Advanced Cell Technology, Inc. (“ACT”;
or the “Company”), a leader in the field of regenerative medicine, announced today that investigators for the Company’s Phase I/II clinical trials for Stargardt’s macular dystrophy (SMD) and dry age-related macular degeneration (dry AMD) have observed evidence of engraftment of the transplanted human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells and visual acuity gain in patients treated over the 18 months since the trials were first initiated. The Company believes this observation of engraftment and persistence is clinically important because 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. Additionally, investigators have not observed any issues of safety relating to the transplanted human embryonic stem cell (hESC)-derived RPE cells in any of the 18 patients treated. The company believes this warrants early (though cautious) optimism as the company looks forward to initiating the second half of the three trials.
“Now that we are at the halfway point in all three of our ongoing trials, we have had the opportunity to stop and assess not only the data relating to safety, but also to the activity of the transplanted RPE cells,” said Gary Rabin, chairman and CEO. “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. 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.”
“We are optimistic about the prospects of our RPE cells ultimately impacting positively on various forms of macular degeneration,” said Robert Lanza, M.D., ACT’s chief scientific officer. “As we have added additional clinical trial sites, the pace of patient treatments has picked up. At the same time, we are extending our observations to a greater number of patients, as well as collecting data over extended periods of time after the surgery.”