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Updated: Aug 22, 2026

MicroRNA Amplification and Recognition through Locked-nucleic-acid In situ Hybridization as a Novel Detection and Quantification Method
Published on: October 7, 2025
Unlocking the potential of microRNA as predictive biomarkers for efficacy and adverse events in immune checkpoint
Xi Wang1, Baojun Duan2, Jun Bai2
1Department of Medical Oncology, Shaanxi Provincial People's Hospital, Xi'an, Shaanxi 710068, P.R. China.
Abstract:
Immune checkpoint inhibitors (ICIs) have shown promise in cancer therapy by enhancing antitumor immune responses, but patient responses are variable, with some experiencing limited efficacy and others suffering from severe immune‑related adverse events (irAEs). Identifying predictive biomarkers for ICI efficacy and toxicity is required for optimizing patient selection and treatment outcomes. MicroRNAs (miRNAs), small non‑coding RNA molecules that regulate gene expression, have emerged as potential biomarkers due to their role in immune regulation and tumor biology. Studies have shown that miRNA profiles in tumor tissues and blood can predict ICI responses. Specific miRNAs, such as miR‑155, miR‑21 and miR‑146a, are associated with enhanced antitumor responses, whereas others, such as miR‑34a and miR‑200c, are linked to resistance by modulating immune evasion. Additionally, altered miRNA expression reflects immune activation or dysregulation, playing a role in irAEs. The present review discussed the potential of miRNAs as predictive biomarkers for ICI therapy, their challenges in clinical integration and their promise for improving ICI treatment precision and safety, although further research is required for clinical application.
Insights
MicroRNAs (miRNAs) show promise as biomarkers for predicting patient responses to immune checkpoint inhibitors (ICIs) in cancer therapy. Further research is needed to integrate these biomarkers for safer and more effective ICI treatments.
Area of Science:
- Oncology
- Immunology
- Molecular Biology
Background:
- Immune checkpoint inhibitors (ICIs) offer promising cancer therapy by boosting antitumor immunity.
- However, patient responses to ICIs are highly variable, leading to inconsistent efficacy and immune-related adverse events (irAEs).
- Predictive biomarkers are crucial for optimizing patient selection and treatment strategies for ICIs.
Purpose of the Study:
- To review the potential of microRNAs (miRNAs) as predictive biomarkers for ICI efficacy and toxicity.
- To discuss the challenges and promise of integrating miRNAs into clinical practice for cancer immunotherapy.
Main Methods:
- Literature review of studies investigating miRNA profiles in relation to ICI therapy.
- Analysis of miRNA roles in immune regulation, tumor biology, and irAEs.
Main Results:
- miRNA profiles in tumor tissues and blood can predict patient responses to ICIs.
- Specific miRNAs (e.g., miR-155, miR-21, miR-146a) are linked to enhanced antitumor responses.
- Other miRNAs (e.g., miR-34a, miR-200c) are associated with resistance and immune evasion.
- Altered miRNA expression correlates with immune activation, dysregulation, and the development of irAEs.
Conclusions:
- miRNAs hold significant potential as predictive biomarkers for ICI therapy, aiding in patient stratification.
- Clinical integration of miRNA biomarkers faces challenges but promises to enhance precision and safety in cancer immunotherapy.
- Further research is essential to validate and implement miRNA-based strategies in clinical settings.
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