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Updated: Jul 19, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
mTOR and cancer therapy
1Department of Molecular Pharmacology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
Abstract:
Proteins regulating the mammalian target of rapamycin (mTOR), as well as some of the targets of the mTOR kinase, are overexpressed or mutated in cancer. Rapamycin, the naturally occurring inhibitor of mTOR, along with a number of recently developed rapamycin analogs (rapalogs) consisting of synthetically derived compounds containing minor chemical modifications to the parent structure, inhibit the growth of cell lines derived from multiple tumor types in vitro, and tumor models in vivo. Results from clinical trials indicate that the rapalogs may be useful for the treatment of subsets of certain types of cancer. The sporadic responses from the initial clinical trials, based on the hypothesis of general translation inhibition of cancer cells are now beginning to be understood owing to a more complete understanding of the dynamics of mTOR regulation and the function of mTOR in the tumor microenvironment. This review will summarize the preclinical and clinical data and recent discoveries of the function of mTOR in cancer and growth regulation.
Insights
The mammalian target of rapamycin (mTOR) pathway is implicated in cancer. Inhibitors like rapamycin and rapalogs show promise in treating certain cancers by affecting tumor growth.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- The mammalian target of rapamycin (mTOR) pathway plays a crucial role in cell growth and proliferation.
- Aberrant mTOR signaling, through overexpression or mutation of key proteins, is frequently observed in various cancers.
Purpose of the Study:
- To review preclinical and clinical data on the function of mTOR in cancer.
- To summarize recent discoveries regarding mTOR's role in tumor growth regulation.
Main Methods:
- Review of preclinical studies investigating mTOR inhibitors in cancer cell lines and tumor models.
- Analysis of clinical trial data evaluating the efficacy of rapamycin analogs (rapalogs) in cancer patients.
Main Results:
- Rapamycin and rapalogs demonstrate in vitro and in vivo inhibition of cancer cell growth.
- Clinical trials suggest potential therapeutic benefits of rapalogs for specific cancer subsets.
Conclusions:
- Understanding mTOR regulation dynamics and its role in the tumor microenvironment is key to interpreting clinical responses.
- mTOR inhibitors represent a promising therapeutic strategy for subsets of cancers, warranting further investigation.
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