Treating eye disease with stem cells

Advanced Cell Technology has filed an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) to initiate a Phase I/II multicenter study using embryonic-stem-cell-derived retinal cells to treat patients with Stargardt’s Macular Dystrophy (SMD). Degenerative retinal diseases are among the most common causes of untreatable blindness in the world and while Age-Related Macular Degeneration is the most common of these diseases, SMD is still one of the most common forms of juvenile macular blindness.

“Our research clearly shows that stem-cell-derived retinal cells can rescue visual function in animals that otherwise would have gone blind. We are hopeful that the cells will be similarly efficacious in patients,” said Dr Robert Lanza, ACT’s Chief Scientific Officer.

This treatment uses stem cells to re-create cells in the retina that support the photoreceptors. These cells, called retinal pigment epithelium, are often the first to die off in SMD and AMD, which in turn leads to loss of vision.