New directions for emerging therapies in acute myeloid leukemia: the next chapter

Blood Cancer J. 2020 Oct 30;10(10):107. doi: 10.1038/s41408-020-00376-1.

Abstract

Conventional therapy for acute myeloid leukemia is composed of remission induction with cytarabine- and anthracycline-containing regimens, followed by consolidation therapy, including allogeneic stem cell transplantation, to prolong remission. In recent years, there has been a significant shift toward the use of novel and effective, target-directed therapies, including inhibitors of mutant FMS-like tyrosine kinase 3 (FLT3) and isocitrate dehydrogenase (IDH), the B-cell lymphoma 2 inhibitor venetoclax, and the hedgehog pathway inhibitor glasdegib. In older patients the combination of a hypomethylating agent or low-dose cytarabine, venetoclax achieved composite response rates that approximate those seen with standard induction regimens in similar populations, but with potentially less toxicity and early mortality. Preclinical data suggest synergy between venetoclax and FLT3- and IDH-targeted therapies, and doublets of venetoclax with inhibitors targeting these mutations have shown promising clinical activity in early stage trials. Triplet regimens involving the hypomethylating agent and venetoclax with FLT3 or IDH1/2 inhibitor, the TP53-modulating agent APR-246 and magrolimab, myeloid cell leukemia-1 inhibitors, or immune therapies such as CD123 antibody-drug conjugates and programmed cell death protein 1 inhibitors are currently being evaluated. It is hoped that such triplets, when applied in appropriate patient subsets, will further enhance remission rates, and more importantly remission durations and survival.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Allografts
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Benzimidazoles / therapeutic use
  • Bridged Bicyclo Compounds, Heterocyclic / therapeutic use
  • Cytarabine / therapeutic use
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Leukemia, Myeloid, Acute / mortality
  • Leukemia, Myeloid, Acute / therapy*
  • Phenylurea Compounds / therapeutic use
  • Quinuclidines / therapeutic use
  • Sulfonamides / therapeutic use

Substances

  • Benzimidazoles
  • Bridged Bicyclo Compounds, Heterocyclic
  • Phenylurea Compounds
  • Quinuclidines
  • Sulfonamides
  • Cytarabine
  • glasdegib
  • venetoclax
  • eprenetapopt