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Experimental Drug Works in Chronic Leukemia

WebMD Health News

April 4, 2000 (San Francisco) -- Doctors say an experimental drug that showed extraordinary early success in controlling leukemia may also work in a rare but lethal brain cancer, and in a form of lung cancer.

Known under its research name of STI571, the drug, taken by mouth, is designed to interfere with a key substance in the body that is required for development of chronic myelogenous leukemia (CML). CML is a blood disease that strikes 4,500 people in the U.S. each year in which the body overproduces white blood cells. About a third of the people are candidates for bone marrow transplants, while the rest have to rely on the drug interferon. From time of diagnosis, the average person will die within four to six years.

But for patients participating in the STI571 studies, their white blood cells returned to normal levels if they were taking appropriate amounts of the drug -- about four tablets once a day, according to lead researcher Brian Druker, MD. Druker, associate professor of medicine at the Oregon Health Sciences University in Portland, presented his findings at a meeting of cancer researchers here.

"The patients are still taking the medication, about eight to 10 months later, because if we look closely in their bone marrow, we can still see evidence of the leukemia," Druker tells WebMD. The patients entered into the study had been diagnosed with CML for an average of three and a half years.

Druker explained that STI571 is designed to halt the disease from progressing. He said the pill also appears to be effective against a brain cancer called glioblastoma, and for small cell lung cancer as well.

"We are really witnessing an enormous payoff of the Unites States investment in cancer research," said William Hait, MD, director of the Cancer Institute of New Jersey in New Brunswick. "It is uncommon to see this kind of response in [the early stages of a] clinical trial. To see even a 25% response rate would be great. Because all these patients have been extensively treated with other medication, the hurdle is set very high to see a response ... These results are extraordinary."

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