For only the second time, the Food and Drug Administration approved a company's request to test an embryonic stem cell-based therapy on human patients. Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing its retinal cell treatment this year in a dozen patients with Stargardt's macular dystrophy, an inherited degenerative eye disease that leads to blindness in children. In July, the FDA released its hold on the first trial of an embryonic stem cell based treatment, for spinal cord injury.
ACT's trial will involve injecting retinal pigment epithelium (RPE) cells, which nurture the retina, into volunteers with the most advanced forms of Stargardt's, in an attempt to replace dying and no longer functioning photoreceptor cells. In animals, the infusion of healthy cells improved vision and rescued the function of some diseased cells.