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 medical research aimed at improved therapies and a cure. Today, we are the nation’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 country, 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 continues to lead the way in funding scleroderma research. It has maintained 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. In the fiscal year ending 2012, the SRF funded more than $1,000,000 in direct research grants.

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 SRF’s collaborative approach 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.

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.

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.

Each year, the SRF hosts a Scientific Workshop where SRF-funded researchers and other investigators 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 lead staff are:

Amy Hewitt
Executive Director

Charles Spaulding
Vice President, Communications

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Research News

Evidence-based detection of pulmonary arterial hypertension in systemic sclerosis: the DETECT study

Author: J Gerry Coghlan,Christopher P Denton,Ekkehard Grünig,et al
Date Published: May-2013
Source: Annals of the Rheumatic Diseases

Objective: Earlier detection of pulmonary arterial hypertension (PAH), a leading cause of death in systemic sclerosis (SSc), facilitates earlier treatment. The objective of this study was to develop the first evidence-based detection algorithm for PAH in SSc.

Conclusions The novel, evidence-based DETECT algorithm for PAH detection in SSc is a sensitive, non-invasive tool which minimises missed diagnoses, identifies milder disease and addresses resource usage.

Systemic sclerosis (scleroderma, SSc) is a vascular disease

Author: Marco Matucci-Cerinic, Bashar Kahaleh, Fredrick M. Wigley
Date Published: May-2013
Source: Arthritis & Rheumatism

Molecular Signatures in Skin Associated with Clinical Improvement during Mycophenolate Treatment in Systemic Sclerosis

Author: Monique Hinchcliff, Chiang-Ching Huang, Tammara A Wood, J Matthew Mahoney, et al
Date Published: May-2013
Source: Journal of Investigative Dermatology

Heterogeneity in systemic sclerosis (SSc) confounds clinical trials. We previously identified “intrinsic” gene expression subsets by analysis of SSc skin. Here we test the hypotheses that skin gene expression signatures including intrinsic subset are associated with modified Rodnan skin score (MRSS) improvement during mycophenolate mofetil (MMF) treatment. Gene expression and intrinsic subset assignment were measured in 12 SSc patients’ biopsies and 10 controls at baseline, and from serial biopsies of 1 cyclophosphamide-treated patient and 9 MMF-treated patients.

The degree of skin involvement identifies distinct lung disease outcomes and survival in systemic sclerosis

Author: Tricia R Cottrell, Robert A Wise, Fredrick M Wigley & Francesco Boin
Date Published: April-2013
Source: Annals of the Rheumatic Diseases

Abstract - Objective To determine whether the pattern of skin involvement can predict clinical features, risk of restrictive lung disease (RLD) and survival in a large scleroderma (SSc) cohort. Methods Demographic and clinical data collected over 30 years from 2205 patients with SSc were retrospectively analysed after subdividing subjects into four subtypes based on pattern of skin fibrosis: type 0 (no skin involvement), type 1 (limited to metacarpophalangeal joints), type 2 (distal to elbows/knees) and type 3 (proximal to elbows/knees). Clinical features associated with skin subsets were identified by regression analyses. Kaplan–Meier and Cox proportional hazards models were used to compare time to RLD and survival across subtypes.

Open label study of escalating doses of oral treprostinil diethanolamine in patients with systemic sclerosis and digital ischemia: pharmacokinetics and correlation with digital perfusion.

Author: Shah AA, Schiopu E, Hummers LK, Wade M, Phillips K, Anderson C, Wise R, Boin F, Seibold JR, Wigley F, Rollins KD.
Date Published: April-2013
Source: Arthritis Research & Therapy

Abstract - INTRODUCTION: Treprostinil diethanolamine is an innovative salt form of the prostacyclin analogue, treprostinil sodium, developed as an oral sustained release (SR) osmotic tablet. The availability of a formulation permitting convenient systemic delivery might have applicability to scleroderma vascular complications. We evaluated pharmacokinetics and perfusion in scleroderma patients with digital ischemia following escalating twice-daily doses of treprostinil diethanolamine SR. METHODS: In this dual-center, open-label, phase I pharmacokinetic study, scleroderma patients with digital ulcers were enrolled. Drug concentrations and perfusion, quantified by laser Doppler imaging, were measured over 12 hours at the 2mg and 4mg (or maximally tolerated) doses.

News for Patients

Evidence-based detection of pulmonary arterial hypertension in systemic sclerosis: the DETECT study

Author: J Gerry Coghlan,Christopher P Denton,Ekkehard Grünig,et al
Date Published: May-2013
Source: Annals of the Rheumatic Diseases

Objective: Earlier detection of pulmonary arterial hypertension (PAH), a leading cause of death in systemic sclerosis (SSc), facilitates earlier treatment. The objective of this study was to develop the first evidence-based detection algorithm for PAH in SSc.

Conclusions The novel, evidence-based DETECT algorithm for PAH detection in SSc is a sensitive, non-invasive tool which minimises missed diagnoses, identifies milder disease and addresses resource usage.

Pulmonary hypertension often misdiagnosed, research finds - U. of C. study stresses need to identify condition earlier

Author: Jessica Tobacman
Date Published: May-2013
Source: The Baltimore Sun

Whitney Gaspar has long known that she didn't have much endurance. When she was in secondary school, Gaspar said she ran a "slow mile" in gym class of 14 to 15 minutes and "avoided stuff (that required) a lot of endurance." Then in December 1999, she caught the flu and couldn't walk four or five steps without her lips turning blue, said Gaspar, 36, of Plainfield. Doctors misdiagnosed Gaspar as having asthma. In February 2000, she finally got the correct diagnosis:

Actelion gets go-ahead to continue new drug trial

Author: Caroline Copley
Date Published: May-2013
Source: Reuters

Actelion should continue a late-stage study into a new heart and lung drug, independent monitors have recommended, with final results now expected by mid-2014, giving the biotech firm hope it has a further product in its pipeline. Actelion said in a statement the Independent Data Monitoring Committee had told the company it had unanimously recommended the continuation of a late-stage study in selexipag with no modifications, adding final results should come next year.

The FDA Wants You!

Author: Melissa Healy
Date Published: April-2013
Source: Los Angeles Times

The Food and Drug Administration, acting on a law signed by President Obama in 2012, has launched the third phase of an initiative to increase patient participation in the regulation of drugs and medical devices. On Wednesday, the agency went live with a new website aimed at demystifying the regulatory process for consumers and patient advocates. The new FDA website, called FDA Patient Network, is designed to educate consumers on the process of getting medical devices and drugs from the idea stage to pharmacy and hospital shelves. Along with the FDA Commissioner's blog, this is the agency's bid to be more transparent.

Faster Drug Approvals a Tonic for Pharma Industry

Author: Ben Hirschler
Date Published: May-2013
Source: Reuters

A pickup in new drug approvals, the promise of faster regulatory decisions and more targeted medicines have quickened the pulse of the pharmaceutical industry as a big wave of patent expiries recedes. Manufacturers are producing more targeted medicines, designed to treat very specific groups of patients, thanks to a new understanding of the genetic basis of many diseases - most notably cancer.

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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