title: Small molecule inhibitors in acute myeloid leukemia: from the bench to the clinic.
abstract: Many patients with acute myeloid leukemia will eventually develop refractory or relapsed disease. In the absence of standard therapy for this population, there is currently an urgent unmet need for novel therapeutic agents. Targeted therapy with small molecule inhibitors represents a new therapeutic intervention that has been successful for the treatment of multiple tumors (e.g., gastrointestinal stromal tumors, chronic myelogenous leukemia). Hence, there has been great interest in generating selective small molecule inhibitors targeting critical pathways of proliferation and survival in acute myeloid leukemia. This review highlights a selective group of intriguing therapeutic agents and their presumed targets in both preclinical models and in early human clinical trials.
authors.affiliation: Department of Medicine, Division of Oncology, Washington University School of Medicine, St Louis, Missouri, USA.
authors.forename: Muneera
authors.initials: M
authors.lastname: Al-Hussaini
authors.forename: John F
authors.initials: JF
authors.lastname: DiPersio
mesh_terms: D000818:Animals; D004791:Enzyme Inhibitors / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality; D051379:Mice; D009364:Neoplasm Recurrence, Local; D009687:Nuclear Proteins; D000090243:Nucleophosmin; D011379:Prognosis; D016019:Survival Analysis
chemical_list: D004791:Enzyme Inhibitors; D009687:Nuclear Proteins; D000090243:Nucleophosmin
keywords: acute myeloid leukemia; small molecule inhibitor; therapeutic agent