Cephalon's anticancer compound CEP-701 (lestaurtinib) inhibits a specific genetic mutation targeted by the compound in patients' blood samples and were predictive of a positive clinical response, according to preliminary data from a Phase II study of patients with acute myelogenous leukemia (AML) who bear a FLT3 activating mutation at first relapse from standard induction chemotherapy. The early data suggest that chemotherapy followed by the oral compound CEP-701 may offer a clinical benefit compared to chemotherapy alone.
CEP-701 is under development as a treatment for FLT3-mutated AML, a hematologic cancer characterized by uncontrolled growth of myeloid cells of the blood and bone marrow. According to the American Cancer Society, an estimated 12,000 people in the U.S. will be diagnosed with AML in 2005. Approximately 25 to 30 percent of AML patients have a FLT3 genetic mutation that is associated with a poorer prognosis for relapse and survival.