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Restoring sight and movement

Macular degeneration experts Erik van Kuijk, M.D., Ph.D., and Deborah Ferrington, Ph.D., are part of the University team charged with finding a cure for the debilitating eye disease. (Photo: Scott Streble)

Anonymous gift targets cures for macular degeneration and spinal cord injuries

She had $10 million to give and knew what she wanted from it: no frills, no fuss, no recognition—just a cure for macular degeneration and a way to repair spinal cord injuries.

So she’s throwing her support behind two research efforts at the University of Minnesota by creating endowments through her estate plans—$8 million for macular degeneration research in the Department of Ophthalmology and Visual Neurosciences and $2 million for stem cell research focused on repairing the spinal cord after injury.

The donor has supported both areas of research in the past with smaller amounts. But now, after having built trust in the researchers, she’s giving their projects a major vote of confidence.

The University has a proven track record of translating promising laboratory breakthroughs into new treatments for patients through its constellation of experts, facilities, and technologies. It’s a longtime leader in stem cell therapies, too, having performed the world’s first bone marrow transplant (a type of stem cell therapy) in 1968.

So what better place to pursue the next generation of cures?

Repairing vision — together

Age-related macular degeneration (AMD) is a leading cause of vision loss in older adults, according to the National Eye Institute. People with AMD see a dark, blurry spot at the center of their field of vision, making everyday activities like reading, driving, and recognizing faces difficult.

There’s no known cure for AMD, which gets progressively worse. But University researchers hope to change that. Under the direction of Erik van Kuijk, M.D., Ph.D., head of the Department of Ophthalmology and Visual Neurosciences, a multidisciplinary team of ophthalmologists, biochemists, neuroscientists, immunologists, electrophysiologists, and imaging experts is tackling this enormous task in partnership with the Stem Cell Institute.

For starters, scientist Deborah Ferrington, Ph.D., is testing several promising compounds to learn whether they can prevent AMD from worsening or possibly even prevent the disease altogether. The anonymous donor gave $150,000 earlier this year to jump-start the work.

The most successful compounds will later be tested in mice that model human AMD, and eventually the best option(s) will be refined and studied in people who have AMD.

The Minnesota Lions Eye Bank at the University has been a huge asset to Ferrington’s work, she says. While most eye banks use the corneas from deceased donors’ eyes for sight-restoring transplants and discard the rest of the tissue, in Minnesota, that excess tissue is saved for macular degeneration research.

“I know of only a handful of eye banks that are set up to do this,” Ferrington says.

Ultimately, there’s a good chance that stem cell therapies might contribute to regenerating the parts of the eye damaged by AMD, van Kuijk says.

Get moving again

While AMD typically affects older adults, most people who suffer traumatic spinal cord injuries are under 30 years old, according to the Centers for Disease Control and Prevention. There were about 200,000 people in the United States living with spinal cord injuries as of 2010, and 12,000 to 20,000 more people suffer these injuries every year.

University neurosurgeon Ann Parr, M.D., Ph.D., and her lab team are steadily working toward a clinical therapy for people who have spinal cord injuries by using human skin cells to create oligodendrocytes, cells that form the myelin that insulates the nerves, brain, and spinal cord.

Myelin is lost after injury, Parr says, but the neurons can still be preserved. The theory is that oligodendrocytes could be given back to patients to restore myelin, allowing the nerves to function again.

Parr’s team is in the process of proving that theory true. With a previous gift of $1 million from the same anonymous donor, Parr used mouse skin cells to make oligodendrocytes and then transplanted those cells into rats to see if their bodies could make myelin. Indeed they could, Parr’s lab tests have shown. (The team is also doing similar experiments using oligodendrocytes from human skin cells.) Additional studies will evaluate whether the rats’ mobility improves as well.

Stem cell scientist Ann Parr, M.D., Ph.D., and her lab team are working toward a clinical therapy for people who have suffered spinal cord injuries. (Photo: Scott Streble)

Using it wisely

Neither Parr nor van Kuijk—nor anyone on their research teams—knows who the anonymous donor is. But as they pursue answers in their labs, they’re thinking about the best ways to use her money as she intended.

From a practical standpoint, the endowments the donor is creating provide a permanent and sustained source of funding for the research.

“What we need to do on the other end is make sure that the science we do continues to be rigorous,” van Kuijk says.

And from a personal standpoint, being in charge of a research project specifically selected by a donor comes with a special responsibility, Parr says.

“It’s an honor to be chosen like that,” she says. “We have certainly done our best to make sure that her donation has the maximum impact.”

By Nicole Endres

To learn more about supporting research in age-related macular degeneration or spinal cord repair, contact Chuck Semrow at 612-624-6313 or csemrow@umn.edu.

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