Advances in the treatment of IDH-mutant gliomas

Curr Opin Neurol. 2024 Dec 1;37(6):708-716. doi: 10.1097/WCO.0000000000001316. Epub 2024 Sep 11.

Abstract

Purpose of review: Isocitrate dehydrogenase (IDH) mutation is a defining molecular driver of WHO grade 2-4 astrocytomas and oligodendrogliomas. In this article, we review the recent therapeutic approaches specifically targeting IDH-mutant gliomas and summarize ongoing clinical trials in this population.

Recent findings: The IDH inhibitor vorasidenib recently demonstrated its efficacy after surgical resection in grade 2 IDH-mutated gliomas. Several studies in patients with IDH-mutant gliomas are currently exploring various strategies to target IDH mutations, including the use of small-molecule inhibitors, immunotherapies, peptide vaccines and agents targeting metabolic and epigenomic vulnerabilities.

Summary: Mutant-IDH targeting holds significant promise in treating progressive or recurrent IDH-mutant gliomas. Recent results with IDH inhibitors will change practice and influence the existing guidelines in a near future.

Publication types

  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / genetics
  • Brain Neoplasms* / therapy
  • Glioma* / drug therapy
  • Glioma* / genetics
  • Glioma* / therapy
  • Humans
  • Isocitrate Dehydrogenase* / antagonists & inhibitors
  • Isocitrate Dehydrogenase* / genetics
  • Mutation*

Substances

  • Isocitrate Dehydrogenase
  • Antineoplastic Agents