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Foundation News » Stargardt Disease
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Clinical Trial Recruitment Underway for Stem Cell Treatments

June 2, 2011 - Back in November, when Advanced Cell Technology (ACT), a biotechnology company, got the OK from the U.S. Food and Drug Administration to move ahead with the first-ever human studies of retinal-disease treatments derived from human stem cells, it had yet to confirm study sites, which also needed internal approvals.

ACT has recently announced that the second of two Phase I/II clinical-trial sites received institutional review board (IRB) approval, thus clearing the way for the recruitment of 24 participants afflicted with either dry age-related macular degeneration (AMD) or Stargardt disease, a juvenile form of macular degeneration—both of which cause progressive, devastating vision loss.

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 is ready to start groundbreaking studies in the development of stem cell therapies for retinal diseases,” 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 www.clinicaltrials.gov and search using these clinical trial identifiers: NCT01345006 and NCT01344993.

 
 

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