Midostaurin, enasidenib, CPX-351, gemtuzumab ozogamicin, and venetoclax bring new hope to AML

Blood. 2017 Dec 7;130(23):2469-2474. doi: 10.1182/blood-2017-08-784066. Epub 2017 Oct 19.

Abstract

In 2017, 4 drugs received US Food and Drug Administration marketing approval for acute myeloid leukemia (AML) treatment: targeted therapies for mutant FLT3 and IDH2, a liposomal cytarabine-daunorubicin formulation for therapy-related AML and AML with myelodysplasia-related changes, and resurgence of an antibody-drug conjugate designed to target CD33. Promising results also emerged for the BCL-2 inhibitor venetoclax combined with low-intensity therapy in older patients unfit for intensive chemotherapy. This quintet of new drugs is likely to reshape the therapeutic landscape of AML.

Publication types

  • Review

MeSH terms

  • Aminoglycosides / pharmacology
  • Aminoglycosides / therapeutic use*
  • Aminopyridines / pharmacology
  • Aminopyridines / therapeutic use*
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use*
  • Clinical Trials as Topic
  • Cytarabine / administration & dosage*
  • Daunorubicin / administration & dosage*
  • Gemtuzumab
  • Humans
  • Isocitrate Dehydrogenase / antagonists & inhibitors
  • Isocitrate Dehydrogenase / genetics
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / metabolism
  • Leukemia, Myeloid, Acute / mortality
  • Liposomes
  • Molecular Targeted Therapy*
  • Mutation
  • Proto-Oncogene Proteins c-bcl-2 / antagonists & inhibitors
  • Sialic Acid Binding Ig-like Lectin 3 / antagonists & inhibitors
  • Staurosporine / analogs & derivatives*
  • Staurosporine / pharmacology
  • Staurosporine / therapeutic use
  • Sulfonamides / pharmacology
  • Sulfonamides / therapeutic use*
  • Treatment Outcome
  • Triazines / pharmacology
  • Triazines / therapeutic use*
  • fms-Like Tyrosine Kinase 3 / antagonists & inhibitors
  • fms-Like Tyrosine Kinase 3 / genetics

Substances

  • Aminoglycosides
  • Aminopyridines
  • Antibodies, Monoclonal, Humanized
  • Bridged Bicyclo Compounds, Heterocyclic
  • CD33 protein, human
  • Liposomes
  • Proto-Oncogene Proteins c-bcl-2
  • Sialic Acid Binding Ig-like Lectin 3
  • Sulfonamides
  • Triazines
  • Cytarabine
  • enasidenib
  • Gemtuzumab
  • IDH2 protein, human
  • Isocitrate Dehydrogenase
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3
  • Staurosporine
  • midostaurin
  • venetoclax
  • Daunorubicin