Tumour-specific STING agonist synthesis via a two-component prodrug system

Nat Chem. 2025 Dec;17(12):1941-1951. doi: 10.1038/s41557-025-01930-9. Epub 2025 Sep 16.

Abstract

Pharmacological activation of STING holds promise in cancer treatment. A recent trend is the development of tumour-specific or conditionally activated STING agonists for enhanced safety and efficacy. Here we explore an unconventional prodrug activation strategy for on-tumour synthesis of a potent agonist. Leveraging the unique mechanism of MSA2, a small-molecule agonist that dimerizes non-covalently before binding to STING, we showed that its analogues bearing reactive functional groups readily and selectively form covalent dimers under mild conditions and in complex environments. We identified a reacting pair that led to a thioether-linked dimer with submicromolar potency in cell-based assays. Caging one of the reactants with a self-immolative β-glucuronide moiety resulted in a two-component prodrug system that near-exclusively formed the active compounds in tumours overexpressing β-glucuronidase. These results exemplify the use of small-molecule recognition for on-site generation of active compounds from benign precursors.

MeSH terms

  • Animals
  • Antineoplastic Agents* / chemical synthesis
  • Antineoplastic Agents* / chemistry
  • Antineoplastic Agents* / pharmacology
  • Cell Line, Tumor
  • Humans
  • Membrane Proteins* / agonists
  • Membrane Proteins* / metabolism
  • Neoplasms* / drug therapy
  • Neoplasms* / metabolism
  • Prodrugs* / chemical synthesis
  • Prodrugs* / chemistry
  • Prodrugs* / pharmacology

Substances

  • Prodrugs
  • STING1 protein, human
  • Membrane Proteins
  • Antineoplastic Agents