ACT Expands Human Studies of Stem-Cell Derived Retinal Treatment

April 30, 2013

Advanced Cell Technology (ACT) has increased enrollment and dosing levels in Phase I/II clinical trials for its retinal therapy derived from human embryonic stem cells for people with Stargardt disease and dry age-related macular degeneration (AMD). Twenty people have been enrolled in its two U.S. human studies, which began in July 2011. The company plans to treat a total of 32 patients in its U.S. trials. ACT has also begun transplanting 150,000 cells — up from 100,000 — underneath the patients’ retinas.

Patients with better visual acuity — no worse than 20/100 — are now being enrolled and treated in the clinical trials. Researchers believe that the treatment may have a better effect on vision for people with less advanced disease.

ACT’s U.S. Stargardt disease study and its U.S. dry AMD study are underway at: Jules Stein Eye Institute at the University of California, Los Angeles; Bascom-Palmer Eye Institute at the University of Miami; and Wills Eye Institute in Philadelphia. Its AMD study is also taking place at Massachusetts Eye and Ear Infirmary in Boston.

A clinical trial for ACT’s treatment of Stargardt disease is also underway in the United Kingdom. Seven patients have been treated thus far, and one has received the 150,000-cell dose.

ACT’s treatment involves transplantation of retinal pigment epithelial (RPE) cells derived from human embryonic stem cells into the patient’s retina. 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.

Though the primary goal of the studies is to determine the treatment’s safety, the investigators are also evaluating changes in vision.