title: Elacytarabine (CP-4055) in the treatment of acute myeloid leukemia.
abstract: Elacytarabine (formerly CP-4055) is a lipid-conjugated derivative of the nucleoside analog cytarabine. Elacytarabine was rationally designed to circumvent cytarabine resistance related to decreased cellular uptake, due to the ability of the lipophilic drug moiety to enter the cell without the requirement of specialized nuclear transport proteins, including the hENT1. In preclinical and clinical studies, elacytarabine has demonstrated both safety and efficacy in acute myeloid leukemia (AML), with noteworthy activity among the cytarabine-refractory AML population. Elacytarabine was granted orphan drug designation status from the European Commission in 2007 and from the US FDA in 2008, with a fast-track approval designation from the FDA in 2010. Results of a recent randomized Phase III clinical trial, however, failed to show superiority of elacytarabine over the investigator's choice of therapy for relapsed or refractory AML.
authors.affiliation: Department of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Courtney D
authors.initials: CD
authors.lastname: DiNardo
authors.forename: Susan
authors.initials: S
authors.lastname: O'Brien
authors.forename: Varsha V
authors.initials: VV
authors.lastname: Gandhi
authors.forename: Farhad
authors.initials: F
authors.lastname: Ravandi
mesh_terms: D003561:Cytarabine / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000493:pharmacokinetics; D017277:Drug Approval; D019008:Drug Resistance, Neoplasm / Q000235:genetics; D064420:Drug-Related Side Effects and Adverse Reactions; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology; D016032:Randomized Controlled Trials as Topic; D016896:Treatment Outcome
chemical_list: D003561:Cytarabine; C047645:5'-oleoyl cytarabine