Sorafenib promotes graft-versus-leukemia activity in mice and humans through IL-15 production in FLT3-ITD-mutant leukemia cells

Nat Med. 2018 Mar;24(3):282-291. doi: 10.1038/nm.4484. Epub 2018 Feb 12.

Abstract

Individuals with acute myeloid leukemia (AML) harboring an internal tandem duplication (ITD) in the gene encoding Fms-related tyrosine kinase 3 (FLT3) who relapse after allogeneic hematopoietic cell transplantation (allo-HCT) have a 1-year survival rate below 20%. We observed that sorafenib, a multitargeted tyrosine kinase inhibitor, increased IL-15 production by FLT3-ITD+ leukemia cells. This synergized with the allogeneic CD8+ T cell response, leading to long-term survival in six mouse models of FLT3-ITD+ AML. Sorafenib-related IL-15 production caused an increase in CD8+CD107a+IFN-γ+ T cells with features of longevity (high levels of Bcl-2 and reduced PD-1 levels), which eradicated leukemia in secondary recipients. Mechanistically, sorafenib reduced expression of the transcription factor ATF4, thereby blocking negative regulation of interferon regulatory factor 7 (IRF7) activation, which enhanced IL-15 transcription. Both IRF7 knockdown and ATF4 overexpression in leukemia cells antagonized sorafenib-induced IL-15 production in vitro. Human FLT3-ITD+ AML cells obtained from sorafenib responders following sorafenib therapy showed increased levels of IL-15, phosphorylated IRF7, and a transcriptionally active IRF7 chromatin state. The mitochondrial spare respiratory capacity and glycolytic capacity of CD8+ T cells increased upon sorafenib treatment in sorafenib responders but not in nonresponders. Our findings indicate that the synergism of T cells and sorafenib is mediated via reduced ATF4 expression, causing activation of the IRF7-IL-15 axis in leukemia cells and thereby leading to metabolic reprogramming of leukemia-reactive T cells in humans. Therefore, sorafenib treatment has the potential to contribute to an immune-mediated cure of FLT3-ITD-mutant AML relapse, an otherwise fatal complication after allo-HCT.

Publication types

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

MeSH terms

  • Activating Transcription Factor 4 / genetics*
  • Animals
  • CD8-Positive T-Lymphocytes / immunology
  • Cellular Reprogramming / genetics
  • Gene Expression Regulation, Neoplastic / drug effects
  • Graft vs Host Disease / drug therapy
  • Graft vs Host Disease / genetics
  • Graft vs Host Disease / pathology
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Humans
  • Interferon Regulatory Factor-7 / genetics*
  • Interleukin-15 / genetics*
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics
  • Leukemia, Myeloid, Acute / pathology
  • Mice
  • Sorafenib / administration & dosage
  • Sorafenib / adverse effects
  • Tandem Repeat Sequences / genetics
  • Transplantation, Homologous / adverse effects
  • fms-Like Tyrosine Kinase 3 / genetics*

Substances

  • ATF4 protein, human
  • IL15 protein, human
  • IRF7 protein, human
  • Interferon Regulatory Factor-7
  • Interleukin-15
  • Activating Transcription Factor 4
  • Sorafenib
  • FLT3 protein, human
  • fms-Like Tyrosine Kinase 3