Related Experiment Video

Updated: Aug 15, 2026

Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants
07:38

Intracellular Phosphoflow Cytometry of Acute Myeloid Leukemia Patient-Derived Xenotransplants

Published on: June 6, 2025

mTOR, a new therapeutic target in acute myeloid leukemia

Christian Récher1, Cédric Dos Santos, Cécile Demur

  • 1Département d'Oncogenèse et Signalisation dans les Cellules Hématopoïétiques, Institut National de la Santé et de la Recherche Médicale (Inserm) U563, CPTP, Toulouse, France. recher.c@chu-toulouse.fr

Cell Cycle (Georgetown, Tex.)
|October 6, 2005
PubMed

Insights

Mammalian target of rapamycin (mTOR) inhibitors show promise for treating acute myeloid leukemia (AML). Low-concentration rapamycin effectively inhibits AML cell growth while sparing normal cells, offering new therapeutic strategies for this challenging disease.

Area of Science:

  • Oncology
  • Molecular Biology
  • Hematology

Background:

  • The mammalian target of rapamycin (mTOR) pathway regulates critical cellular processes like cell cycle progression, apoptosis, and angiogenesis.
  • Aberrant mTOR pathway activation is implicated in various cancers, including hematological malignancies such as acute myeloid leukemia (AML).
  • Current therapeutic strategies for AML have limitations, necessitating the exploration of novel treatment approaches.

Purpose of the Study:

  • To review the role of the mTOR pathway in AML pathogenesis.
  • To discuss the anti-leukemic effects of mTOR inhibitors, particularly rapamycin.
  • To explore potential mechanisms of resistance and strategies to enhance rapamycin efficacy in AML.

Main Methods:

  • Review of existing literature on mTOR signaling in cancer and AML.
  • Analysis of preclinical data on the effects of rapamycin and its analogues on AML cells.
  • Examination of clinical trial data involving mTOR inhibitors in AML patients.

Main Results:

  • Rapamycin demonstrates potent anti-neoplastic effects in solid tumor models and is under clinical investigation.
  • Low-concentration rapamycin significantly inhibits AML cell clonogenic properties in a majority of cases, while sparing normal hematopoietic cells.
  • Clinical responses have been observed in patients with poor-risk AML treated with rapamycin.

Conclusions:

  • The mTOR pathway is a potential therapeutic target in AML.
  • Rapamycin exhibits significant anti-leukemic activity and warrants further investigation for AML treatment.
  • Understanding resistance mechanisms and optimizing rapamycin delivery are crucial for improving clinical outcomes in AML.

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...
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...
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...
Treatment Resistent Cancers02:56

Treatment Resistent Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...