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Updated: Jun 26, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Agnostic drug development revisited
Alberto Hernando-Calvo1, Alice Rossi2, Maria Vieito1
1Department of Medical Oncology, Vall d́Hebron Barcelona Hospital Campus, Barcelona, Spain; Vall d́Hebron Institute of Oncology, Barcelona, Spain.
Abstract:
The advent of molecular profiling and the generalization of next generation sequencing in oncology has enabled the identification of patients who could benefit from targeted agents. Since the tumor-agnostic approval of pembrolizumab for patients with MSI-High tumors in 2017, different molecularly-guided therapeutics have been awarded approvals and progressively incorporated in the treatment landscape across multiple tumor types. As the number of tumor-agnostic targets considered druggable expands in the clinic, novel challenges will reshape the drug development field involving all the stakeholders in oncology. In this review, we provide an overview of current tumor-agnostic approvals and discuss promising candidate therapeutics for tumor-agnostic designation and challenges for their broad implementation.
Insights
Molecular profiling and next-generation sequencing identify patients for targeted cancer therapies. This review covers current tumor-agnostic approvals and future challenges in drug development.
Area of Science:
- Oncology
- Genomics
- Pharmacology
Background:
- Molecular profiling and next-generation sequencing (NGS) have revolutionized oncology.
- Tumor-agnostic drug approvals, like pembrolizumab for MSI-High tumors, are increasing.
- Targeted therapies are becoming a cornerstone of cancer treatment.
Purpose of the Study:
- To review current tumor-agnostic drug approvals in oncology.
- To identify promising candidate therapeutics for tumor-agnostic designation.
- To discuss challenges in the broad implementation of these therapies.
Main Methods:
- Literature review of current tumor-agnostic approvals.
- Analysis of emerging molecular targets and candidate therapeutics.
- Discussion of drug development and clinical implementation challenges.
Main Results:
- An overview of FDA-approved tumor-agnostic therapies is presented.
- Several promising molecular targets and associated therapeutics are highlighted.
- Key challenges in drug development and clinical integration are identified.
Conclusions:
- The landscape of tumor-agnostic cancer therapy is rapidly evolving.
- Addressing challenges in drug development and implementation is crucial for expanding patient access.
- Continued research and collaboration are essential for advancing precision oncology.
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