The company used retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESC), and said the patient is recovering uneventfully.
“So far, there have not been any complications or side effects due to the stem cell-derived RPE cells, and we will continue monitoring the patients for safety, tolerability and efficacy of this therapy,” said Gary Rabin, chairman and CEO of Advanced Cell Technology.
The dry AMD trial is one of three clinical trials the company is running in the United States and Europe. Each trial will enroll 12 patients, with cohorts of three patients in an ascending dosage format. The trials are designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or Stargardt’s macular dystrophy (SMD) at 12 months, the study’s primary endpoint.
Preliminary results relating to both early safety and biological function for the first two patients in the United States, one SMD patient and one dry AMD patient, were reported in The Lancet in January 2012.
According to Robert Lanza, chief scientific officer at Advanced Cell Technology, although the purpose of the trial is to test the safety of stem cells injected into the eye, both initial patients “had measurable improvement in their vision that persisted through the duration of the study.”
Although the two women’s sight had improved, both remain legally blind. Lanza cautioned that the findings were preliminary, and that the improvements could disappear and complications could emerge. Nevertheless, he thinks the cases will provide useful lessons for the field.
Dennis Lox, MD, a sports, physical and regenerative medicine specialist in the Tampa Bay area, said, “The field of stem cell research has expanded greatly in the United States. This trial, as well as other clinical trials underway, adds greatly to our overall understanding of stem cells and how they can be utilized to treat human conditions.”