In November 2010, the U.S. Food and Drug Administration (FDA) gave clearance for Advanced Cell Technology (ACT), based in Marlborough, Massachusetts, to commence Phase I/II clinical trials of a new therapy for Stargardt’s Macular Dystrophy, an incurable form of juvenile macular dystrophy that leads to blindness. Twelve patients will receive intraocular injections of healthy retinal cells derived from human embryonic stem cells (hESC). In animal studies, the therapy was effective in preventing further vision loss and restoring sight.
The ACT study is only the second clinical trial using hESC to gain approval. In January 2009, the FDA cleared Geron, based in Menlo Park, California, to begin Phase I trials using a hESC-based therapy to treat spinal cord injuries. Injecting oligodendrocyte progenitor cells derived from stem cells at the site of the injury was shown to stimulate nerve growth and significantly improve locomotion in an animal model.
While the use of stem cells in research remains controversial, the clinical studies will provide some clarity about the potential benefits of hESC-based therapies in humans. As William Caldwell, Chairman and CEO of ACT, remarked, “[w]ith the initiation of this clinical trial, and that of Geron’s earlier this fall, the field of regenerative medicine is poised to take embryonic stem cell therapies from the realm of nebulous potential to that of tangible and real treatments that will make a significant difference in the lives of millions of people worldwide. This is truly a ‘game changer’ for the medical community.”