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USC Roski Eye Institute Researchers Report First Results in Stem Cell-Based Clinical Trial for Dry Age-Related Macular Degeneration

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Researchers, clinicians, scientists from the USC Roski Eye Institute and collaborators report encouraging results of a first-in-kind stem-cell based implant in a featured article in Science Translational Medicine entitled, “A Bioengineered Retinal Pigment Epithelial Monolayer for Advanced, Dry Age-Related Macular Degeneration.”

The novel minimally invasive stem cell-based therapy for dry age-related macular degeneration (AMD) was developed by a team at USC Roski Eye Institute, led by Mark S. Humayun MD, PhD, and David R. Hinton, MD, which was funded by the California Institute for Regenerative Medicine. The implant consists of stem cell-derived retinal pigment epithelium cells (RPE) on an ultrathin synthetic substrate. The implanted scaffold of RPE are localized and can function to support and replenish light sensing cells of the eye, which would help restore and prevent vision loss in patients with AMD.

“This is the first human trial of this novel stem cell–based implant, which is designed to replace a single-cell layer that degenerates in patients with dry age-related macular degeneration,” says lead author and surgeon for the study Amir H. Kashani, MD, PhD, assistant professor of clinical ophthalmology at the Keck School of Medicine of USC. “This implant has the potential to stop the progression of the disease or even improve patients’ vision. Proving its safety in humans is the first step in accomplishing that goal.”

Photo courtesy of Britney O. Pennington, PhD
Photo courtesy of Britney O. Pennington, PhD

The first results of the phase I/IIa clinical trial conducted at the USC Roski Eye Institute has been reported on four patients which were followed up to one year to assess safety. It was determined that the implant is safe and integrates well with the patient’s retinal tissue. One patient had improvement in visual acuity by up to 17 letters and two patients had gains in visual function, which was measured by how well they could use the area of the retina treated by the implant. None of the patients showed evidence of progression in vision loss.

Dry AMD can have a profound affect on the quality of life of an individual. In time as the disease progresses, patients will be unable to recognize faces, read or even drive. It is projected that over 3 million will be diagnosed with dry AMD by 2020.

“Our study shows that this unique stem cell–based retinal implant thus far is well-tolerated, and preliminary results suggest it may help people with advanced dry age-related macular degeneration,” says coauthor and lead inventor of the implant Mark S. Humayun, MD, PhD, director of the USC Institute for Biomedical Therapeutics, co-director of the USC Roski Eye Institute, affiliate principal investigator with the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at USC and University Professor of Ophthalmology at the Keck School.

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Other USC researchers include Biju B. Thomas, PhD; Debbie Mitra, PhD; and Danhong Zhu, MD, PhD.

Collaborating institutions for the study include Regenerative Patch Technologies LLC, which also contributed to the funding of the study, as well as Camtek LLC, the California Institute of Technology, Retina Vitreous Associates Medical Group, California Retina Consultants, Atlantis Eyecare, City of Hope, University of California, Santa Barbara and Denney Research Center. Additional sources of funding for the study include Lori Mars and David Fields Gift, Estate of Beatrice Apple, William K. Bowes Foundation, Vermont Community Foundation, Breaux Foundation, Wilcox Family Foundation and Research to Prevent Blindness.

For more information about the study, visit https://clinicaltrials.gov/ct2/show/NCT02590692?term=nct02590692&rank=1. To participate in the study, please call (323) 442-6335.

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Disclosures: Regenerative Patch Technologies LLC was founded by Mark Humayun, MD, PhD, and David R. Hinton, MD, from Keck Medicine of USC and Dennis O. Clegg, PhD, from the University of California, Santa Barbara. The technology to produce the stem cell–based retinal implant is exclusively licensed to Regenerative Patch Technologies LLC from the University of Southern California, the California Institute of Technology and the University of California, Santa Barbara. Humayun and Hinton have an equity interest in and are consultants for Regenerative Patch Technologies LLC.

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