Overcoming clinical BCR-ABL1 compound mutant resistance with combined ponatinib and asciminib therapy

Cancer Cell. 2024 Sep 9;42(9):1486-1488. doi: 10.1016/j.ccell.2024.08.004. Epub 2024 Aug 29.

Abstract

BCR-ABL1 compound mutations can lead to resistance to ABL1 inhibitors in chronic myeloid leukemia (CML), which could be targeted by combining the ATP-site inhibitor ponatinib and the allosteric inhibitor asciminib. Here, we report the clinical validation of this approach in a CML patient, providing a basis for combination therapy to overcome such resistance.

Publication types

  • Case Reports
  • Letter

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / pharmacology
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Drug Resistance, Neoplasm* / drug effects
  • Drug Resistance, Neoplasm* / genetics
  • Female
  • Fusion Proteins, bcr-abl* / antagonists & inhibitors
  • Fusion Proteins, bcr-abl* / genetics
  • Humans
  • Imidazoles* / pharmacology
  • Imidazoles* / therapeutic use
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / drug therapy
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / genetics
  • Male
  • Mutation*
  • Niacinamide / analogs & derivatives
  • Protein Kinase Inhibitors* / pharmacology
  • Protein Kinase Inhibitors* / therapeutic use
  • Pyrazoles
  • Pyridazines* / pharmacology
  • Pyridazines* / therapeutic use

Substances

  • Pyridazines
  • ponatinib
  • asciminib
  • Fusion Proteins, bcr-abl
  • Imidazoles
  • Protein Kinase Inhibitors
  • BCR-ABL1 fusion protein, human
  • Niacinamide
  • Pyrazoles