Chronic myeloid leukemia (CML) is diagnosed in about 6,000 people annually in the USA, with 20-30% of them dying within 2 years. There are three progressive phases - an initial chronic phase, an accelerated phase, and the blast crisis. The leukemic cells contain the Philadelphia chromosome, which is associated with generation of an oncogenic protein BCR-ABL. A new drug, STI571, is a fairly specific blocker of BCR-ABL tyrosine kinase activity, and was therefore considered ideal for trial in CML patients and in Ph-positive ALL patients. The results were remarkable.
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