Mechanism-guided drug repurposing against therapeutically actionable resistance domains in cancer: from resistance biology to clinical translation

Tumor resistance remains a central obstacle in cancer treatment. It blunts the activity of otherwise effective agents and narrows the therapeutic window, so that patients may accrue toxicity without commensurate benefit, and it drives progression and relapse. Resistance is rarely a single lesion; it is the emergent product of interacting programs that limit intracellular drug accumulation through ATP-binding cassette (ABC) transporter-mediated efflux, provide extrinsic protection within tumor microenvironment (TME) niches, sustain the survival and plasticity of cancer stem cells (CSCs), and maintain quiescent dormant cells that seed late recurrence. Drug repurposing, the identification of new therapeutic uses for approved or clinically characterized drugs, is attractive in this setting because many agents developed outside oncology act on the transport, metabolic, stromal, inflammatory and stress-adaptive pathways on which these programs depend. In principle, existing human pharmacokinetic, formulation and safety data may shorten development timelines and reduce early attrition relative to de novo discovery, although mechanism confirmation and prospective clinical validation remain obligatory. Metabolic inhibitors, anti-inflammatory agents, antiparasitic and antiviral drugs have all been proposed to disrupt survival mechanisms engaged under therapeutic pressure, yet candidate selection and mechanistic attribution in resistant tumors remain difficult. In this review we apply a single mechanism-first filter to representative candidates in each of the four domains above, separating preclinical signals from clinically validated outcomes and stating explicitly where a conclusion rests on one primary study. We then examine how resistance biology can be linked to biomarker-defined patient selection. Finally, we consider the trial architectures, including basket, umbrella and platform designs, through which repurposed agents can be evaluated without diluting a context-restricted mechanistic signal.

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Publication Details

Journal
Molecular Cancer
Published
2026-10-14
DOI
https://doi.org/10.1186/s12943-026-02815-2
Primary Topic
Lung Cancer Treatments and Mutations
Type
article
Field-Weighted Citation Impact
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article

Mechanism-guided drug repurposing against therapeutically actionable resistance domains in cancer: from resistance biology to clinical translation

栾思源, Jichun Shao, Xue Li, Zhu Xinyuan et al.
Molecular Cancer
Lung Cancer Treatments and Mutations
article

Mechanism-guided drug repurposing against therapeutically actionable resistance domains in cancer: from resistance biology to clinical translation

栾思源, Jichun Shao, Xue Li, Zhu Xinyuan, Jianfeng Zhou, Yong Yuan, Yixuan Tao, Xin Xiao, Haowen Zhang
article en

Abstract

Tumor resistance remains a central obstacle in cancer treatment. It blunts the activity of otherwise effective agents and narrows the therapeutic window, so that patients may accrue toxicity without commensurate benefit, and it drives progression and relapse. Resistance is rarely a single lesion; it is the emergent product of interacting programs that limit intracellular drug accumulation through ATP-binding cassette (ABC) transporter-mediated efflux, provide extrinsic protection within tumor microenvironment (TME) niches, sustain the survival and plasticity of cancer stem cells (CSCs), and maintain quiescent dormant cells that seed late recurrence. Drug repurposing, the identification of new therapeutic uses for approved or clinically characterized drugs, is attractive in this setting because many agents developed outside oncology act on the transport, metabolic, stromal, inflammatory and stress-adaptive pathways on which these programs depend. In principle, existing human pharmacokinetic, formulation and safety data may shorten development timelines and reduce early attrition relative to de novo discovery, although mechanism confirmation and prospective clinical validation remain obligatory. Metabolic inhibitors, anti-inflammatory agents, antiparasitic and antiviral drugs have all been proposed to disrupt survival mechanisms engaged under therapeutic pressure, yet candidate selection and mechanistic attribution in resistant tumors remain difficult. In this review we apply a single mechanism-first filter to representative candidates in each of the four domains above, separating preclinical signals from clinically validated outcomes and stating explicitly where a conclusion rests on one primary study. We then examine how resistance biology can be linked to biomarker-defined patient selection. Finally, we consider the trial architectures, including basket, umbrella and platform designs, through which repurposed agents can be evaluated without diluting a context-restricted mechanistic signal.

Molecular Cancer
China National Nuclear Corporation (CN), Chengdu Medical College (CN), Sichuan University (CN), West China Medical Center of Sichuan University (CN), West China Hospital of Sichuan University (CN), First Affiliated Hospital of Chengdu Medical College (CN)
Good health and well-being
Openalex Percentile: Top 13%
Lung Cancer Treatments and Mutations
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