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Updated: Aug 7, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Targeting mTOR for cancer treatment
Belen Rubio-Viqueira1, Manuel Hidalgo
1The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, The Baunting and Blaustein Cancer Research Building, 1650 Orleans Street, Baltimore, MD 21231, USA.
Mammalian target of rapamycin (mTOR) inhibitors show promise as anticancer agents, demonstrating antitumor activity in preclinical models and early clinical trials. Further research is needed to identify predictive markers for patient selection and optimize their clinical use.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- The mammalian target of rapamycin (mTOR) pathway regulates cellular growth and proliferation.
- Aberrant mTOR signaling is implicated in human cancers, making it a key therapeutic target.
- Rapamycin and its analogs exhibit significant antitumor effects in preclinical cancer models.
Purpose of the Study:
- To review the mTOR pathway's role in cancer and its potential as a drug target.
- To discuss current mTOR inhibitors in clinical development.
- To highlight challenges in developing mTOR inhibitors for cancer therapy.
Main Methods:
- Review of existing literature on mTOR pathway, inhibitors, and clinical trials.
- Analysis of preclinical and clinical data regarding mTOR inhibitor efficacy and safety.
- Discussion of pharmacodynamic assessments and predictive marker development.
Main Results:
- mTOR inhibitors have shown antitumor activity in various refractory cancers.
- These inhibitors are generally well-tolerated, complicating dose selection for clinical trials.
- Pharmacodynamic assessments in surrogate tissues are used, but interpatient variability is high.
Conclusions:
- mTOR inhibitors represent a promising class of anticancer drugs.
- Improved patient selection through predictive markers is crucial for clinical success.
- Further clinical studies are necessary to fully realize the therapeutic potential of mTOR inhibitors.
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