Work

Efficacy of Rapamycin in Scleroderma: A Case Study

Year: 2008

Type: article

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.

Source: Lymphatic Research and Biology

DOI: 10.1089/lrb.2008.1006

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

Institutions: University of Miami, Emory University

Language: English

Topic: Systemic Sclerosis and Related Diseases

Subfield: Pathology and Forensic Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: green

Funder: National Institute of Arthritis and Musculoskeletal and Skin Diseases