Related Experiment Video

Updated: May 28, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
10:27

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts

Published on: July 25, 2020

Targeted therapy in sarcomas: mammalian target of rapamycin inhibitors from bench to bedside

Bruno Vincenzi1, Andrea Napolitano, Loretta D'Onofrio

  • 1University Campus Bio-Medico, Medical Oncology, via Alvaro del Portillo, 200, Rome, Italy.

Expert Opinion on Investigational Drugs
|October 21, 2011
PubMed
Abstract

Insights

Targeting the mammalian target of rapamycin (mTOR) pathway shows promise for treating rare sarcomas. mTOR inhibitors demonstrate antitumor activity in metastatic sarcoma patients, offering hope for improved outcomes.

Area of Science:

  • Oncology
  • Molecular Biology
  • Cancer Research

Background:

  • Sarcomas are rare, heterogeneous cancers originating from mesenchymal tissues, frequently diagnosed in children.
  • The mammalian target of rapamycin (mTOR) pathway is frequently disrupted during tumorigenesis and plays a critical role in sarcoma development.
  • Targeting the mTOR pathway is a promising strategy for developing tailored sarcoma therapies.

Purpose of the Study:

  • To review the mechanisms and biological implications of the mTOR pathway.
  • To explore the role of the mTOR pathway in soft tissue sarcoma pathogenesis.
  • To summarize preclinical and clinical evidence of mTOR inhibitors in sarcoma treatment.

Main Methods:

  • Literature review of mTOR pathway mechanisms and sarcoma pathogenesis.
  • Analysis of preclinical data on mTOR inhibitors in sarcoma models.
  • Compilation of clinical trial results for mTOR inhibitors in sarcoma patients.

Main Results:

  • mTOR pathway dysregulation is implicated in sarcoma development.
  • Preclinical studies show mTOR inhibitors have potential antitumor effects.
  • Clinical investigations indicate mTOR inhibitors exhibit promising activity in metastatic sarcomas.

Conclusions:

  • mTOR inhibitors have demonstrated significant antitumor activity in patients with metastatic sarcoma who have exhausted standard treatments.
  • These inhibitors represent a promising therapeutic avenue to improve the prognosis for patients with rare sarcomas.
  • Further research and clinical trials are warranted to optimize mTOR-targeted therapies for sarcoma.

More Related Videos

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies09:29

Development and Maintenance of a Preclinical Patient Derived Tumor Xenograft Model for the Investigation of Novel Anti-Cancer Therapies

Published on: September 30, 2016

Related Concept Videos

Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
mTOR Signaling and Cancer Progression03:03

mTOR Signaling and Cancer Progression

The mammalian target of rapamycin or mTOR protein was discovered in 1994 due to its direct interaction with rapamycin. The protein gets its name from a yeast homolog called TOR. The mTOR protein complex in mammalian cells plays a major role in balancing anabolic processes such as the synthesis of proteins, lipids, and nucleotides and catabolic processes, such as autophagy in response to environmental cues, such as availability of nutrients and growth factors.
The mTOR pathway or the...
mTOR Signaling and Cancer Progression03:03

mTOR Signaling and Cancer Progression

The mammalian target of rapamycin or mTOR protein was discovered in 1994 due to its direct interaction with rapamycin. The protein gets its name from a yeast homolog called TOR. The mTOR protein complex in mammalian cells plays a major role in balancing anabolic processes such as the synthesis of proteins, lipids, and nucleotides and catabolic processes, such as autophagy in response to environmental cues, such as availability of nutrients and growth factors.
The mTOR pathway or the...
PI3K/mTOR/AKT Signaling Pathway01:22

PI3K/mTOR/AKT Signaling Pathway

The mammalian target of rapamycin  (mTOR) is a serine/threonine kinase that regulates growth, proliferation, and cell survival in response to hormones, growth factors, or nutrient availability. This kinase exists in two structurally and functionally distinct forms: mTOR complex 1  (mTORC1) and mTOR complex 2  (mTORC2). The first form (mTORC1) is composed of a rapamycin-sensitive Raptor and proline-rich Akt substrate, PRAS40. In contrast,  mTORC2 consists of a rapamycin-insensitive companion...