Faculty, Staff and Student Publications

Publication Date

6-4-2025

Journal

The Oncologist

DOI

10.1093/oncolo/oyaf152

PMID

40536268

PMCID

PMC12204399

PubMedCentral® Posted Date

6-19-2025

PubMedCentral® Full Text Version

Post-print

Abstract

Precision oncology has transformed the treatment landscape for patients with advanced solid tumors. Tumor-agnostic therapies, those that have been approved based on genetic mutations or biomarkers across tumor histology types, are important examples of how the implementation of precision oncology can expand therapeutic options for patients, especially those with rare cancer types and treatment-refractory disease. In this review, we first discuss how advances in next-generation sequencing and molecular profiling have enabled the identification of shared actionable alterations. Subsequently, we explore the current landscape of tumor-agnostic therapies that have received approval from the Food and Drug Administration. We discuss the strengths and limitations of these therapies and evaluate the clinical trial data leading to their approval. In addition, we detail updated results from these clinical trials and additional observational studies reported after the approval. Several factors such as tumor histology, specific alteration types, and the presence of co-alterations are associated with the efficacy of these therapies. Also, challenges remain in understanding resistance mechanisms and predicting response. Looking ahead, we discuss how improved diagnostic tools, novel experimental strategies, and innovative trial designs may further advance the field of precision oncology and improve therapeutic options for patients.

Keywords

Humans, Precision Medicine, Neoplasms, Medical Oncology, Biomarkers, Tumor, High-Throughput Nucleotide Sequencing, precision oncology, tumor-agnostic therapy, molecular profiling, targeted therapy, biomarker-driven treatment

Published Open-Access

yes

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