title: The acute promyelocytic leukaemia success story: curing leukaemia through targeted therapies.
abstract: The recent finding that almost all patients with acute promyelocytic leukaemia (APL) may be cured using a combination of retinoic acid (RA) and arsenic trioxide (As(2)O(3)) (N Engl J Med, 369, 2013 and 111) highlights the progress made in our understanding of APL pathogenesis and therapeutic approaches over the past 25 years. The study of APL has revealed many important lessons related to transcriptional control, nuclear organization, epigenetics and the role of proteolysis in biological control. Even more important has been the clinical demonstration that molecularly targeted therapy can eradicate disease.
authors.affiliation: Université Paris Diderot, Sorbonne Paris Cité, Hôpital St. Louis, Paris Cedex, France; INSERM UMR 944, Equipe labellisée par la Ligue Nationale contre le Cancer, Institut Universitaire d'Hématologie, Hôpital St. Louis, Paris Cedex, France; CNRS UMR 7212, Hôpital St. Louis, Paris Cedex, France.
authors.forename: K L
authors.initials: KL
authors.lastname: Rice
authors.forename: H
authors.initials: H
authors.lastname: de Thé
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D000077237:Arsenic Trioxide; D001152:Arsenicals / Q000008:administration & dosage; D002471:Cell Transformation, Neoplastic / Q000235:genetics; D044127:Epigenesis, Genetic / Q000502:physiology; D006801:Humans; D015473:Leukemia, Promyelocytic, Acute / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D058990:Molecular Targeted Therapy / Q000379:methods*; D010087:Oxides / Q000008:administration & dosage; D018160:Receptors, Cytoplasmic and Nuclear / Q000378:metabolism; D018168:Receptors, Retinoic Acid / Q000378:metabolism; D000072482:Retinoic Acid Receptor alpha; D047490:Retinoid X Receptor alpha / Q000378:metabolism; D015398:Signal Transduction / Q000502:physiology; D014212:Tretinoin / Q000008:administration & dosage
chemical_list: D001152:Arsenicals; D010087:Oxides; C000607071:RARA protein, human; D018160:Receptors, Cytoplasmic and Nuclear; D018168:Receptors, Retinoic Acid; D000072482:Retinoic Acid Receptor alpha; D047490:Retinoid X Receptor alpha; D014212:Tretinoin; D000077237:Arsenic Trioxide
keywords: APL; PML; arsenic trioxide; retinoic acid