Positive Safety Results for Macular Degeneration Treatment

Advanced Cell Technology, Inc. has announced that Phase 1/2 clinical data published online in The Lancet demonstrate positive long-term safety results using ACT’s proprietary Retinal Pigment Epithelium (RPE) cells for the treatment of Stargardt’s macular degeneration (SMD) and dry age-related macular degeneration (AMD). The publication features data from 18 U.S.-based patients with at least six months of post-transplant follow-up.

“These study results represent an important milestone and strengthen our leadership position in regenerative ophthalmology,” said Paul K. Wotton, Ph.D., President and Chief Executive Officer. “We would like to thank the patients for their willingness to participate in these studies. Our findings underscore the potential to repair or replace tissues damaged from diseases. We plan to initiate comprehensive Phase 2 clinical trials for the treatment of both AMD and SMD, two disease states where there is currently no effective treatment.”

These two studies provide the first evidence of the mid- to long-term safety, survival, and potential biologic activity of pluripotent stem cell progeny into humans with any disease. In addition to showing no adverse safety issues related to the transplanted tissue, anatomic evidence confirmed successful engraftment of the RPE cells, which included increased pigmentation at the level of the RPE layer after transplantation in 13 of 18 patients.

Robert Lanza, M.D., Chief Scientific Officer of ACT and co-senior author of the paper, commented, “Diseases affecting the eye are attractive first-in-man applications for this type of investigational therapy due to the immune-privileged nature of the eye. Despite the degenerative nature of these diseases, the vision of 10 of 18 patients showed measurable improvement at the six month follow up, after transplantation of the RPE cells. Furthermore, the cells have been well tolerated for a median period of 22 months with two of the patients treated more than three years ago. We are pleased that there have been no serious safety issues attributable to the cells observed in any of the patients.”

Vision was measured using the widely accepted standard for visual acuity testing, the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity exam.

Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute, principal investigator and lead author of the publication said “The data published in The Lancet support the potential safety and biological activity of stem cell-derived retinal tissue. Once again, surgical access to the subretinal space has proven safe. However, for the first time in humans, terminally differentiated stem cell progeny seem to survive, and do so without safety signals. Combined with the functional signals observed, these data suggest that this regenerative strategy should move forward. This is a hopeful and exciting time for ophthalmology and regenerative medicine.”