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 three ongoing stem cell clinical trials in the United States and Europe has authorized the Company to move forward with enrolling and treating the patients in the next cohort of each of the trials. Per each trial’s protocol, the first patient of “cohort 3” will be injected with 150,000 human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells. This represents a fifty percent increase in the dose of RPE cells as compared to the previously treated cohort of patients. The first patients treated at this escalated dose will be evaluated, with interim DSMB review after six weeks, at which time the determination to continue treating additional patients at this dosage will be made. Additionally, the DSMB has authorized the Company to proceed with treating patients in the two U.S. trials as part of additional cohorts recently approved by the FDA. In particular, the FDA approved the addition of two new cohorts of four patients each – one cohort for each of the Stargardt’s Macular Dystrophy (SMD) and dry age-related macular degeneration (dry AMD) trials – which can include patients with better vision. The specific protocol for these “cohort 2a” patients establishes eligibility for enrollment based on visual acuity as good as 20/100. Previous patients enrolled in the trials have visual acuity no better than 20/400 and ranging to patients whose visual acuity had deteriorated to hand-motion only sight. In addition to establishing the safety of the transplanted RPE cells, the opportunity to treat patients earlier in the course of progression of these diseases improves the likelihood of enrolling patients having a greater number of photoreceptors that, while inactive due to loss of the native RPE layer, are dormant but able to be rescued by the reestablishment of a functional RPE layer from the transplanted cells. Enrollment of the additional patients as part of cohort 2a will be simultaneous with enrollment of patients in the 150,000 cell dose cohort 3 patients.
“With DSMB approval now secured, we look forward to proceeding with the third, higher-dosage cohort in all three trials in coming weeks, as well as initiating a separate cohort, 2a, of patients who represent earlier stages of these degenerative diseases,” commented Gary Rabin, chairman and CEO of ACT. “Moreover, we anticipate that the added cohort 2a patients may not only shed additional light on the safety and tolerability of our RPE cells, but offer us the opportunity to gather anatomical and functional data that can be used to help in the design and selection of endpoints for our eventual phase II studies.”The three clinical trials in the U.S. and Europe investigate hESC-derived RPE cells for the treatment of dry AMD and SMD, both forms of macular degeneration. These trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or SMD at 12 months, the study’s primary endpoint. With the addition of the new cohort 2a patients, each of the U.S. trials will now enroll a total of 16 patients across the ascending dosage format of 50,000 to 200,000 RPE cells. “We are pleased to receive the unanimous recommendation of the DSMB to initiate the third, higher-dosage patient treatments in the US and EU trials for Stargardt’s disease and dry AMD,” said Robert Lanza, M.D., chief scientific officer of ACT. “In addition, we are excited by the prospects of being able to work with better vision patients being enrolled in the 2a cohort. We think this provides a unique opportunity to gain preliminary data and insight from the treatment of patients that begin to resemble the early and intermediate stage dry AMD and SMD patients we hope to be able to treat in the next phase of these trials.”