Using Stem Cells to Cure Blindness

Scientists are designing stem-cell-based therapies for degenerative retinal diseases.

Scientists are taking the first major step in using stem cells to replace retinal cells lost to degenerative eye diseases such as macular degeneration and retinitis pigmentosa. According to findings published today, researchers at the University of Washington in Seattle can reliably make retinal cells from embryonic stem cells. The researchers are now implanting the cells into blind animals to see if the cells can restore vision.

Stem-cell-based therapies could one day stop people with macular degeneration or other retinal diseases from losing their sight. (Source: Istockphoto.com/cgouin)

"This work is the first step toward retinal reconstitution," says Stephen Rose, chief research officer at the Foundation Fighting Blindness, a nonprofit funding agency based in Owings Mills, MD.

The retina is a layer of cells lining the back of the eye that contains specialized neurons, known as photoreceptors, to convert light into electrical signals, as well as other neurons, known as retinal ganglion cells, to send those messages to the brain. In age-related macular degeneration and retinitis pigmentosa, the photoreceptors degenerate over time, leading to loss of vision.

"Those are the diseases we think can be targeted by stem cells," says Thomas Reh, a developmental biologist at the University of Washington who led the work. "If we can replace the photoreceptors, we think we can restore vision."

Scientists have been attempting to transplant eye cells for decades. While they have had some success in animal models using cells derived from fetuses or other sources, there has been little progress in humans, largely because of a lack of cells. "Finding a fountain source of cells you can effectively get out of bottle and squirt into someone's eye is really the way to go," says Raymond Lund, a retinal cell expert at the Oregon Health and Science University in Portland, OR.

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Generating large numbers of retinal cells from embryonic stem cells could solve that problem. Achieving this feat with human cells has been difficult -- generating each type of tissue from stem cells requires its own special recipe, and some cell types are more difficult to make than others. Reh and his team used cues from normal eye development to find a unique mix of ingredients that trigger retinal cell development. The key, says Reh, is three proteins, called growth factors, known to be involved in head and eye development.

According to a paper published today in the Proceedings of the National Academy of Sciences, the researchers can reliably generate retinal progenitor cells, which then have the ability to turn into any cell type in the retina, such as photoreceptors, retinal ganglion cells, or other cells. Preliminary results show that when the cells are transplanted into retinas either in a dish or in live animals, the cells migrate to different layers of the retina and begin to express proteins characteristic of the resident cells, including photoreceptors. The researchers are also developing ways to efficiently turn the progenitor cells into photoreceptors in a dish.