Going the Extra Mile: Compassionate Trial Helps Patients with Retinitis Pigmentosa

by Ginger Pyron

Try this: Before you recycle that next paper-towel roll, take a moment to peer through its corridor to the small circle of light.

Remember the long-ago fascination of peering through a cardboard tube? Your childhood eyes met a new perspective: all the wide, surrounding world reduced to just one small circle. For a few moments’ glimpse, that circle can intrigue and entertain. But as someone’s only option—and as a view certain to become smaller and smaller—such “tunnel vision” quickly loses all novelty.

Inherited degeneration of the retina, called retinitis pigmentosa (RP), increasingly narrows the field of vision. Night blindness comes first, then a gradual shrinking of the periphery; sight dwindles to the size of a cardboard-tube circle, then to a dot of light like that at the end of a straw, and progresses steadily toward blindness. And there’s no known treatment for it. Yet.

This big yet continues to motivate the retinal surgeons and scientists of Emory Eye Center, much to the recent benefit of 10 patients from across the U.S. and Mexico. Starting with a newly invented device and ending with newly aroused hope, these patients’ jubilant stories are ones in which everybody—from families to physicians—wins.

The initial trials

In 2005 the “NT-501,” a one-of-a-kind implant for RP patients, underwent a clinical trial conducted by its maker, Neurotech. The six-month trial was encouraging. Designed to provide long-term release of a therapeutic protein (ciliary neurotrophic factor, or CNTF) directly into the back of the eye via Neurotech’s patented Encapsulated Cell Technology (ECT), NT-501 showed promising results in halting the progression of RP among participants.

The next step: Test the implant again—and again. As Neurotech continued its second and third studies (2006-2007), Emory Eye Center agreed to conduct a separate “compassionate trial” for 10 people with varying degrees of RP. These men and women, selected by Foundation Fighting Blindness and Neurotech, urgently sought help for their condition; a number of them had not been eligible for an earlier trial. Expecting the typical eight- to nine-month time frame for the study, the Eye Center’s trial team later learned that new circumstances dictated a hard choice: Either complete the entire trial before December 20, or wait until the following August.

Compassion in action

From its Latin roots, the word compassion means “to suffer with.” For the sake of the 10 suffering and hopeful patients, and despite a December roster already crammed with appointments, Emory chose to push ahead with all speed.

Within one and a half months, Emory had maneuvered its way through a university approval process that usually takes six months to a year. And within a highly compressed schedule—during just two short weeks in December—the Eye Center’s team accomplished the almost impossible.
Before receiving the implant, each of the 10 patients had to undergo extensive examinations and testing that involved laborious documentation and spanned many hours each day. Three clinical staff members, working with good will, efficiency, and stamina little short of heroism, engineered the complex processing. If the Eye Center offered an award called “Grace Under Pressure,” the three hands-down winners for 2007 would be clinical trial coordinators Donna Leef, Stacey Andelman, and Alcides Fernandez. From faculty and patients alike, they could expect a standing ovation.

Shouldering the task of performing all 10 surgeries at a stepped-up pace, Eye Center retina specialists Jiong Yan and Daniel F. Martin inserted each implant, one day after another. Neurotech donated the implants, but Emory covered all other expenses of the trial, including the surgical costs.

Jiong Yan observes in retrospect, “Two weeks! That was an almost unbelievable time frame for this trial. But we knew why we had made this decision, and all of us were willing to do whatever was needed. Everything went perfectly. It was a triumph of teamwork.”

Results: win-win

Ten people, including those featured here, celebrated the end-of-year holidays with the gift of a permanent implant. They and their families entered 2008 with fresh new hope.

At Emory Eye Center, a team of dedicated specialists and staff members enjoyed not only a well-earned rest, but the assurance that their intensive two-week push furthered an ongoing mission: seeking to open the vision and the future of these 10 patients—and of countless others who will come later.

For people who see well, the disheartening progression of RP is hard to imagine. Try this: Before you recycle that next paper-towel roll, take a moment to peer through its corridor to the small circle of light. Then, during your next restaurant meal, look way down to the tiny bright spot at the end of your drinking straw. What if your vision had shrunk that much? And what if you knew that the circle would become even smaller? When you come back to your full vision, notice the rich peripheral details you enjoy. They’re so easy to take for granted—until the day you realize they’ve begun to recede.

Emory Eye Center exists to protect those life-enhancing details. We exist to help preserve vision for people like these lucky 10. In myriad and compassionate ways, we offer our resources to you.

Article from Emory Eye, Summer 2008, pp. 2-3.

Technician Stacey Andelman checks Meredith Tyree's vision.

The Hope of Watching My Children Grow Up

At age 16, with a history of night blindness and fender-benders, I got the doctor’s verdict: “You have retinitis pigmentosa, and there’s no cure. Start learning Braille, because by the time you’re 45, you’ll probably be blind.” My dad immediately began raising money for vision research. He told me, “We’re going to find a cure, Tiger.”

Now I’m 42, with two young sons, a household to run, and this new implant—which my father’s hope and dedication helped make possible. I won’t receive a visual field test for another few months, but I can tell that my clarity has improved.

For me, that’s an extra bonus. I have only 5% vision, and if I can hang onto even that much, it’s all I’d ask for.

I can’t say enough good things about the wonderful staff at Emory Eye Center. They stopped everything else to help us, working long days and all weekend. All of us patients were a little fearful, and they handled us with love and patience. We think they walk on water! By offering this trial, they’re giving us such a huge gift—the hope of watching our children grow up, of having independence and freedom.

And they do it so humbly.

Somewhere there’s another 16-year-old who’s just now hearing those shattering words: “You have RP.” I want her to know she doesn’t have to live just waiting for the worst to come. There’s promise, and there’s hope.

If my dad were alive today, he’d say, “I told you! We’re going to make it happen!”

Meredith Tyree is a mother and homemaker, Midlothian, Va.

Article from Emory Eye, Summer 2008, p. 7.