Efficacy of rapamycin in scleroderma: A case study

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

Research output: Contribution to journalArticle

21 Citations (Scopus)

Abstract

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
Pages (from-to)217-219
Number of pages3
JournalLymphatic Research and Biology
Volume6
Issue number3-4
DOIs
StatePublished - Dec 1 2008
Externally publishedYes

Fingerprint

Sirolimus
Collagen
Endothelial Cells
Transforming Growth Factor beta
Wound Healing
Fibrosis
Fibroblasts
Clinical Trials
Cytokines
Skin
Therapeutics

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Efficacy of rapamycin in scleroderma : A case study. / Fried, Levi; Kirsner, Robert; Bhandarkar, Sulochana; Arbiser, Jack L.

In: Lymphatic Research and Biology, Vol. 6, No. 3-4, 01.12.2008, p. 217-219.

Research output: Contribution to journalArticle

Fried, Levi ; Kirsner, Robert ; Bhandarkar, Sulochana ; Arbiser, Jack L. / Efficacy of rapamycin in scleroderma : A case study. In: Lymphatic Research and Biology. 2008 ; Vol. 6, No. 3-4. pp. 217-219.
@article{a1cae9a52a9943f293a095ae54a0e25b,
title = "Efficacy of rapamycin in scleroderma: A case study",
abstract = "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.",
author = "Levi Fried and Robert Kirsner and Sulochana Bhandarkar and Arbiser, {Jack L.}",
year = "2008",
month = "12",
day = "1",
doi = "10.1089/lrb.2008.1006",
language = "English",
volume = "6",
pages = "217--219",
journal = "Lymphatic Research and Biology",
issn = "1539-6851",
publisher = "Mary Ann Liebert Inc.",
number = "3-4",

}

TY - JOUR

T1 - Efficacy of rapamycin in scleroderma

T2 - A case study

AU - Fried, Levi

AU - Kirsner, Robert

AU - Bhandarkar, Sulochana

AU - Arbiser, Jack L.

PY - 2008/12/1

Y1 - 2008/12/1

N2 - 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.

AB - 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.

UR - http://www.scopus.com/inward/record.url?scp=58149096523&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=58149096523&partnerID=8YFLogxK

U2 - 10.1089/lrb.2008.1006

DO - 10.1089/lrb.2008.1006

M3 - Article

C2 - 18950288

AN - SCOPUS:58149096523

VL - 6

SP - 217

EP - 219

JO - Lymphatic Research and Biology

JF - Lymphatic Research and Biology

SN - 1539-6851

IS - 3-4

ER -