Eradicating acute myeloid leukemia in a Mll(PTD/wt):Flt3(ITD/wt) murine model: a path to novel therapeutic approaches for human disease

Blood. 2013 Nov 28;122(23):3778-83. doi: 10.1182/blood-2013-06-507426. Epub 2013 Oct 1.

Abstract

The coexpression of the MLL partial tandem duplication (PTD) and the FLT3 internal tandem duplication (ITD) mutations associate with a poor outcome in cytogenetically normal acute myeloid leukemia (AML). In mice, a double knock-in (dKI) of Mll(PTD/wt) and Flt3(ITD/wt) mutations induces spontaneous AML with an increase in DNA methyltransferases (Dnmt1, 3a, and 3b) and global DNA methylation index, thereby recapitulating its human AML counterpart. We determined that a regulator of Dnmts, miR-29b, is downregulated in bone marrow of dKI AML mice. Bortezomib exerted a dose-dependent increase in miR-29b expression in AML blasts ex vivo, followed by decreased Dnmts, reduced proliferation, and increased apoptosis. In vivo, bortezomib was not active against dKI AML, yet liposomal-encapsulated bortezomib, as a single agent, reversed downregulation of miR-29b in vivo and induced a long-term (90-day) disease-free remission in 80% of dKI AML mice that exhibited high leukemic burden at the start of therapy, yet showed no signs of relapse at autopsy. Taken together, these data support that liposomal bortezomib, as a single agent, eradicates Mll(PTD/wt):Flt3(ITD/wt) AML in mouse and may represent a powerful and potentially curative approach to high-risk human disease.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / administration & dosage
  • Boronic Acids / administration & dosage
  • Bortezomib
  • DNA Methylation
  • Drug Carriers
  • Histone-Lysine N-Methyltransferase / genetics*
  • Humans
  • Leukemia, Experimental / genetics
  • Leukemia, Experimental / metabolism
  • Leukemia, Experimental / therapy
  • Leukemia, Myeloid, Acute / genetics*
  • Leukemia, Myeloid, Acute / metabolism
  • Leukemia, Myeloid, Acute / therapy*
  • Liposomes
  • Mice
  • Mice, Mutant Strains
  • MicroRNAs / genetics
  • MicroRNAs / metabolism
  • Mutation
  • Myeloid-Lymphoid Leukemia Protein / genetics*
  • Proteasome Inhibitors / administration & dosage
  • Pyrazines / administration & dosage
  • RNA, Neoplasm / genetics
  • RNA, Neoplasm / metabolism
  • Tandem Repeat Sequences
  • fms-Like Tyrosine Kinase 3 / genetics*

Substances

  • Antineoplastic Agents
  • Boronic Acids
  • Drug Carriers
  • Liposomes
  • MIRN29 microRNA, mouse
  • MicroRNAs
  • Proteasome Inhibitors
  • Pyrazines
  • RNA, Neoplasm
  • Myeloid-Lymphoid Leukemia Protein
  • Bortezomib
  • Histone-Lysine N-Methyltransferase
  • Kmt2a protein, mouse
  • Flt3 protein, mouse
  • fms-Like Tyrosine Kinase 3