Inhibition of interleukin-1 receptor-associated kinase-1 is a therapeutic strategy for acute myeloid leukemia subtypes

Leukemia. 2018 Nov;32(11):2374-2387. doi: 10.1038/s41375-018-0112-2. Epub 2018 Mar 29.

Abstract

Interleukin-1 receptor-associated kinase 1 (IRAK1), an essential mediator of innate immunity and inflammatory responses, is constitutively active in multiple cancers. We evaluated the role of IRAK1 in acute myeloid leukemia (AML) and assessed the inhibitory activity of multikinase inhibitor pacritinib on IRAK1 in AML. We demonstrated that IRAK1 is overexpressed in AML and provides a survival signal to AML cells. Genetic knockdown of IRAK1 in primary AML samples and xenograft model showed a significant reduction in leukemia burden. Kinase profiling indicated pacritinib has potent inhibitory activity against IRAK1. Computational modeling combined with site-directed mutagenesis demonstrated high-affinity binding of pacritinib to the IRAK1 kinase domain. Pacritinib exposure reduced IRAK1 phosphorylation in AML cells. A higher percentage of primary AML samples showed robust sensitivity to pacritinib, which inhibits FLT3, JAK2, and IRAK1, relative to FLT3 inhibitor quizartinib or JAK1/2 inhibitor ruxolitinib, demonstrating the importance of IRAK1 inhibition. Pacritinib inhibited the growth of AML cells harboring a variety of genetic abnormalities not limited to FLT3 and JAK2. Pacritinib treatment reduced AML progenitors in vitro and the leukemia burden in AML xenograft model. Overall, IRAK1 contributes to the survival of leukemic cells, and the suppression of IRAK1 may be beneficial among heterogeneous AML subtypes.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Animals
  • Benzothiazoles / therapeutic use
  • Bridged-Ring Compounds / therapeutic use
  • Cell Line, Tumor
  • Child
  • Female
  • Humans
  • Interleukin-1 Receptor-Associated Kinases / metabolism*
  • Janus Kinase 2 / metabolism
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / metabolism*
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Middle Aged
  • Mutation / drug effects
  • Mutation / genetics
  • Nitriles
  • Phenylurea Compounds / therapeutic use
  • Protein Kinase Inhibitors / therapeutic use
  • Pyrazoles / therapeutic use
  • Pyrimidines / therapeutic use
  • Xenograft Model Antitumor Assays
  • Young Adult
  • fms-Like Tyrosine Kinase 3 / metabolism

Substances

  • 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene
  • Benzothiazoles
  • Bridged-Ring Compounds
  • Nitriles
  • Phenylurea Compounds
  • Protein Kinase Inhibitors
  • Pyrazoles
  • Pyrimidines
  • quizartinib
  • ruxolitinib
  • fms-Like Tyrosine Kinase 3
  • Janus Kinase 2
  • IRAK1 protein, human
  • Interleukin-1 Receptor-Associated Kinases