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Promise and Progress - Experimental Drug Being Tested for Acute Myeloid Leukemia

Faces of Childhood Cancer

Experimental Drug Being Tested for Acute Myeloid Leukemia

Date: June 1, 2004

Blood, January 24, 2004

Investigators are testing an experimental drug that blocks the impact of a gene mutation commonly found in acute leukemia.

The drug, CEP-701, appears to cancel out the effect of mutations of the FLT3 gene, a primary culprit in an aggressive and treatment-resistant form of acute myeloid leukemia (AML). CEP-701 may improve the effectiveness of standard chemotherapy, and a clinical research trial combining the two is now under way for adult patients who have relapsed AML and the FLT3 gene mutation. Recent preliminary studies published in Blood ONLINE show five out of 14 patients treated with CEP-701 experienced decreases in leukemic cells in the bone marrow and circulating blood, and laboratory studies confirmed that the drug blocked the effect of the FLT3 mutation.

For more information about the study, contact B. Douglas Smith, M.D., at 410-614-5068 or

This research is funded by the National Cancer Institute, the Leukemia and Lymphoma Society and the Children’s Cancer Foundation.