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Published: Jul 20, 2006 12:00 AM
Modified: Jul 20, 2006 05:27 AM

Study puts hope in stem cells

Duke researchers target scleroderma

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Sullivan then led a preliminary study that showed 19 of 33 scleroderma patients treated with such transplants saw no further progression of their disease.

Vulnerability is risky

Stem cell transplant as a therapy for autoimmune disease is not limited to scleroderma. Duke is participating in an NIH-funded clinical trial of 100 patients to test such transplants for lupus. NIH also is funding a preliminary study that will look at the transplants to treat multiple sclerosis.

Still, some doctors who treat autoimmune diseases are skeptical.

Wiping out patients' immune systems is dangerous because it makes them vulnerable to infections. A nasty bug can be deadly if it gets ahold of them before their immune systems regenerate. Five of the 33 scleroderma patients in Sullivan's preliminary study died of therapy-related causes. Just as many patients died from the effects of the disease.

"As a treating physician, how do you convey the risks?" said Dr. Nortin Hadler, a professor of medicine at UNC-Chapel Hill and attending rheumatologist at UNC Hospitals. He has an active caseload of about 100 scleroderma patients, some of whom come to him from outside North Carolina.

Still, he said, he is eager to see the trial's results.

"In the best of all worlds, [stem cell transplants] will work for the rapidly progressive group, and we'll see less and less of the end-stage disease," Hadler said.

In spite of his earlier work, Sullivan reserves judgment about which treatment being tested will be better. "There are compelling data for both camps," he said.

Williams, the patient from Louisville, thinks transplants will be more effective.

He was diagnosed with systemic scleroderma in May 2005, six months after his hands began to become red and swollen. By the time he came to Duke in August to discuss participating in the scleroderma trial, the disease had attacked his lungs, and his hands were so stiff he found it difficult to make a fist. He took Tylenol two or three times a day for joint pain.

Since his transplant, Williams hasn't needed pain medicine, and the tightness in his hands is getting better. He headed home to Louisville last week, where he will be monitored by a doctor there who is participating in the trial.

"I'm real hopeful," Williams said. "I feel like it's working."


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Staff writer Jean P. Fisher can be reached at 829-4753 or jfisher@newsobserver.com.
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