Cancer Drug Helps Scleroderma Patients

They report fewer breathing problems, more energy, study finds

TUESDAY, May 24, 2005 (HealthDay News) -- A drug used for leukemia and certain other cancers also helps to treat the lung disease associated with scleroderma, a debilitating connective tissue disease, new research shows.

The drug, cyclophosphamide (Cytoxan, Neosar) improved the patients' breathing problems and boosted their energy levels, said study author Dr. Donald Tashkin, a professor of medicine at the David Geffen School of Medicine, University of California, Los Angeles (UCLA).

Tashkin presented the research Monday at the American Thoracic Society's international conference, in San Diego.

Scleroderma is a disease in which hardening of the skin is one of the most visible manifestations, according to the Scleroderma Foundation. About 300,000 people have the disease in the United States, according to foundation estimates. The exact cause is not known, but the disease can also affect the blood vessels and internal organs, Tashkin said.

"About 80 percent of these patients develop lung involvement," Tashkin said. Over time the lungs scar, making breathing difficult. This scarring is believed to be caused by inflammation, in which an abnormal number of white blood cells collect in the lungs, he explained.

The more severe the scarring, the higher the mortality rate, the UCLA expert added, and more than half of deaths from the disease are due to lung-related problems.

In the study, Tashkin and his colleagues assigned 162 patients with scleroderma, average age about 48, to either a group that got the drug for a year or another group that got a placebo. To be eligible for the study, the patients needed to have significant shortness of breath, meaning they became short of breath after walking just two or three flights of stairs, Tashkin said.

The team evaluated white blood cell counts and platelet counts, and checked lung function.

Lung function improved significantly in those on the drug compared with those taking placebo. "We also found a significant and meaningful effect on improving shortness of breath," he said.

When the patients answered a health assessment questionnaire, those on the drug also said they had more energy or "peppiness," he said.

How does the therapy work?

"Our initial hypothesis was that it suppresses inflammation," Tashkin said. "It may be doing more than that, we don't know. There is controversy about whether or not the scarring is necessarily related to the inflammation. I believe it is, but maybe not directly."

The drug probably has some additional, undiscovered effects, besides being anti-inflammatory, Tashkin said.

The patients receiving the drug did have more serious adverse effects than the placebo group: five people receiving the medicine got pneumonia, compared to one in the placebo group. In each group, two patients died, but not from drug toxicity, Tashkin said.

A spokeswoman for the Scleroderma Foundation praised the study. "That's phenomenal," Carolyn Weller, vice president of education and research for the foundation, said when told of the results about improved lung function.

"That gives our patient population [treatment] options with early lung disease," she added. Until now, the main option was prednisone, a corticosteroid. "And staying on high dosages [of prednisone] for long periods of time can have an effect on kidney function," she said.

According to Weller, some doctors are already using cyclophosphamide "off label" -- prescribing the drug for a purpose not specifically approved by the U.S. Food and Drug Administration -- to treat the scarring.

"This study will give doctors the data they need to be able to prescribe it on a wider basis," she said.

More information

To learn more about scleroderma, visit the Scleroderma Foundation.

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