be First Embryonic Stem Cell Effort In U.S.

Advanced Cell Technology (ACT) announced today that it has applied to the Food and Drug Administration to conduct a trial using embryonic stem cell–derived retinal pigment epithelial cells to treat Stargardt’s Macular Dystrophy, a congenital eye disease. ACT, which has almost gone under several times in recent years, may now be on the verge of conducting the nation's first trial of an embryonic stem cell therapy, says Chief Scientific Officer Robert Lanza. Geron Corp., which earlier got permission to administer stem cell–derived oligodendrocyte progenitors to treat spinal injury, has been bogged down in delays and may not begin its trial until late next year.

Lanza says eye disease is a good place to start with such cell therapies because the eye doesn't reject foreign tissues, so no imunnosuppressive drugs are necessary. Because Stargardt's is an "orphan disease" with no treatment, he says it should get expedited review from FDA. Within the next few months the company also hopes to apply for permission to test the cells on macular degeneration.