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 2013, 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:

Alex Gonzalez
Director of Development

Amy Hewitt
Executive Director

Charles Spaulding
Vice President, Communications

 
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Research News

Borderline pulmonary arterial pressure in systemic sclerosis patients: a post-hoc analysis of the DETECT study

Author: S. Visovatti, O. Distler, J. Coghlan, C. Denton, et al
Date Published: December-2014
Source: Arthritis Research & Therapy

Patients with mean pulmonary artery pressures (mPAP) of 21 to 24mm Hg have a so-called borderline elevation of mPAP (BoPAP)?a condition thought to represent early-stage pulmonary arterial vasculopathy. Based on the DETECT study, this post-hoc analysis examined patient characteristics of systemic sclerosis (SSc) patients with normal mPAP, BoPAP and elevated mPAP, fulfilling pulmonary arterial hypertension (PAH) criteria.

Advances in the Evaluation and Management of Esophageal Disease of Systemic Sclerosis

Author: D. Carlson, M. Hinchcliff, J. Pandolfino
Date Published: December-2014
Source: Current Rheumatology Reports

Symptoms of heartburn and dysphagia as well as objective findings of abnormal esophageal acid exposure and esophageal dysmotility are common in patients with systemic sclerosis (SSc). Treatments for SSc esophageal disease are generally limited to gastroesophageal reflux disease (GERD) treatment with proton pump inhibitors. Progresses made in esophageal diagnostic testing offer the potential for improved clinical characterization of esophageal disease in SSc that may help direct management decisions.

Autotaxin Is Highly Expressed in Systemic Sclerosis (SSc) Skin, Mediates Dermal Fibrosis Via IL-6, and Is a Target for SSc Therapy

Author: F. Castelino, L. George, G. Bain, L. Goulet, R. Lafyatis and A. Tager
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin (ATX) is an enzyme present in biological fluids that is responsible for the production of the lipid mediator, lysophosphatidic acid (LPA). We previously implicated LPA and its receptor, LPA1 in SSc pathogenesis.1 Here we studied the role of ATX in SSc dermal fibrosis using the bleomycin (BLM) mouse model and skin biopsy samples from SSc patients and healthy controls. We evaluated the role of IL-6, a cytokine implicated in SSc, in mediating ATX-induced fibrosis. Additionally, we investigated the therapeutic potential of targeting ATX, by using a novel ATX inhibitor, PAT-048 in this model.

SAR100842, an Antagonist of Lysophaphatidic Acid Receptor 1, As a Potential Treatment for Patients with Systemic Sclerosis: Results from a Phase 2a Study

Author: C. Denton, A. Jagerschmidt, M. Jasson, O. Distler and Y. Allanore
Date Published: December-2014
Source: American College of Rheumatology

Preclinical genetic and pharmacological studies suggest a role for Lysophosphatidic acid (LPA) involvement in three key processes of systemic sclerosis: fibrosis, microangiopathy and immunoinflammation. We have assessed SAR100842, a potent selective orally available antagonist of LPA1 receptor and explored safety, skin biomarkers of disease activity and clinical efficacy in patients with diffuse cutaneous SSc (dcSSc) in a phase 2a clinical trial.

MY ACR2014 Favorites: Systemic Sclerosis Advances and a Challenge

Author: Kristine Lohr, MD
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin is responsible for production of lysophosphatidic acid (LPA), which is implicated in scleroderma pathogenesis. In the first report above, using a murine model of dermal fibrosis, an inhibitor of autotaxin attenuated dermal fibrosis and IL-6 expression. Autotaxin expression was increased in skin from systemic sclerosis patients, compared to healthy controls. The authors concluded that targeting autotaxin may be a therapeutic strategy for scleroderma fibrosis. Combining this information with the second report above, describing efficacy of an oral antagonist of the LPA receptor in a Phase 2a study of systemic sclerosis patients, I offered substituting tocilizumab for adalimumab to my patient with an overlap syndrome [RF/CCP+ RA, primary Sjogren syndrome (+ANA/SSA/SSB) and recent progression of morphea].

News for Patients

MY ACR2014 Favorites: Systemic Sclerosis Advances and a Challenge

Author: Kristine Lohr, MD
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin is responsible for production of lysophosphatidic acid (LPA), which is implicated in scleroderma pathogenesis. In the first report above, using a murine model of dermal fibrosis, an inhibitor of autotaxin attenuated dermal fibrosis and IL-6 expression. Autotaxin expression was increased in skin from systemic sclerosis patients, compared to healthy controls. The authors concluded that targeting autotaxin may be a therapeutic strategy for scleroderma fibrosis. Combining this information with the second report above, describing efficacy of an oral antagonist of the LPA receptor in a Phase 2a study of systemic sclerosis patients, I offered substituting tocilizumab for adalimumab to my patient with an overlap syndrome [RF/CCP+ RA, primary Sjogren syndrome (+ANA/SSA/SSB) and recent progression of morphea].

Autotaxin Protein Can Be Targeted in SSc Therapy

Author: Maureen Newman
Date Published: December-2014
Source: Scleroderma News

A group from Massachusetts General Hospital is placing possible blame for systemic sclerosis (SSc) pathogenesis on autotaxin (ATX), an enzyme involved in the production of the lipid signaling molecule lysophophatidic acid (LPA). Dr. Falvia V. Castelino, a researcher in the Rheumatology Department, presented the group’s work, “Autotaxin is Highly Expressed in SSc Skin, Mediates Dermal Fibrosis Via IL-6, and Is a Target for SSc Therapy,” at the 2014 American College of Rheumatology Meeting in November.

Metabolism Influences Fibrosis in SSc

Author: Maureen Newman
Date Published: December-2014
Source: Scleroderma News

Fibrosis and metabolism: the two were recently linked in a study presented at the 2014 American College of Rheumatology Meeting in November. “Adiponectin Is an Endogenous Anti-Fibrotic and Target in Systemic Sclerosis: Novel Link Between Fibrosis and Metabolism,” presented by Dr. Feng Fang from Northwestern University and Feinberg School of Medicine, described a possible approach to a new pharmacological treatment of systemic sclerosis (SSc) based on these new findings.

Cureveda Receives NIH Grants for Systemic Sclerosis and COPD Drug

Author: Anna Tan, RN
Date Published: December-2014
Source: Lung Disease News

Biotechnology company Cureveda LLC just announced that it has received two National Institutes of Health (NIH) Phase I Small Business Technology Transfer (STTR) grants worth $450,000 in total. Cureveda specializes in treatments for inflammatory and fibrotic diseases, and the new funding will be used for VEDA-1209 preclinical testing as a breakthrough therapeutic for systemic sclerosis and chronic obstructive pulmonary disease (COPD).

FDA Grants Cytori Conditional Approval for a U.S. Pivotal Clinical Trial in Scleroderma

Author: Cytori
Date Published: December-2014
Source: BusinessWire

Cytori Therapeutics, Inc. (NASDAQ:CYTX) today announced that the U.S. Food and Drug Administration (FDA) has granted conditional approval for an Investigational Device Exemption (IDE) for a pivotal clinical trial, named the ‘STAR’ trial, to evaluate Cytori Cell TherapyTM as a potential treatment for impaired hand function in scleroderma, a rare autoimmune disease affecting approximately 50,000 patients in the United States.

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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