Immunomodulatory effects of pevonedistat, a NEDD8-activating enzyme inhibitor, in chronic lymphocytic leukemia-derived T cells

Leukemia. 2021 Jan;35(1):156-168. doi: 10.1038/s41375-020-0794-0. Epub 2020 Mar 16.

Abstract

Novel targeted agents used in therapy of lymphoid malignancies, such as inhibitors of B-cell receptor-associated kinases, are recognized to have complex immune-mediated effects. NEDD8-activating enzyme (NAE) has been identified as a tractable target in chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma. We and others have shown that pevonedistat (TAK-924), a small-molecule inhibitor of NAE, abrogates NF-κB signaling in malignant B cells. However, NF-κB pathway activity is indispensable in immune response, and T-cell function is altered in patients with CLL. Using T cells derived from patients with CLL, we demonstrate that although targeting NAE results in markedly differential expression of NF-κB-regulated genes and downregulation of interleukin (IL)-2 signaling during T-cell activation, T cells evade apoptosis. Meanwhile, NAE inhibition favorably modulates polarization of T cells in vitro, with decreased Treg differentiation and a shift toward TH1 phenotype, accompanied by increased interferon-γ production. These findings were recapitulated in vivo in immunocompetent mouse models. T cells exposed to pevonedistat in washout experiments, informed by its human pharmacokinetic profile, recover NAE activity, and maintain their response to T-cell receptor stimulation and cytotoxic potential. Our data shed light on the potential immune implications of targeting neddylation in CLL and lymphoid malignancies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / therapeutic use
  • Cell Line, Tumor
  • Cyclopentanes / pharmacology*
  • Cyclopentanes / therapeutic use
  • Enzyme Inhibitors / pharmacology
  • Enzyme Inhibitors / therapeutic use
  • Humans
  • Immunomodulation / drug effects*
  • Leukemia, Prolymphocytic, T-Cell / drug therapy
  • Leukemia, Prolymphocytic, T-Cell / immunology*
  • Leukemia, Prolymphocytic, T-Cell / metabolism*
  • Leukemia, Prolymphocytic, T-Cell / pathology
  • Lymphocyte Activation / drug effects
  • Lymphocyte Activation / immunology
  • Models, Biological
  • NEDD8 Protein / antagonists & inhibitors*
  • NEDD8 Protein / metabolism*
  • Pyrimidines / pharmacology*
  • Pyrimidines / therapeutic use
  • Receptors, Antigen, T-Cell / metabolism
  • Signal Transduction
  • T-Lymphocytes / drug effects
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism
  • T-Lymphocytes / pathology
  • T-Lymphocytes, Helper-Inducer / drug effects
  • T-Lymphocytes, Helper-Inducer / immunology
  • T-Lymphocytes, Helper-Inducer / metabolism

Substances

  • Antineoplastic Agents
  • Cyclopentanes
  • Enzyme Inhibitors
  • NEDD8 Protein
  • NEDD8 protein, human
  • Pyrimidines
  • Receptors, Antigen, T-Cell
  • pevonedistat