Advanced Cell Technology, Inc. announced treatment of the final patient in the first patient cohort in its Phase 1/2 clinical trial for Stargardt's macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The surgery was performed on Friday, July 27 at Moorfields Eye Hospital in London, the same site as the first two treatments, by a team of surgeons led by Professor James Bainbridge, consultant surgeon at Moorfields and Chair of Retinal Studies at University College London. The outpatient transplant surgery was performed successfully without any complications, and the patient is recovering uneventfully. This is the tenth patient overall to now be treated with the RPE cell therapy developed by the company.
"Our European trial is making very steady progress, having now completed enrollment of the first patient cohort," commented Gary Rabin, chairman and CEO. "We are very encouraged and look forward to receiving clearance to initiate the treatment of the second patient cohort in the coming weeks."
The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the study's primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format. It is similar in design to the U.S. trial for SMD that was initiated in July 2011.
"This is a significant month for the company," continued Mr. Rabin. "One year ago we treated the first of our patients in our two U.S. clinical trials. The one-year follow-up for those initial patients indicates that the improvements in visual acuity we initially reported have in fact persisted now for a year. Indeed, we are consistently observing improvements in subjective and objective visual acuity for patients being treated at the various clinical centers involved in our trials. Again, these trials are still at very early stages, but these preliminary results indicate that we are on the right track."
The European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially designated ACT's human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells as an orphan medicinal product for the treatment of SMD.