Biomarkers of immune checkpoint inhibitor response and toxicity: Challenges and opportunities

Immunol Rev. 2023 Sep;318(1):157-166. doi: 10.1111/imr.13249. Epub 2023 Jul 20.

Abstract

Immune checkpoint inhibitors have transformed cancer therapy, but their optimal use is still constrained by lack of response and toxicity. Biomarkers of response may facilitate drug development by allowing appropriate therapy selection and focusing clinical trial enrollment. However, aside from PD-L1 staining in a subset of tumors and rarely mismatch repair deficiency, no biomarkers are routinely used in the clinic. In addition, severe toxicities may cause severe morbidity, therapy discontinuation, and even death. Here, we review the state of the field with a focus on our research in therapeutic biomarkers and toxicities from immune checkpoint inhibitors.

Keywords: MHC-II; biomarkers; immune checkpoint inhibitors; immune-related adverse events; immunotherapy; tumor mutational burden.

Publication types

  • Review
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biomarkers
  • Biomarkers, Tumor
  • Brain Neoplasms*
  • Colorectal Neoplasms*
  • Humans
  • Immune Checkpoint Inhibitors / adverse effects
  • Immunotherapy / adverse effects
  • Neoplastic Syndromes, Hereditary*

Substances

  • Immune Checkpoint Inhibitors
  • Biomarkers
  • Biomarkers, Tumor