Advanced Cell Technology, Inc. (OTCBB:ACTC) announced today that it 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). Among the most common causes of untreatable blindness in the world are degenerative diseases of the retina. As many as 10 million people in the United States have photoreceptor degenerative disease. While most of these patients have Age-Related Macular Degeneration (AMD), a smaller number of patients have Stargardt’s, an Orphan disease and one of the most common forms of juvenile macular blindness. The treatment for eye disease uses stem cells to re-create a type of cell in the retina that supports the photoreceptors needed for vision. These cells, called retinal pigment epithelium (RPE), are often the first to die off in SMD and AMD, which in turn leads to loss of vision.
While there is currently no treatment for SMD, several years ago ACT and its collaborators found that human embryonic stem cells could be a source of RPE cells. Subsequent studies found that the cells could restore vision in animal models of macular degeneration. In the Royal College of Surgeon (RCS) rat model, implantation of RPE cells resulted in 100% improvement in visual performance over untreated controls without any adverse effects. The cells survived for more than 220 days and sustained extensive photoreceptor rescue. Functional rescue was also achieved in the ‘Stargardt’s’ mouse with near-normal functional measurements recorded at more than 70 days.
“It has been over a decade since human embryonic stem cells were first discovered,” said Dr. Robert Lanza, ACT’s Chief Scientific Officer. “The field desperately needs a big clinical success. After years of research and political debate, we’re finally on the verge of showing the potential clinical value of embryonic stem cell research. 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.”