Efficacy of rapamycin in scleroderma: A case study

Levi Fried, Robert S. Kirsner, Sulochana Bhandarkar, Jack L. Arbiser

Research output: Contribution to journalArticlepeer-review

23 Scopus citations


Scleroderma is a common autoimmune disorder with no effective therapy. Current concepts of scleroderma include the hypothesis that scleroderma results from excess conversion of endothelial cells to fibroblast like cells, called endothelial mesenchymal transformation. This process is thought to be mediated by cytokines including transforming growth factor beta (TGFb), which causes increased collagen synthesis, resulting in fibrosis, the hallmark of the disease. In vitro studies have hypothesized that rapamycin may be of benefit in scleroderma due to antagonism of collagen synthesis. Given that rapamycin has antiangiogenic activities, inhibits wound healing, and prevents the synthesis of collagen in vivo, we tried rapamycin in a patient with scleroderma. We observed rapid improvement in skin stiffness and mobility. Our results provide the rationale for larger clinical trials of rapamycin in scleroderma and other fibrotic disorders.

Original languageEnglish (US)
Pages (from-to)217-219
Number of pages3
JournalLymphatic Research and Biology
Issue number3-4
StatePublished - Dec 1 2008
Externally publishedYes

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine


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