Tumor Mutational Burden as a Predictor of Immunotherapy Response: Is More Always Better?
Journal Article (Journal Article)
Immune checkpoint inhibitors, including antibodies that block programmed cell death protein-1 (PD-1) and PD-L1, have transformed the management of many cancers. However, the majority of patients have primary or acquired resistance to these immunotherapies. There is a significant unmet need for predictive biomarkers that can reliably identify patients who derive a clinically meaningful response from PD-1/PD-L1 blockade. High tumor mutational burden (TMB-H) has shown promise as a biomarker in lung cancer, but the broad applicability of TMB-H as a biomarker of response across all solid tumors is unclear. The FDA has approved the PD-1 inhibitor, pembrolizumab, as a therapy for all solid tumors with TMB equal to or greater than 10 mutations/megabase as measured by the FoundationOne CDx assay. This approval was based on an exploratory analysis of the KEYNOTE-158 study, which was a single-arm, phase II multi-cohort study of pembrolizumab for select, previously treated advanced solid tumors. Here, we elucidate the caveats of using TMB as a biomarker with a universal threshold across all solid tumors. While we recognize the importance of this and other FDA pan-cancer approvals, several questions about TMB as a predictive biomarker remain unanswered. In this perspective, we discuss clinical trial evidence in this area. We review the relationship between TMB and the tumor immune microenvironment. We highlight the risks of extrapolating evidence from a limited number of tumor histologies to all solid tumors, and we propose avenues for future research.
Full Text
Duke Authors
Cited Authors
- Strickler, JH; Hanks, BA; Khasraw, M
Published Date
- March 1, 2021
Published In
Volume / Issue
- 27 / 5
Start / End Page
- 1236 - 1241
PubMed ID
- 33199494
Pubmed Central ID
- PMC9912042
Electronic International Standard Serial Number (EISSN)
- 1557-3265
Digital Object Identifier (DOI)
- 10.1158/1078-0432.CCR-20-3054
Language
- eng
Conference Location
- United States