Related Experiment Video

Updated: May 8, 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

Tailoring mTOR-based therapy: molecular evidence and clinical challenges

Gaetano Santulli1, Hana Totary-Jain

  • 1Department of Physiology & Cellular Biophysics, The Clyde & Helen Wu Center for Molecular Cardiology, Columbia University Medical Center, New York, NY 10032, USA.

Pharmacogenomics
|September 13, 2013
PubMed

Insights

The mechanistic target of rapamycin (mTOR) pathway regulates cell growth and is implicated in diseases like cancer. This review explores mTOR pharmacogenomics and biomarkers for treatment response.

Area of Science:

  • Cellular Biology
  • Molecular Biology
  • Pharmacology

Background:

  • The mTOR signaling pathway is a central regulator of cell proliferation, growth, survival, metabolism, and autophagy.
  • Dysregulation of the mTOR pathway is linked to various human diseases, including cancer, diabetes, obesity, autoimmune disorders, neurological conditions, and aging.
  • The therapeutic potential of targeting the mTOR pathway is significant, with numerous ongoing clinical trials.

Purpose of the Study:

  • To review the evolving field of mTOR pharmacogenomics.
  • To highlight emerging diagnostic and prognostic markers, such as microRNAs (miRNAs).
  • To discuss the assessment of successful therapeutic responses to mTOR-targeted treatments.

Main Methods:

  • Literature review of mTOR signaling, pharmacogenomics, and biomarker research.
  • Analysis of current clinical applications and ongoing trials of mTOR inhibitors.
  • Discussion of diagnostic and prognostic marker identification strategies.

Main Results:

  • mTOR inhibitors are approved for organ rejection, post-angioplasty restenosis, and advanced cancers.
  • Pharmacogenomic studies are crucial for understanding individual responses to mTOR-targeted therapies.
  • MicroRNAs (miRNAs) are emerging as promising biomarkers for predicting treatment efficacy.

Conclusions:

  • The mTOR pathway is a critical therapeutic target for a range of human diseases.
  • Pharmacogenomic approaches and novel biomarkers like miRNAs are essential for optimizing mTOR-targeted treatments.
  • Further research into mTOR pharmacogenomics and biomarker development will enhance clinical outcomes.

More Related Videos

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms08:46

Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms

Published on: December 9, 2015

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids08:52

Profiling Sensitivity to Targeted Therapies in EGFR-Mutant NSCLC Patient-Derived Organoids

Published on: November 22, 2021

Related Concept Videos

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...
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...
Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...