Ara-C suppresses H3 K27-altered spinal cord diffuse midline glioma growth and enhances immune checkpoint blockade sensitivity

Sci Adv. 2025 Apr 18;11(16):eadu3956. doi: 10.1126/sciadv.adu3956. Epub 2025 Apr 16.

Abstract

H3 K27-altered spinal cord diffuse midline glioma (H3-SCDMG) poses therapeutic challenges. Analysis of 73 clinical samples revealed heightened proliferation in H3-SCDMG versus wild-type tumors, suggesting therapeutic vulnerabilities. Drug screening identified cytarabine (Ara-C) as highly effective in inhibiting proliferation in H3 K27M cell models, recently established patient-derived cells, and patient-derived xenograft models. Mechanistically, Ara-C can suppress tumor growth through DNA damage, cell-cycle arrest, and apoptosis. An investigator-initiated clinical trial involving four patients showed benefits in three cases. In addition, a subset of cells exhibited senescence and senescence-associated secretory phenotype post-Ara-C treatment, accompanied by several immune checkpoint ligands' up-regulation and more immune cell infiltration. Combining Ara-C with dual Programmed cell death protein 1 (PD-1) and TIGIT blockade emerged as a promising strategy to disrupt immune evasion by senescent cells, enhancing antitumor responses. These findings highlight Ara-C's potential as a monotherapy and in synergy with immunotherapy for H3-SCDMG, offering potential strategies for clinical management.

MeSH terms

  • Animals
  • Apoptosis / drug effects
  • Cell Cycle Checkpoints / drug effects
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Cytarabine* / pharmacology
  • Cytarabine* / therapeutic use
  • Female
  • Glioma* / drug therapy
  • Glioma* / genetics
  • Glioma* / immunology
  • Glioma* / metabolism
  • Glioma* / pathology
  • Histones* / genetics
  • Histones* / metabolism
  • Humans
  • Immune Checkpoint Inhibitors* / pharmacology
  • Immune Checkpoint Inhibitors* / therapeutic use
  • Mice
  • Spinal Cord Neoplasms* / drug therapy
  • Spinal Cord Neoplasms* / genetics
  • Spinal Cord Neoplasms* / immunology
  • Spinal Cord Neoplasms* / metabolism
  • Spinal Cord Neoplasms* / pathology
  • Xenograft Model Antitumor Assays

Substances

  • Immune Checkpoint Inhibitors
  • Cytarabine
  • Histones