Related Experiment Video
Updated: Sep 19, 2025

Integration of Wet and Dry Bench Processes Optimizes Targeted Next-generation Sequencing of Low-quality and Low-quantity Tumor Biopsies
Published on: April 11, 2016
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.
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.
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.
More Related Videos
10:27Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
09:37Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Related Concept Videos
Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
Tumor Immunotherapy
Treatment Resistant Cancers
Cancer Vaccines
Cancer vaccines come in two categories: preventive (prophylactic) and treatment (active). Preventive vaccines, such as the Human Papillomavirus (HPV) vaccine, protect against viruses that cause certain...
Microorganisms in Medicine and Therapeutics
Tumor Progression
Colon cancer is one of the best-documented examples of tumor progression. Early mutation in the APC gene in colon cells causes a small growth on the colon wall called a polyp. With time, this polyp grows into a benign, pre-cancerous tumor. Further...