AC220 is an investigational drug which works by blocking certain targets on cancer cells (enzymes called “receptor tyrosine kinases,” or RTKs). It appears to work best in patients with leukemia who have mutations in the FLT3 gene. Patients with this mutation are less likely to go into remission and more likely to experience a recurrence of their cancer.
In this study, researchers are determining the highest dose of AC220 that can be given safely with the standard drugs cytarabine and etoposide in children with acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML) that has returned or persists despite prior therapy, and to assess its preliminary effectiveness. This study is being conducted by the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) Consortium.