About Us - The Scleroderma Research Foundation

Research is the key…

The Scleroderma Research Foundation (SRF) was established in 1987 by patient turned activist Sharon Monsky, when research on this potentially life threatening illness was nearly nonexistent. Since our founding, we’ve stood firm in our belief that the best way to help scleroderma patients is to fund the most promising medical research aimed at improved therapies and a cure. The SRF is America's largest nonprofit investor in scleroderma research. Patients and their loved ones find hope in the fact the SRF is dedicated exclusively to funding medical research that will help them live longer, fuller lives. Thanks in large part to the SRF and its many generous donors, research and awareness is progressing at a faster pace than ever before.

The SRF funds research investigators at some of the top universities in the United States and abroad, including Dartmouth, Harvard, Johns Hopkins, Royal Free and University College in the UK, Stanford University, Northwestern, Boston University, the University of Michigan, the University of Washington and others. Led by a Scientific Advisory Board comprised of some of the most highly-regarded scientists in the nation, the SRF's research program brings together experts from the fields of immunology and vascular biology as well as cutting-edge technology for the benefit of scleroderma patients.

The SRF is proud to maintain its position as the single largest nonprofit funding source for scleroderma research and devotes a greater percentage of its annual budget to scleroderma research, more than any other nonprofit organization.

Medical research to find better treatments for scleroderma patients is both time-consuming and expensive. Thanks entirely to thousands of supporters and generous donors, the SRF is able to expedite research progress and bring top scientists into the field of scleroderma research. The unique collaborative approach conceived by founder Sharon Monsky is enabling scientists from leading institutions across the nation—and around the world—to work together and develop an understanding of how the disease begins, how it progresses and what can be done to slow, halt or reverse the disease process.

Centers of Excellence

As another core feature of its research program, the SRF continues to provide funding to establish and support Scleroderma Centers where clinical research can be advanced. At these Centers, clinicians with large numbers of patients can collaborate with researchers and new scleroderma doctors and specialists can be trained.

Next Generation Investigators

Knowing that future discovery will come from the next generation of scientists, the SRF continues to provide grants to young investigators. Postdoctoral fellowship grants allow researchers to enter the field of scleroderma research and work alongside established investigators. As an indicator of success, several SRF-funded fellows are now dedicating their early careers to the field of scleroderma research.

Annual Scientific Workshop

Each year, the SRF hosts a Scientific Workshop where SRF-funded researchers and leaders from academia and industry engage in high level discussions about the state of scleroderma research. In addition, the SRF supports important educational initiatives such as the International Scleroderma Workshop. Collectively, these programs promote the sharing of ideas and new discoveries that further progress toward a cure.

Current IRS Form 990 and audited financial statements are available for review as Adobe PDF downloads in the Legal Notices and Privacy Information section of this website.

The continued success of the SRF research program is entirely dependent upon charitable gifts. These gifts come in many forms from generous people around the world who recognize that the SRF is dedicated to solving the mystery of scleroderma.

The SRF administrative offices are led by:

Amy Hewitt
Executive Director

Alex Gonzalez
Director of Development

Brendan Doherty
Director of Communications


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Research News

Platelets induce thymic stromal lymphopoietin production by endothelial cells: Contribution to human systemic sclerosis fibrosis

Author: Marie-Elise Truchetet MD-PhD1, Béatrice Demoures, Jorge E. Guimaraes, Anne Bertrand, Paôline Laurent1, Valérie Jolivel1, et al
Date Published: July-2016
Source: Arthritis & Rheumatology

Objective To investigate the relationship between vascular damage and fibrosis in systemic sclerosis (SSc), we tested the hypothesis that platelets contribute to skin fibrosis via the activation of dermal microvascular endothelial cells and subsequent production of pro-fibrotic mediators.

Methods A total of 203 SSc patients and 30 healthy donors (HDs) were prospectively enrolled between 2012 and 2015 at the University Hospital of Bordeaux. Immunohistochemistry and immunofluorescence analyses were performed on skin biopsies from 18 SSc patients and 5 HDs. Serum TSLP levels were measured (ELISA) in the entire cohort. Human dermal microvascular endothelial cells and fibroblasts were purified from biopsies. Extracellular matrix production by cultured fibroblasts was assessed by RT-qPCR.

Systemic sclerosis: The need for structured care

Author: Kathleen Morrisroe, Tracy Frech, Janine Schniering, Britta Maurer, Mandana Nikpour
Date Published: July-2016
Source: Clinical Rheumatology

Autoimmune connective tissue diseases (CTDs) have a propensity to affect multiple organ systems as well as physical function, quality of life, and survival. Their clinical heterogeneity, multisystem involvement, and low worldwide prevalence present challenges for researchers to establish a study design to help better understand the course and outcomes of CTDs.

Systemic sclerosis (SSc) is a notable example of a CTD, wherein longitudinal cohort studies (LCS) have enabled us to elucidate disease manifestations, disease course, and risk and prognostic factors for clinically important outcomes, by embedding research in clinical practice. Nevertheless, further efforts are needed to better understand SSc especially with regard to recognizing organ involvement early, developing new therapies, optimizing the use of existing therapies, and defining treatment targets.

Structural organization of the inactive X chromosome in the mouse

Author: Luca Giorgetti, Bryan R. Lajoie, Ava C. Carter, Mikael Attia, Ye Zhan, Jin Xu, Chong Jian Chen, Noam Kaplan, Howard Y. Chang, Edith Heard & Job Dekker
Date Published: July-2016
Source: Nature

X-chromosome inactivation (XCI) involves major reorganization of the X chromosome as it becomes silent and heterochromatic. During female mammalian development, XCI is triggered by upregulation of the non-coding Xist RNA from one of the two X chromosomes. Xist coats the chromosome in cis and induces silencing of almost all genes via its A-repeat region1, 2, although some genes (constitutive escapees) avoid silencing in most cell types, and others (facultative escapees) escape XCI only in specific contexts3. A role for Xist in organizing the inactive X (Xi) chromosome has been proposed4, 5, 6. Recent chromosome conformation capture approaches have revealed global loss of local structure on the Xi chromosome and formation of large mega-domains, separated by a region containing the DXZ4 macrosatellite.

An Open-label, Phase II Study of the Safety and Tolerability of Pirfenidone in Patients with Scleroderma-associated Interstitial Lung Disease: the LOTUSS Trial

Author: Dinesh Khanna, Carlo Albera, Aryeh Fischer, Nader Khalidi, Ganesh Raghu, Lorinda Chung, Dan Chen, Elena Schiopu, Margit Tagliaferri, James R. Seiboldand Eduard Gorina
Date Published: June-2016
Source: The Journal of Rheumatology

Systemic sclerosis-associated interstitial lung disease (SSc-ILD) shares a number of clinical features and pathogenic mechanisms with idiopathic pulmonary fibrosis (IPF). This study was designed to evaluate the tolerability of the IPF treatment pirfenidone in SSc-ILD. The known gastrointestinal, skin, and liver adverse events (AE) of pirfenidone are of importance given the involvement of these organs in SSc.

Clinical characteristics and predictors of gangrene in patients with systemic sclerosis and digital ulcers in the Digital Ulcer Outcome Registry: a prospective, observational cohort

Author: Daniel Rosenberg, Barbara Schwierin and Marco Matucci-Cerinic Yannick Allanore, Christopher P Denton, Thomas Krieg, Peter Cornelisse
Date Published: June-2016
Source: Annals of the Rheumatic Diseases

Digital vasculopathy in systemic sclerosis (SSc) consists of a spectrum of Raynaud’s phenomenon (RP), digital ulcers (DUs), critical digital ischaemia and escalation to gangrene. The complications of severe digital vasculopathy often require hospital-based management with intravenous therapies and surgery.1–3 Although gangrene is not infrequent in the clinic, data on the prevalence and implications of gangrene in patients with SSc are scarce.

News for Patients

Adempas PAH Benefits Extend to SSc

Author: Nancy Walsh
Date Published: July-2016
Source: MedPage Today

Treatment with riociguat (Adempas) increased exercise capacity among patients whose pulmonary arterial hypertension was associated with connective tissue disease (PAH-CTD) in a post-hoc analysis of a phase III clinical trial, researchers reported.

Among PAH-CTD patients randomized to receive riociguat in dosages up to 2.5 mg three times per day, the mean 6-minute walking distance increased by 18 meters at week 12 compared with a decrease of 8 m (about 26 ft.) for those given placebo, for a mean treatment difference of +28 m (95% CI -4 to 61), according to Christopher P. Denton, MD, of University College London, and colleagues.

Digital ulcers in systemic sclerosis

Author: Christopher P. Denton
Date Published: July-2016
Source: The Lancet

Systemic sclerosis (scleroderma) is one of the most recalcitrant of the immune-mediated rheumatic diseases because of its clinical impact and the high mortality associated with the internal organ complications that it causes.1 Lung fibrosis has emerged as a major cause of scleroderma-related death, and with better treatments for pulmonary arterial hypertension, the other main lethal lung manifestation of scleroderma, this looks set to continue.

Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial

Author: D.P. Tashkin, MD, M.D. Roth, MD, P.J. Clements, MD, D.E. Furst, MD, D. Khanna, MD, E.C. Kleerup, MD, J. Goldin, MD, E. Arriola, PharmD, E.R. Volkmann, MD, et al
Date Published: July-2016
Source: The Lancet

12 months of oral cyclophosphamide has been shown to alter the progression of scleroderma-related interstitial lung disease when compared with placebo. However, toxicity was a concern and without continued treatment the efficacy disappeared by 24 months. We hypothesised that a 2 year course of mycophenolate mofetil would be safer, better tolerated, and produce longer lasting improvements than cyclophosphamide.

Taking it personally, cutting-edge biotech snags $45 million

Author: Ron Leuty
Date Published: July-2016
Source: San Francisco Business Times

This year-old company is taking on fibrotic diseases — and it's a personal battle.

Advanced Echo Detection of Heart Disease in Scleroderma

Author: Johns Hopkins Rheumatology
Date Published: July-2016
Source: Johns Hopkins Medicine

A new imaging technique reveals evidence of heart dysfunction in Scleroderma patients with undiagnosed heart disease. In a team effort between the Johns Hopkins Divisions of Rheumatology, Cardiology, and Pulmonary/Critical Care Medicine, led by Monica Mukherjee, M.D. and Ami A. Shah, M.D., researchers coupled traditional echocardiography (echo) with a new technique called “speckle-tracking” to reveal the presence of right heart dysfunction that was not detected by traditional methods. This new method may identify patients with Scleroderma who are at high risk for developing disability and even death due to severe heart disease.

Ways to Give

There are many ways that you can support the work of the Scleroderma Research Foundation. We are grateful for your commitment to helping the SRF fund research that will result in improved therapies and, ultimately, a cure.

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