Human Study Begins for First-of-its-Kind Stem Cell Treatment for AMD

August 08, 2013

The RIKEN Institute in Japan has started a clinical study for a wet age-related macular degeneration (AMD) treatment derived from induced pluripotent stem cells (iPSC) —  mature cells that have been genetically reprogrammed to a stem cell-like state. The therapy involves replacement of a layer of cells known as retinal pigment epithelial (RPE) cells, which provide critical waste and nutritional support for photoreceptors, but are damaged by leaky blood vessels caused by wet AMD. To produce new RPE cells, researchers will take skin cells from each patient, induce them to become stem cells, and then coax them forward to become RPE cells. This is the first-ever clinical trial of a potential retinal disease treatment derived from iPSC.

The first step in the treatment is removal of leaky blood vessels and the patient’s damaged RPE cells. New RPE cells will then be transplanted as a single layer adjacent to the photoreceptors — light-sensitive cells in the retina, which is how the cells normally exist. Researchers believe that transplanting a layer of RPE cells — as opposed to a bolus or clump — gives the cells the best chance to survive and function effectively. 

“RIKEN’s pioneering stem cell study is covering new and exciting ground in terms of the type of cells being used and the strategy for transplanting them in the retina,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “While we are always cautious about our expectations for an early-stage trial, this effort will inform the development of future therapies for retinal diseases.”

Discovered in 2007 by Nobel Prize recipient Shinya Yamanka, M.D., Ph.D., and Jamie Thomson, M.D., Ph.D., iPSC offer many of the benefits of embryonic stem cells. Namely, they can be made into virtually any cell type in the body, and can be easily replicated for use as therapies.  Also, when a patient receives his or her own iPSC-derived cells, researchers believe there will be less chance of rejection by the immune system.

Three other clinical trials of stem cell therapies for retinal diseases are currently underway. You can learn more about them through the following Foundation Fighting Blindness articles:

ACT Expands Human Studies of Stem-Cell Derived Retinal Treatment

StemCells, Inc. Launches Clinical Trial for Dry AMD Treatment

Bone Marrow Stem Cells in Clinical Trial for Retinal Diseases