title: Preclinical and clinical development of deferitrin, a novel, orally available iron chelator.
abstract: Deferitrin, a novel, orally available iron chelator, is in the early stage of clinical development for the treatment of chronic iron overload due to transfusional therapy. Preclinical pharmacology studies demonstrate iron excretion largely by the fecal route. Initial clinical studies have shown deferitrin to be well absorbed. Further clinical studies are ongoing to determine the efficiency and safety of deferitrin.
authors.affiliation: Genzyme, Drug Discovery and Development, 153 Second Ave., Waltham, MA 02492, USA. joanne.donovanmd@genzyme.com
authors.forename: Joanne M
authors.initials: JM
authors.lastname: Donovan
authors.forename: Melissa
authors.initials: M
authors.lastname: Plone
authors.forename: Rafif
authors.initials: R
authors.lastname: Dagher
authors.forename: Mark
authors.initials: M
authors.lastname: Bree
authors.forename: Judith
authors.initials: J
authors.lastname: Marquis
mesh_terms: D000284:Administration, Oral; D000818:Animals; D002264:Carboxylic Acids / Q000008:administration & dosage / Q000493:pharmacokinetics / Q000627:therapeutic use* / Q000633:toxicity; D015913:Chelation Therapy; D017321:Clinical Trials, Phase I as Topic; D004285:Dogs; D004353:Drug Evaluation, Preclinical; D005243:Feces / Q000737:chemistry; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000493:pharmacokinetics / Q000627:therapeutic use* / Q000633:toxicity; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008252:Macaca fascicularis; D015394:Molecular Structure; D051381:Rats; D013844:Thiazoles / Q000008:administration & dosage / Q000493:pharmacokinetics / Q000627:therapeutic use* / Q000633:toxicity; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy*
chemical_list: C120073:4'-hydroxydesazadesferrithiocin; D002264:Carboxylic Acids; D007502:Iron Chelating Agents; D013844:Thiazoles; D007501:Iron