Single-cell sequencing reveals VEGFR as a potential target for CAR-T cell therapy in chordoma

Chordomas are rare bone tumors with poor prognosis upon recurrence. This study identifies potential immunotherapy targets on chordoma cells using single-cell RNA sequencing. The study synthesizes CAR-T cells targeting VEGFR and VEGFR/TGF-β, evaluating their antitumor activity in vitro, including in a primary chordoma organoid model. The findings suggest VEGFR as a potential target for CAR-T cell therapies in chordoma, with modulated TGF-β signaling enhancing CAR-T cell efficacy.

• 1
  Single-cell transcriptome sequencing identified VEGFR and TGF-β as therapeutic targets in chordoma.
• 2
  VRGFR CAR-T cells and VEGFR/TGF-β scFv CAR-T cells exhibited significant antitumor effects through CAR-T cell activation and cytokine secretion.
• 3
  VEGFR/TGF-β scFv CAR-T cells showed enhanced and sustained cytotoxicity of chordoma cell lines in vitro compared with VRGFR CAR-T cells.