DSMB authorizes ACT to proceed with next set of patients in stem cell clinical trials

Sep 29 2011

Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that the independent Data and Safety Monitoring Board (DSMB) overseeing the Company's two ongoing stem cell clinical trials in the United States authorized ACT to move forward with enrolling and treating the next set of patients in each of the trials. In July, surgeons led by 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 treated the first patients in the Company's 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). Each patient received an injection of 50,000 hESC-derived RPE cells in one of their eyes. Both the Dry AMD and SMD clinical trials are dose-escalation studies involving twelve patients in each, and are designed to evaluate the safety and tolerability of the injected RPE cells. The study design involves four cohorts of three patients each being treated at a predetermined dose of RPE cells, ranging from 50,000 to 200,000 cells. Based on the results of the first patient in each study, the DSMB authorized the Company to move forward with the next two patients in the studies. Each of these additional patients will also be treated with 50,000 RPE cells.

"The unanimous recommendation of the DSMB to proceed with the next four patients represents a significant milestone for our clinical programs. We hope that the positive trend in clinical results derived from treating the first patients with our RPE cells will continue," said Robert Lanza, M.D., chief scientific officer of ACT. "The DSMB approval is an important step towards confirming the safety of our cells. We hope that the treatment of the next patients will provide further evidence that our stem cell-derived RPE cells are a safe and effective treatment option for patients with a range of macular degenerative diseases such as Dry AMD and SMD."

The progress of disease in both Stargardt's Macular Dystrophy 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. The progressive loss of RPE cells in the macula leads to the eventual loss of photoreceptors. Over time, this can cause severe central vision deterioration and even blindness as the macula becomes less functional. ACT's therapeutic programs utilize transplanted hESC-derived RPE cells to treat these conditions by replacing the missing RPE cells in the patient's eyes before all RPE function and photoreceptor activity is lost.

"The outcome of this in-depth safety review by an unbiased team of experts is very reassuring," said Gary Rabin, chairman and CEO of ACT. "The DSMB, which includes independent experts in macular degeneration, has recommended that the study continue at the present dosage of cells. We hope that in the near future we will be able to share both safety and physiological data we are continuing to obtain in these trials. For now, it will have to suffice to say that we are extremely pleased and believe that these trials will validate our earlier pre-clinical findings. In the meantime, we have begun to set our sights on expanding our clinical trials to in Europe, having been recently approved to begin our SMD trial in the United Kingdom, and various countries in Asia. Our trials have captured the interest of some of the best eye hospitals and surgeons in the world, and it is our aim to bring that expertise to bear in our efforts to find treatment for these debilitating diseases."