The breakthrough is one more step in the development of a treatment for restoring vision in people with a variety of retinal diseases including retinitis pigmentosa and macular degeneration -- conditions that affect more than nine million Americans. While more work needs to be done before this transplantation approach can be studied in humans, the Foundation is strongly committed to moving this type of promising therapy into the clinic.

What's most noteworthy about the study is that the transplanted cells became full-fledged retinal cells and integrated into the host retina. Though scientists have previously performed retinal cell transplantation studies with varying success, never before have they achieved functional integration of photoreceptors-- the cells that provide vision.

Details of the transplantation study were published today in the journal Nature. Major media outlets including The Washington Post, Boston Globe, Fox News, and the BBC are reporting on the breakthrough.

"Over the past couple of years, various research teams have transplanted retinal cells and tissue into the eye and they survived. While these promising studies have advanced the field, photoreceptor development and functional integration have been elusive," says Stephen Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. "You need integration if you are going to fully repair damage and restore vision. This group of researchers has done just that. It's an important advancement."

The investigative team, which included Anand Swaroop, Ph.D., from the University of Michigan Kellogg Eye Center, used immature mouse retinal cells for transplantation. A key to their success was choosing cells that were at just the right developmental stage-- they are committed to becoming sight-giving photoreceptors and are also capable of integration.

The retinal cell transplantation study was successfully performed on three different mouse models of retinal degeneration.

The researchers used two different techniques to verify that the mice had restored vision.

"We've made significant progress toward bringing cellular therapy to the clinic," says Rose. "This new knowledge tells us that retinal cell transplantation is a viable strategy for restoring vision. We're excited to move this promising technique to the next level."

By Ben A. Shaberman
01/18/2007

The Foundation Fighting Blindness is building a roadmap to quickly move promising stem cell treatments into clinical trials.

Researchers funded by the Foundation Fighting Blindness are making major strides in moving stem cell therapies into the clinic to treat the entire spectrum of retinal degenerative diseases.

Specifically, they are having great success in preclinical studies of stem cell therapies to restore vision. For the first time, scientists used stem cells to make photoreceptor cells, which are essential for vision, and enabled them to integrate into the retina. This success brings us much closer to the reality of human clinical trials of stem cell therapies to save and restore vision.

The Foundation's goal is to significantly accelerate the progress being made in stem cell research. In collaboration with leading experts, the Foundation is drafting a tactical roadmap to more quickly move emerging treatments into clinical trials. The Foundation hosted a stem cell research meeting on December 14, 2006 in Washington, D.C., to present and discuss the latest stem cell research for retinal diseases, and begin drafting the roadmap. Meeting attendees included a dozen of the world's top researchers who are developing stem cell therapies to treat retinal degenerative diseases, as well as leading scientists who are advancing stem cell treatments for other neurological diseases.

Also present was Congressman Pete Session (R-TX), who is committed to working in congress and with other branches of the federal government to advance stem cell research. Congressman Sessions recognizes the promise of stem cells in the treatment of a wide range of diseases, illnesses, and injuries including: diabetes, Alzheimer's disease, Parkinson's disease, and spinal cord injuries.

The congressman set a hopeful and enthusiastic tone for the potential for stem cell therapies to save and restore vision in the near future when he noted that, "One month before the Wright Brothers flew, the New York Times quoted the Patent Office that said it would take 10,000 to 1,000,000 more years for man to fly." Congressman Sessions has a personal interest in supporting stem cell research, because he has a family member affected with retinitis pigmentosa.

During the meeting, the team of experts began drafting a two-part roadmap. Part one is focused on advancing therapies for saving and preserving the

By Warren King, Seattle Times
Medical Reporter

UW scientists reported that they have successfully used the stem cells to treat diseased tissue in mouse retinas, a key portion of the eye.

"This is a very promising development for cell replacement in the retina," said Tom Reh, UW professor of biological structure and leader of the research.

Reh said that if the stem-cell research at the UW and other institutions continues to be successful, the first human tests of the technique could begin in about two years.

The UW team used a mix of "growth factors" - natural proteins that encourage cell growth - to coax the embryonic cells into becoming retinal cells. It was the first use of human stem cells using

the technique for the retina; previous research has been conducted with mouse stem cells.

The embryonic cells used in the research were from a cell line existing before August 2001, when President Bush, citing ethical reasons, prohibited federal funding of research on future lines.

The retina, at the back of the eye, receives images and sends them to the brain through the optic nerve. Macular degeneration, deterioration of the centre of the retina, affects 10 million people in the United States and is the leading cause of blindness in the elderly, according to the National Eye Institute. Diabetic retinopathy, which damages retinal blood vessels, affects 5.3 million adults, according to the American Academy of Ophthalmology, and retinitis pigmentosa, a genetic disease, affects about 75,000 people.

The UW researchers, who have done extensive previous research on the retina, spent about two years working on their stem-cell research. Finding the right combination of growth factorsto develop the retina cells was the key.

"The idea was that we already have these proteins [growth factors] that already control development naturally," said Reh.

After growing the embryonic cells in the lab for several weeks, the researchers first placed them in growth factors important to head development in humans and mice. They then added another factor that other scientists have found leads to large eye development in frogs.

That combination stimulated the embryonic cells to become retinal "progenitor" cells, sort of the parents of retina cells. The development occurred in two weeks, about twice as fast as during normal development in the uterus.

Finally, when the scientists mixed the new cells with damaged mouse retina, the cells replaced key cells: cones, responsible for color perception; rods, which enable night vision; and amacrine cells, which form the other layer of the retina.
mouse embryonic cells for similar research, praised the "highly efficient" technique of the University of Washington team. "The results have profound implications for the future use of such cells in cell-replacement strategies to counteract retinal degeneration," Meyer said.

Reh said his team now have begun injecting the new cells into the eyes of retina-damaged mice, measuring nerve reactions to see whether there is actual vision improvement. He said the scientists also will be watching the work of a California company using the same cell line to generate cells for repair of spinal-cord injuries.

"If things continue to look good over the next six months and other research moves ahead, we should be in a position to use this for eye diseases," he said.

 Reh, along with Deepak Lamba, the lead author of the research report, and the UW team reported their findings in the week's online version of the Proceedings of the National Academy of Sciences.

Other University of Washington researchers also have been using stem cells to make important

Jason Meyer, a University of Wisconsin scientist who has used advances. In June, UW scientists reported that they had put human liver stem cells into mice, where they replaced dead liver cells. The research could lead to treatments for livers damaged by hepatitis,