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Enzyme Inhibitor May Cure Chronic Myeloid Leukemia

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Enzyme Inhibitor May Cure Chronic Myeloid Leukemia

Studies of a novel therapeutic drug, STI571, have demonstrated for the first time that we have the potential to develop an anti-cancer drug based on a specific molecular abnormality in a human cancer.

Chronic myeloid leukemia (CML) is a cancer of the blood cells characterized by the replacement of bone marrow with malignant, leukemic cells. These cells all possess the same specific chromosomal abnormality, a translocation between chromosomes 9 and 22 that results in the formation of a hybrid gene know as BCR-ABL. BCR-ABL codes for a constitutively activated tyrosine kinase, the expression of which leads to uncontrolled cell growth. BCR-ABL is present in virtually all cases of CML throughout the course of the disease, and in some cases of acute lymphoblastic leukemia (ALL). Phase-1, dose-escalating, clinical trials of STI571 in patients with CML, who had failed treatment with interferon alpha, showed startling results. Complete hematologic responses were observed in 53 of 54 patients who were treated with daily doses of 300 mg or more of STI571, and this response occurred in the first four weeks of treatment. Fifty-one of these 53 patients remain in remission.

Unfortunately, the results of the second study, on patients with more advanced forms of the disease, were not as promising. Twenty-one of 38 patients in myeloid blast crisis had hematologic responses to the drug, with 4 of these having a complete response. Seven of these patients continue to receive treatment and remain in remission. However, all but one of the patients with lymphoid blast crisis or ALL have relapsed.

Patients treated with STI571 experienced only limited side-effects including nausea, myalgias, edema and diarrhea.

Source

  1. Druker BJ, et al. New England Journal of Medicine 2001; 344:1031-37.
  2. Druker BJ, et al., New England Journal of Medicine 2001; 344:1038-42.
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