Treating Leukemias, Myelodysplasia, and Myeloproliferative Disorders
The Adult Leukemia Group is responsible for the diagnosis and treatment of patients with acute and chronic leukemia, myelodysplastic syndrome (MDS), and other stem cell hematopoietic disorders. Our clinical research centers around phase I, II, and III clinical trials in these patients.We recently studied a series of patients with idiopathic hypereosinophilic syndrome (IHES). In collaboration with Drs. Cools and Gilliland, we identified a target gene, the FIP1L1-PDGFRA fusion gene, which is a consequence of an interstitial chromosomal deletion. This finding has led to the explanation of the molecular basis of IHES.Our group is currently working on the development of small molecules to treat acute and chronic leukemias and myelodysplasia. We are testing several small molecules in patients with relapsed or refractory acute myelogenous leukemia, studying the compounds PKC-412, CEP-701, and MLN-518 that specifically inhibit FLT3. Mutations in the FLT3 gene, which encodes a receptor tyrosine kinase, are thought to confer a poor prognosis in patients with acute myelogenous leukemia. We also have clinical trials under way that test histone deacetylase inhibitors in advanced hematologic malignancies. Our approach is to bring novel therapeutic agents to the forefront in the treatment of patients with acute and chronic leukemia, MDS, and myeloproliferative disorders.In addition, our group has been focused on the treatment of acute lymphoblastic leukemia (ALL). We are the lead coordinating site for a large clinical trial using dose-intense pediatric-like regimens for adult patients with ALL. We are also testing novel Notch inhibitor molecules for patients with relapsed or refractory T-cell ALL.