Emerging therapies for inv(16) AML

Blood. 2021 May 13;137(19):2579-2584. doi: 10.1182/blood.2020009933.

Abstract

The core binding factor composed of CBFβ and RUNX subunits plays a critical role in most hematopoietic lineages and is deregulated in acute myeloid leukemia (AML). The fusion oncogene CBFβ-SMMHC expressed in AML with the chromosome inversion inv(16)(p13q22) acts as a driver oncogene in hematopoietic stem cells and induces AML. This review focuses on novel insights regarding the molecular mechanisms involved in CBFβ-SMMHC-driven leukemogenesis and recent advances in therapeutic approaches to target CBFβ-SMMHC in inv(16) AML.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents, Immunological / therapeutic use
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Cell Transformation, Neoplastic / genetics*
  • Chromosome Inversion*
  • Chromosomes, Human, Pair 16 / genetics*
  • Chromosomes, Human, Pair 16 / ultrastructure
  • Combined Modality Therapy
  • Core Binding Factor Alpha 2 Subunit / deficiency
  • Core Binding Factor Alpha 2 Subunit / metabolism
  • Core Binding Factor beta Subunit / genetics*
  • Core Binding Factor beta Subunit / physiology
  • Forecasting
  • Gemtuzumab / therapeutic use
  • Gene Expression Regulation, Leukemic
  • Gene Knock-In Techniques
  • Hematopoiesis / drug effects
  • Hematopoiesis / genetics
  • Humans
  • Immunotherapy / methods*
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Mice
  • Molecular Targeted Therapy*
  • Myosin Heavy Chains / genetics*
  • Neoplastic Stem Cells / drug effects
  • Neoplastic Stem Cells / metabolism
  • Neoplastic Stem Cells / pathology
  • Oncogene Proteins, Fusion / antagonists & inhibitors*
  • Oncogene Proteins, Fusion / genetics
  • Oncogene Proteins, Fusion / physiology
  • T-Lymphocytes / immunology
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents, Immunological
  • CBFB protein, human
  • CBFbeta-MYH11 fusion protein
  • Cbfb protein, mouse
  • Core Binding Factor Alpha 2 Subunit
  • Core Binding Factor beta Subunit
  • MYH11 protein, human
  • Oncogene Proteins, Fusion
  • RUNX1 protein, human
  • Runx1 protein, mouse
  • Gemtuzumab
  • Myosin Heavy Chains