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First Patients Enrolled in Stem Cell Clinical Trials for Stargardt Disease and AMD

June 17, 2011 – Advanced Cell Technology (ACT), a biotechnology company, has announced enrollment of the first patients in Phase I/II clinical trials for their retinal-disease treatment derived from human stem cells. Taking place at the Jules Stein Eye Institute at the University of California, Los Angeles and Oregon Health & Science University (OHSU), the trials are for people with Stargardt disease, a juvenile for of macular degeneration,  and dry age-related macular degeneration — both of which cause progressive, devastating vision loss.  The studies will enroll a total of 24 participants and are focusing primarily on safety and tolerability of the treatments.

Last November, ACT received  the O.K. from the U.S. Food and Drug Administration to begin the studies. The institutional review boards (IRBs) for Jules Stein and OHSU gave the go ahead for the trials in May.

An IRB is an independent committee of experts charged with ensuring that a study site meets established standards for safety and ethics in human research. The Jules Stein Eye Institute at the University of California, Los Angeles, received IRB approval late last month, followed by the Casey Eye Institute at Oregon Health & Science University (OHSU) in Portland, Oregon, in May. ACT’s studies, involving the transformation of embryonic stem cells into healthy retinal cells, will focus primarily on safety and tolerability of the treatments.

“It’s exciting to see that ACT has started enrolling patients in these groundbreaking studies for the development of stem cell therapies,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “ACT’s treatments focus on dry AMD, which affects more than eight million people age 50 and over, and Stargardt disease, which robs children and young adults of vision and for which there is no treatment or cure. Stem cells have the potential to restore vision in people with the most advanced retinal disease.”

The Foundation funds approximately $2 million annually in cell- and stem-cell-based research. Since 1984, the Foundation has supported Casey Eye Institute
with a Center grant. This has provided infrastructure support that makes it possible for the Center to perform trials like the ACT trial. Since the 1980s, FFB has funded many preclinical studies that helped make ACT’s clinical trial possible, including those conducted by Dr. Ray Lund, an innovator in stem cell research. Dr. Lund, who has served as a consultant for ACT, says the results of the company’s preclinical stem cell studies were phenomenal and, thus, bode well for the clinical studies.

ACT’s treatment transforms human embryonic stem cells into retinal pigment epithelial (RPE) cells, which degenerate in several retinal conditions, including Stargardt disease and age-related macular degeneration. RPE cells provide essential support 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.

For trial recruitment information, including inclusion and exclusion criteria, visit and search using these clinical trial identifiers:
NCT01345006 and NCT01344993.



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