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Related Experiment Video

Updated: Jun 18, 2026

A Semi-Quantitative Drug Affinity Responsive Target Stability (DARTS) assay for studying Rapamycin/mTOR interaction
05:28

A Semi-Quantitative Drug Affinity Responsive Target Stability (DARTS) assay for studying Rapamycin/mTOR interaction

Published on: August 27, 2019

Rapamycin versus methotrexate in early diffuse systemic sclerosis: results from a randomized, single-blind pilot

Tien-I Karleen Su1, Dinesh Khanna, Daniel E Furst

  • 1University of Southern California, Los Angeles, CA 90095-1670, USA.

Arthritis and Rheumatism
|December 2, 2009
PubMed
Summary

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Rapamycin showed a good safety profile for systemic sclerosis (SSc) patients, with hypertriglyceridemia as a manageable side effect. Efficacy was comparable to methotrexate, but further research is needed for early diffuse SSc.

Area of Science:

  • Immunology
  • Rheumatology
  • Pharmacology

Background:

  • Systemic sclerosis (SSc) is a chronic autoimmune disease characterized by fibrosis.
  • Current treatments for diffuse SSc have limited efficacy.
  • Rapamycin, an mTOR inhibitor, has immunomodulatory and anti-fibrotic properties.

Purpose of the Study:

  • To evaluate the safety and efficacy of rapamycin compared to methotrexate (MTX) in treating diffuse SSc.
  • To assess clinical and laboratory parameters in patients with early diffuse SSc.

Main Methods:

  • A 48-week, single-blind, randomized study comparing rapamycin and MTX in 18 patients with diffuse SSc (<=5 years duration).
  • Evaluated modified Rodnan skin thickness score (MRSS) and Health Assessment Questionnaire disability index.

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Last Updated: Jun 18, 2026

A Semi-Quantitative Drug Affinity Responsive Target Stability (DARTS) assay for studying Rapamycin/mTOR interaction
05:28

A Semi-Quantitative Drug Affinity Responsive Target Stability (DARTS) assay for studying Rapamycin/mTOR interaction

Published on: August 27, 2019

  • Compared baseline and 48-week clinical and laboratory parameters.

    • Main Results:

    • Rapamycin was generally well-tolerated, with hypertriglyceridemia as the main side effect, which was treatable.
    • Both rapamycin and MTX groups showed significant improvement in MRSS from baseline.
    • No significant differences in disease activity scores or changes from baseline were observed between the two groups.

    Conclusions:

    • Rapamycin demonstrates a reasonable safety profile in select patients with scleroderma.
    • Larger clinical trials are necessary to confirm the efficacy of rapamycin for early diffuse SSc.