The present and future of precision oncology and tumor-agnostic therapeutic approaches

Nakul M Shah1, Funda Meric-Bernstam2

  • 1Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, United States.

The Oncologist
|June 19, 2025
PubMed

Insights

Tumor-agnostic therapies, approved based on genetic biomarkers, are transforming precision oncology for advanced solid tumors. These treatments expand options for rare and refractory cancers by targeting shared alterations.

Area of Science:

  • Oncology
  • Genetics
  • Pharmacology

Background:

  • Precision oncology utilizes molecular profiling to guide cancer treatment.
  • Tumor-agnostic therapies target specific genetic alterations irrespective of tumor origin.
  • These therapies offer new hope for patients with rare or treatment-resistant cancers.

Purpose of the Study:

  • To review the landscape of FDA-approved tumor-agnostic therapies.
  • To analyze the clinical trial data supporting their approval and updated outcomes.
  • To discuss factors influencing efficacy, limitations, and future directions in precision oncology.

Main Methods:

  • Review of next-generation sequencing and molecular profiling advancements.
  • Analysis of Food and Drug Administration (FDA)-approved tumor-agnostic therapies.
  • Evaluation of clinical trial data, updated results, and observational studies.

Main Results:

  • Advances in sequencing enable identification of shared actionable alterations.
  • Tumor-agnostic therapies have expanded treatment options across various cancer types.
  • Efficacy is influenced by tumor histology, alteration type, and co-alterations, with challenges in predicting response and understanding resistance.

Conclusions:

  • Tumor-agnostic therapies represent a significant advancement in precision oncology.
  • Further research into resistance mechanisms and improved diagnostic tools is crucial.
  • Innovative strategies and trial designs will enhance future therapeutic options for cancer patients.

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