Related Experiment Video

Updated: Dec 18, 2025

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
10:27

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts

Published on: July 25, 2020

7.7K

Developing Drugs for Tissue-Agnostic Indications: A Paradigm Shift in Leveraging Cancer Biology for Precision

Nathan D Seligson1,2, Todd C Knepper3, Susanne Ragg4

  • 1Department of Pharmacotherapy and Translational Research, University of Florida, Jacksonville, Florida, USA.

Clinical Pharmacology and Therapeutics
|June 15, 2020
PubMed

Insights

Tissue-agnostic cancer therapies, like pembrolizumab and NTRK inhibitors, offer precision medicine. Future development faces challenges in complex biology and resistance mechanisms.

Area of Science:

  • Oncology
  • Precision Medicine
  • Cancer Therapeutics

Background:

  • Targeted therapies have revolutionized cancer treatment.
  • Biomarker-driven, tissue-agnostic trials represent a major advance in precision cancer medicine.
  • Few tissue-agnostic indications have received US Food and Drug Administration (FDA) approval.

Purpose of the Study:

  • To review the history and development of tissue-agnostic cancer therapies.
  • To compare the approval pathways of key tissue-agnostic drugs.
  • To explore future directions and challenges in this therapeutic area.

Main Methods:

  • Review of historical data on tissue-agnostic therapy development.
  • Comparative analysis of FDA-approved tissue-agnostic indications (e.g., pembrolizumab, larotrectinib, entrectinib).
  • Discussion of challenges including cancer biology, clinical trial design, and resistance.

Main Results:

  • Pembrolizumab (for MSI-high/dMMR solid tumors) and NTRK inhibitors (larotrectinib, entrectinib) are approved for tissue-agnostic use.
  • These approvals highlight a shift towards biomarker-driven, tumor-agnostic treatment strategies.
  • Significant hurdles remain for broader adoption and development.

Conclusions:

  • Tissue-agnostic therapies are a growing frontier in precision oncology.
  • Overcoming complex biology and identifying resistance mechanisms are critical for future success.
  • Continued research and innovative clinical trial designs are essential for advancing tissue-agnostic cancer therapeutics.

More Related Videos

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

2.0K
Integration of Wet and Dry Bench Processes Optimizes Targeted Next-generation Sequencing of Low-quality and Low-quantity Tumor Biopsies13:24

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

12.2K

Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
8.5K
Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
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...
5.8K
Cancer02:18

Cancer

Cancers arise due to mutations in genes involved in the regulation of cell division, which leads to unrestricted cell proliferation. Modern science and medicine have made great strides in the understanding and treatment of cancer, including eradicating cancer in some patients. However, there is still no cure for cancer. This is largely due to the fact that cancer is a large group of many diseases.
53.2K