Advanced Cell Technology (ACTC) Reports Milestone Achievement in Phase I/II Embryonic Stem Cell Clinical Trials

Investigators in Advanced Cell Technology’s phase I/II clinical trials for Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD) report that they have observed evidence of engraftment of the transplanted human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as well as visual acuity gain in patients treated throughout the trial’s duration.

The company has reached the halfway point in all three of its ongoing trials, stopping to assess the data and activity of the transplanted RPE cells. These most recent findings mark the first time the company has achieved engraftment and persistence in AMD, and is of clinical value because the engraftment of cells in the proper area and recapitulation of normal RPE cell layer structure is important to the ultimate efficacy of the RPE cells in the treatments, says the company.

“In our first report of hESC-derived cells transplanted into patients, published in the Lancet, we reported no adverse safety signals and that both patients that had been treated by that point showed some signs of visual improvements. For both of those patients, those gains in visual acuity have persisted for nearly 18 months now,” Gary Rabin, ACTC chairman and CEO stated in the press release. “In addition, we have observed persisting engraftment of the transplanted RPE cells in our more recent SMD and dry AMD patients. This is the first time we have shown this in dry AMD patients, which we find particularly encouraging, given that dry AMD represents one of the largest unmet medical needs in the world.”

Robert Lanza, M.D., ACTC’s chief scientific officer, said the company is optimistic that its RPE cells will have a positive impact on various forms of degeneration. Additionally, researchers have not observed any issues of safety relating to the transplanted human embryonic stem cell (hESC)-derived RPE cells in any of the patients treated.

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