July 16, 2004.
By Lois M. Collins,
Deseret Morning News.
Cells in the leg may hold the cure for people with retinitis pigmentosa, a vision-robbing eye disease. And the University of Utah's Moran Eye Center is moving cautiously toward clinical trials to see if what works in a rat will work in a human.
Rats and people get many of the same diseases, including eye diseases, said Raymond D. Lund, Ph.D., director of research at Moran. "So they are really nice models."
Retinitis pigmentosa, often called RP, is often characterized by a ring of vision loss in mid-periphery, with small islands of vision in very far periphery, according to the Foundation Fighting Blindness. Other patients liken the eye disease to seeing through a straw. Some retain a small degree of central vision.
Researchers hope their efforts will eventually provide treatment not only for RP but for other eye diseases such as macular degeneration.
The center's current research builds on a foundation laid by others. For instance, it has been shown that putting certain naturally occurring chemicals into the eyes can stop the damage wrought by certain diseases. The goal has been finding the cells that make the right chemicals.
Researchers found that cells around the nerve fibers in the arms and legs make many of those chemicals, so about five years ago such cells were first put into the backs of rats' eyes to see what would happen.
"Incredibly, they worked," said Lund.
Lund's group published groundbreaking research two years ago showing that healthy cells transplanted into the eyes could stop the deterioration.
Working with a group from Miami using the same cells for spinal cord injury, the U. researchers transitioned to human cells in their rat studies. Again, it worked. And a group of researchers in Canada was able to show that the treated rats "see well. They can retain good vision over a period of at least six months," the duration of the particular study, Lund said.
The next logical step is human trials, he said. "I'm thinking it's nice to show all these sorts of things in rats, but we're not in the business of curing rats with blinding diseases. It would be nice if we could take it to patients."
Now a $100,000 grant from the Stephen A. and Elaine Wynn Charitable Foundation in Las Vegas will "give us the opportunity to first finish necessary bits of work we would like to do in small animals, particularly things like safety," Lund said, then take the experiments further.
"We want to transition toward clinical trials but not get ahead of ourselves," he said. "Still, I'm surprised. We're closer (to human trials) than I thought we would be."
Among the questions they want to answer before beginning human studies are whether there are unknown safety issues, how late in an animal's life transplantation is effective (some of the eye diseases they hope to cure primarily affect the elderly) and how to take what they've learned in the rat lab into a human clinic.
Once they know those answers, Lund said, they'll begin planning their first very small safety trial in humans.
Stephen Wynn serves on the Moran advisory board.
© 2004 Deseret News Publishing Company.
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