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Medical News & Perspectives
January 19, 2000

Researchers Buoyed by Promise of Targeted Leukemia Therapy

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JAMA. 2000;283(3):317-321. doi:10.1001/jama.283.3.317

New Orleans—The concept that tailoring therapy to the specific molecular abnormality responsible for a disease will lead to safer and more effective drugs received a dramatic boost with results reported from a study of a new agent that targets the genetic flaw causing chronic myelogenous leukemia (CML).

In the study, reported here at the annual meeting of the American Society of Hematology, all 31 patients with CML who were given a daily pill containing at least 300 mg of a drug called STI-571 had a complete normalization of their blood counts, indicating a remission of the disease. At the same time, the drug, which targets a tyrosine kinase enzyme found only in the leukemia cells, appeared to eliminate all cells containing the chromosomal abnormality in three patients and substantially reduced the number of such cells in another eight, said Brian J. Druker, MD, of Oregon Health Sciences University, Portland.

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