Cancer-causing drug safe and effective in treating eye disease

February 05, 2002

Ophthalmologists at the University of Illinois Medical Center at Chicago have found a highly effective treatment for uveitis, a potentially blinding inflammation of the uvea, the pigmented layers of the eye.

In patients with intractable, sight-threatening, non-infectious uveitis, the drug chlorambucil, a chemotherapeutic agent known to cause cancer in some patients when dispensed long-term, was found effective in alleviating inflammation when administered in high doses for a short time.

The design of the study was a chart review of 53 patients who had been treated at the medical center with high-dose, short-term chlorambucil between 1973 and 1999. With an average follow-up period of four years, 77 percent of the treated patients experienced no recurrence of uveitis, and all patients remained cancer-free. The results appear in the February issue of Ophthalmology, the clinical journal of the American Academy of Ophthalmology.

"The exciting thing about chlorambucil is that it seems to reduce the inflammation in many patients for the long term," said lead author Dr. Debra Goldstein, assistant professor of ophthalmology at the UIC College of Medicine and associate director of the medical center's uveitis service. "Many patients treated with other drugs, such as prednisone and cyclosporine, relapse as soon as the treatment is stopped."

However, Goldstein cautioned, because of the carcinogenic nature of chlorambucil, patients need to be closely monitored.

"Only physicians specializing in or very familiar with the treatment of uveitis and the use of immunosuppressive agents should utilize this treatment," she said. "The treating ophthalmologist may want to work in concert with another physician comfortable with the use of agents such as chlorambucil."

Uveitis is a condition that can be enigmatic and attributable to any number of causes, both infectious and non-infectious. Chlorambucil is used only in non-infectious cases, which are more common.

In less severe cases, uveitis can be controlled by traditional therapies. However, when it is sight-threatening, uveitis is difficult to treat, and many patients find it hard to cope with the side effects of long-term steroid therapy.

Although chlorambucil may become the preferred treatment for patients with severe, non-infectious uveitis, the authors don't recommend the drug for long-term use. What is gratifying, Goldstein said, is that most of the patients treated with chlorambucil went into remission after a short course of therapy.

About 2.3 million people in the United States suffer from ocular inflammatory disorders such as uveitis, according to statistics on blindness and blinding diseases compiled by the University of Washington. Uveitis is estimated to cause 10 percent to 15 percent of blindness in this country.
Other authors on the paper are Drs. Fernandino Fontanilla, Suruchi Kaul, Ozlem Sahin and Howard Tessler, all of UIC.

For more information about UIC, visit

University of Illinois at Chicago

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