Temozolomide-induced guanine mutations create exploitable vulnerabilities of guanine-rich DNA and RNA regions in drug-resistant gliomas

Sci Adv. 2022 Jun 24;8(25):eabn3471. doi: 10.1126/sciadv.abn3471. Epub 2022 Jun 22.

Abstract

Temozolomide (TMZ) is a chemotherapeutic agent that has been the first-line standard of care for the aggressive brain cancer glioblastoma (GBM) since 2005. Although initially beneficial, TMZ resistance is universal and second-line interventions are an unmet clinical need. Here, we took advantage of the known mechanism of action of TMZ to target guanines (G) and investigated G-rich G-quadruplex (G4) and splice site changes that occur upon TMZ resistance. We report that TMZ-resistant GBM has guanine mutations that disrupt the G-rich DNA G4s and splice sites that lead to deregulated alternative splicing. These alterations create vulnerabilities, which are selectively targeted by either the G4-stabilizing drug TMPyP4 or a novel splicing kinase inhibitor of cdc2-like kinase. Last, we show that the G4 and RNA binding protein EWSR1 aggregates in the cytoplasm in TMZ-resistant GBM cells and patient samples. Together, our findings provide insight into targetable vulnerabilities of TMZ-resistant GBM and present cytoplasmic EWSR1 as a putative biomarker.

MeSH terms

  • Antineoplastic Agents* / pharmacology
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / genetics
  • Brain Neoplasms* / metabolism
  • Cell Line, Tumor
  • DNA / pharmacology
  • Drug Resistance, Neoplasm / genetics
  • Glioblastoma* / metabolism
  • Guanine / pharmacology
  • Humans
  • Mutation
  • RNA
  • Temozolomide / pharmacology
  • Temozolomide / therapeutic use

Substances

  • Antineoplastic Agents
  • Guanine
  • RNA
  • DNA
  • Temozolomide