Physicians at Duke University Medical Center are leading a national study to learn whether stem cell transplants can correct defective immune systems in patients with scleroderma, a disabling and sometimes deadly disease.

If they work, such transplants would be the first therapy to treat -- and possibly even reverse -- scleroderma. Current treatments just ease its symptoms.

Success also could lead to effective treatment for other autoimmune diseases, which affect more than 20 million Americans.

Scleroderma, like all autoimmune diseases, develops when a person's immune system malfunctions in response to an unknown trigger and attacks body tissue.

"We've used scleroderma because it's the toughest case," said Dr. Keith Sullivan, a Duke oncologist who is lead investigator in the clinical trial. Duke is one of seven transplant centers nationwide participating in the trial and the only one in the Southeast.

With scleroderma, the immune system targets connective tissues throughout the skin and internal organs, causing a cascade of symptoms that can include pain, inflammation, hardened skin and failure of the lungs, kidneys and other organs. Scleroderma is often minor, and it is not contagious.

But about 100,000 people in the United States suffer systemic disease that rapidly spreads throughout their bodies. About half of them die within five years of diagnosis, Sullivan said.

The cause of the disease is not known, though physicians think a combination of genetics and exposure to certain chemicals or environmental contaminants is probably responsible. Doctors also suspect the female hormone estrogen plays a role in triggering it, which could explain why scleroderma occurs three to four times more often in women.

Other autoimmune diseases include lupus, Type 1 diabetes, rheumatoid arthritis and multiple sclerosis. As many as 23.5 million Americans have an autoimmune disorder, according to the National Institutes of Health.

The seven-year scleroderma study, supported by a $20.5 million grant from the NIH, will compare two possible treatments in 226 patients.

Half the participants will get total body radiation and chemotherapy to kill their flawed immune systems. Then they will receive a transplant of stem cells collected from their own blood. The hope is that the stem cells, which have the unique ability to become different types of cells or tissue, will generate a new immune system that does not attack the connective tissue.

The other half of trial participants will get monthly infusions of cyclophosphamide, a cancer drug that is often used to treat scleroderma symptoms. Patients will get higher doses and for a longer duration than commonly prescribed. The goal is to suppress the immune system so that it behaves less aggressively and symptoms slow or stop.

Stephen Williams, 46, who had a stem cell transplant at Duke in May, is optimistic the treatment may allow him to continue as a social worker. Except for the time he took for the transplant, the Louisville, Ky., resident has not missed work because of his scleroderma. But the disease is affecting his lungs, causing him to wonder when he might be disabled.

"My disease seemed to come on pretty quickly," he said. "The hope is that [the stem cell transplant] can improve or reverse it."

Sullivan stumbled onto the idea of treating autoimmune disease with stem cell transplants while practicing at the Fred Hutchinson Cancer Research Center in Seattle in the mid-1990s. Blood cancer patients who got transplants, and who also suffered from autoimmune disease, reported that their autoimmune symptoms eased or appeared to go away.

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