Related Experiment Video
Updated: Aug 8, 2026

Development and Application of Rapamycin-regulated Tyrosine Phosphatases
Published on: September 6, 2024
Mammalian target of rapamycin inhibitors in sarcomas
1Medical Oncology, Mayo Clinic College of Medicine, Rochester, Minnesota 55905, USA. okuno.scott@mayo.edu
Purpose Of Review:
Sarcomas are a rare malignancy accounting for less than 1% of all cancers diagnosed annually. Standard chemotherapy has a response rate of around 25% and newer agents are needed to improve the outcome in patients with advanced sarcomas. The mammalian target of rapamycin plays a central role in cell growth, proliferation, and apoptosis and its inhibition has demonstrated antitumor activity in many tumors and shows promise against sarcomas.
Recent Findings:
Recent studies of mammalian target of rapamycin inhibitors in sarcomas have demonstrated clinical benefit response in sarcomas.
Summary:
Clinical benefit response uses standard Response Evaluation Criteria in Solid Tumors of complete response and partial response as well as stable disease lasting at least 4 months as an endpoint. This endpoint has been shown to select promising new agents against sarcomas. Using this endpoint, the use of the mammalian target of rapamycin inhibitor AP23573 has demonstrated activity against sarcomas. The use of the inhibitor RAD001 (everolimus) along with imatinib in patients with imatinib resistant gastrointestinal stromal tumor has shown promise. Future studies will need to be performed to determine the clinical differences among the mammalian target of rapamycin inhibitors in different subsets of sarcomas.
Insights
Mammalian target of rapamycin (mTOR) inhibitors show promise for treating advanced sarcomas, offering improved outcomes beyond standard chemotherapy. Recent studies demonstrate clinical benefit response in sarcoma patients treated with these novel agents.
Area of Science:
- Oncology
- Pharmacology
- Molecular Biology
Background:
- Sarcomas represent a rare but aggressive group of cancers with limited treatment options.
- Standard chemotherapy offers a low response rate for advanced sarcomas, necessitating novel therapeutic strategies.
- The mammalian target of rapamycin (mTOR) pathway is crucial for cell growth and survival, making it a potential therapeutic target.
Purpose of the Study:
- To review the emerging role of mammalian target of rapamycin (mTOR) inhibitors in sarcoma treatment.
- To evaluate the efficacy and potential of mTOR inhibitors as novel agents against advanced sarcomas.
- To discuss the clinical benefit response endpoint in assessing new sarcoma therapies.
Main Methods:
- Review of recent clinical studies and research on mTOR inhibitors in sarcoma.
- Analysis of data evaluating clinical benefit response, including complete response, partial response, and stable disease.
- Examination of specific mTOR inhibitors like AP23573 and RAD001 (everolimus) in sarcoma treatment.
Main Results:
- Recent studies indicate that mammalian target of rapamycin (mTOR) inhibitors demonstrate clinical benefit response in sarcomas.
- The mTOR inhibitor AP23573 has shown activity against sarcomas.
- RAD001 (everolimus) combined with imatinib shows promise in imatinib-resistant gastrointestinal stromal tumors.
Conclusions:
- The clinical benefit response endpoint effectively identifies promising new agents for sarcoma treatment.
- mTOR inhibitors represent a promising class of drugs for advanced sarcomas.
- Further research is needed to differentiate the clinical efficacy of various mTOR inhibitors across sarcoma subtypes.
Related Concept Videos
mTOR Signaling and Cancer Progression
The mTOR pathway or the...
mTOR Signaling and Cancer Progression
The mTOR pathway or the...
PI3K/mTOR/AKT Signaling Pathway
Targeted Cancer Therapies
There are several types of targeted therapies against specific...
Abnormal Proliferation
Inhibition of Cdk Activity
