Advanced Cell Technology announced the enrollment of the first patients in its two Phase 1/2 clinical trials for Stargardt’s Macular Dystrophy (SMD) and Dry Age-Related Macular Degeneration (Dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).
The Phase 1/2 trials are prospective, open-label studies primarily designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD and Dry AMD. Each study will enroll 12 patients with cohorts of three patients in an ascending dosage format. The primary endpoint of both studies is to determine the safety and tolerability of hESC-derived RPE cells at 12 months. The patients were enrolled at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA).
Principal Investigator Steven Schwartz, MD, Ahmanson professor of ophthalmology at the David Geffen School of Medicine and retina division chief at the Jules Stein Eye Institute at UCLA, says, “This trial will begin the process of understanding whether stem cell-derived RPE cells have the potential to be a safe and effective treatment for these debilitating diseases. We are looking forward to evaluating the safety and tolerability data of these Phase 1/2 trials, and hope that these early trials will also produce key information relating to engraftment and function of the transplanted RPE cells.”
The progress of disease in both SMD and Dry AMD includes atrophy or thinning of the layer of RPE cells in the patient’s macula at the center of the retina, the region specialized for high acuity vision. With the loss of RPE cells in the macula comes the eventual loss of photoreceptors. Over time, the progressive loss of RPE cells and concomitant loss of photoreceptors can cause severe central visual deterioration and even blindness as the macula becomes less functional and central vision is gradually lost. ACT’s SMD and Dry AMD therapeutic programs utilize transplanted RPE cells to treat these conditions by replacing RPE cells in the patient’s eyes before all RPE function is lost.
Release Date: June 16, 2011
Source: Advanced Cell Technology
Filed Under: Drug Discovery