Related Experiment Video

Updated: Jul 7, 2026

Mechanistic Insight into the Development of TNBS-Mediated Intestinal Fibrosis and Evaluating the Inhibitory Effects of Rapamycin
10:21

Mechanistic Insight into the Development of TNBS-Mediated Intestinal Fibrosis and Evaluating the Inhibitory Effects of Rapamycin

Published on: September 12, 2019

Topical rapamycin inhibits tuberous sclerosis tumor growth in a nude mouse model

Aubrey Rauktys1, Nancy Lee, Laifong Lee

  • 1Translational Medicine Division, Department of Medicine, Brigham & Women's Hospital, One Blackfan Circle, Karp Research Building, Boston, MA, 02115 USA. arauktys21@gmail.com

BMC Dermatology
|January 30, 2008
PubMed
Abstract

Insights

Topical rapamycin effectively reduced tumor growth and improved survival in a mouse model for Tuberous Sclerosis Complex (TSC). This topical treatment approach shows promise for managing TSC skin lesions.

Area of Science:

  • Dermatology
  • Oncology
  • Pharmacology

Background:

  • Tuberous Sclerosis Complex (TSC) causes significant skin morbidity.
  • mTOR inhibitors, like rapamycin, are effective for TSC's kidney and brain manifestations.
  • Investigating topical rapamycin for TSC-related skin tumors.

Purpose of the Study:

  • To evaluate the efficacy of topical rapamycin in a mouse model of TSC-related tumors.
  • To assess systemic drug levels following topical rapamycin application.
  • To compare topical rapamycin with injected rapamycin and vehicle controls.

Main Methods:

  • Application of 0.4% and 0.8% rapamycin ointments to mice with TSC-related tumors.
  • Comparison of topical treatment with injected rapamycin and topical vehicle.
  • Measurement of blood and tumor rapamycin levels to determine systemic exposure.

Main Results:

  • Topical rapamycin treatment improved survival rates and reduced tumor growth.
  • Systemic rapamycin levels achieved with topical application were within the therapeutic range.
  • Injected rapamycin demonstrated greater efficacy compared to topical rapamycin.

Conclusions:

  • Topical rapamycin demonstrates inhibitory effects on TSC-related tumor growth.
  • This approach may offer a novel treatment for skin manifestations of TSC, such as facial angiofibromas.
  • Further research could establish topical rapamycin as a viable therapeutic option for cutaneous TSC lesions.

More Related Videos

Development and Application of Rapamycin-regulated Tyrosine Phosphatases06:56

Development and Application of Rapamycin-regulated Tyrosine Phosphatases

Published on: September 6, 2024

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...
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...