Advanced Cell Technology, Inc. announced treatment of the fourth patient, the first in the second patient cohort, in its U.S. clinical trial for Stargardt's Macular Dystrophy (SMD).
The surgery was performed on Wednesday, July 11 at Wills Eye Institute in Philadelphia, by a surgical team lead by Carl D. Regillo, MD, FACS, director of the Wills Eye Clinical Retina Research Unit, attending surgeon in the Wills Eye Retina Service at the Wills Eye Institute, and professor of ophthalmology at Thomas Jefferson University. In keeping with trial protocol, the patient was injected with 100,000 human embryonic stem cell-derived retinal pigment epithelial (RPE) cells, as compared with the 50,000 cell dose used in the three patients of the first cohort. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.
"It is very gratifying to have second cohort, higher-dosage patient treatment underway in our U.S. clinical trial for SMD," commented Gary Rabin, chairman and CEO of ACT. "We are also pleased to be working with Dr. Regillo and his team at Wills Eye Institute, a truly first-class institution that is ranked as one of the best ophthalmology hospitals in the country by U.S. News & World Report."
Initiated in July of last year, the Phase I/II 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. As part of its RPE clinical program, the company is concurrently conducting a clinical trial for dry age-related macular degeneration and second trial for SMD in the United Kingdom.
"Doubling the cell dosage marks an important milestone in our clinical programs," said Robert Lanza, MD, ACT's chief scientific officer. "We look forward to continued progress and safety findings in the coming months, in both our U.S. and European trials."