SUMOylation inhibitor TAK-981 (subasumstat) synergizes with 5-azacytidine in preclinical models of acute myeloid leukemia

Haematologica. 2024 Jan 1;109(1):98-114. doi: 10.3324/haematol.2023.282704.

Abstract

Acute myeloid leukemias (AML) are severe hematomalignancies with dismal prognosis. The post-translational modification SUMOylation plays key roles in leukemogenesis and AML response to therapies. Here, we show that TAK-981 (subasumstat), a first-in-class SUMOylation inhibitor, is endowed with potent anti-leukemic activity in various preclinical models of AML. TAK-981 targets AML cell lines and patient blast cells in vitro and in vivo in xenografted mice with minimal toxicity on normal hematopoietic cells. Moreover, it synergizes with 5-azacytidine (AZA), a DNA-hypomethylating agent now used in combination with the BCL-2 inhibitor venetoclax to treat AML patients unfit for standard chemotherapies. Interestingly, TAK-981+AZA combination shows higher anti-leukemic activity than AZA+venetoclax combination both in vitro and in vivo, at least in the models tested. Mechanistically, TAK-981 potentiates the transcriptional reprogramming induced by AZA, promoting apoptosis, alteration of the cell cycle and differentiation of the leukemic cells. In addition, TAK-981+AZA treatment induces many genes linked to inflammation and immune response pathways. In particular, this leads to the secretion of type-I interferon by AML cells. Finally, TAK-981+AZA induces the expression of natural killer-activating ligands (MICA/B) and adhesion proteins (ICAM-1) at the surface of AML cells. Consistently, TAK-981+AZA-treated AML cells activate natural killer cells and increase their cytotoxic activity. Targeting SUMOylation with TAK-981 may thus be a promising strategy to both sensitize AML cells to AZA and reduce their immune-escape capacities.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents* / therapeutic use
  • Azacitidine / pharmacology
  • Azacitidine / therapeutic use
  • Humans
  • Leukemia, Myeloid, Acute* / genetics
  • Mice
  • Sulfonamides / pharmacology
  • Sulfonamides / therapeutic use
  • Sumoylation

Substances

  • Azacitidine
  • venetoclax
  • Sulfonamides
  • Antineoplastic Agents

Grants and funding

Funding: Funding was provided by the CNRS, the Ligue Nationale contre le Cancer (to DA, MC, MP), the Fondation pour la Recherche Médicale (contract FDM201906008566, LG), INCa_16072, the Fédération Leucémie Espoir, the Association Laurette Fugain, the Fondation ARC pour la recherche sur le cancer and the Agence Nationale pour la Recherche (“Investissements d’avenir” program; ANR-16-IDEX-0006; project SUMOLAM). The HEMODIAG_2020 collection was funded by the Montpellier University Hospital, the Montpellier SIRIC and the Languedoc Roussillon Region. MGX acknowledges financial support from the France Génomique National infrastructure, funded as part of “Investissements d’Avenir” program managed by the Agence Nationale pour la Recherche (contract ANR 10 INBS 09).