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

Clinical Trial Design Issues in Systemic Sclerosis: an Update

Author: Jessica K. Gordon, Robyn T. Domsic
Date Published: May-2016
Source: Springer Link

Systemic sclerosis (scleroderma, SSc) is a multisystem disease characterized by vasculopathy, autoimmunity, and fibrosis. SSc has the highest disease-related mortality rate among the rheumatologic illnesses. In the USA, there remains no FDA-approved therapy. As our understanding of SSc pathogenesis improves, targeted therapies interrupting key pathways and mediators will be studied in clinical trials. However, clinical trials in SSc are fraught with challenges. Validated clinical outcome measures do not exist for all disease manifestations. It can be difficult to discern disease activity from damage. SSc is highly heterogeneous, with multiple different phenotypes, and predicting who will have progressive disease is not currently well understood. Biomarkers are in early stages of development and do not represent surrogate outcomes at this time.

O27 Subcutaneous Tocilizumab in Adults with Systemic Sclerosis: 24 and 48 Week Safety and Efficacy Data from the Fasscinate Trial

Author: C. Denton, D. Khanna, A. Jahreis, J. van Laar, et al
Date Published: May-2016
Source: Oxford Journals Rheumatology

Background: Interleukin-6 (IL-6) appears to play a key role in the pathogenesis of SSc, a debilitating disease with limited treatment options. Methods: A double-blind, placebo-controlled, phase 2, proof-of-concept study of the efficacy and safety of the IL-6 receptor inhibitor tocilizumab (TCZ) (weekly s.c. injection; TCZ 162 mg vs placebo for 48 weeks) in patients ≥18 years of age with active SSc. The primary end point was a mean change in the modified Rodnan skin score (mRSS) from baseline at week 24. Change in the mRSS at week 48, patient-reported outcomes (PROs) and pulmonary function (week 48) were exploratory measures.

Frequency of circulating topoisomerase-I-specific CD4 T cells predicts presence and progression of interstitial lung disease in scleroderma

Author: A. Fava, R. Cimbro, F. Wigley, Q. Liu, A. Rosen and F. Boin
Date Published: May-2016
Source: BioMed Central

Scleroderma is an antigen-driven T cell-mediated autoimmune disease. Presence of anti-topoisomerase-I antibodies is associated with pulmonary fibrosis and predicts increased mortality. Characterization of autoreactive T lymphocytes may shed light on disease pathogenesis and serve as a biomarker for disease activity. Here, we aimed to quantify and functionally characterize circulating topoisomerase I (topo-I)-specific CD4+ T cells and to define their association with presence and progression of interstitial lung disease (ILD) in patients with scleroderma.

M10, a caspase cleavage product of the hepatocyte growth factor receptor, interacts with Smad2 and demonstrates antifibrotic properties in vitro and in vivo.

Author: Atanelishvili I, Shirai Y, Akter T, Buckner T, Noguchi A, Silver RM, Bogatkevich G
Date Published: April-2016
Source: Translational Research

Hepatocyte growth factor receptor, also known as cellular mesenchymal-epithelial transition factor (c-MET, MET), is an important antifibrotic molecule that protects various tissues, including lung, from injury and fibrosis. The intracellular cytoplasmic tail of MET contains a caspase-3 recognition motif "DEVD-T" that on cleavage by caspase-3 generates a 10-amino acid peptide, TRPASFWETS, designated as "M10". M10 contains at its N-terminus the uncharged amino acid proline (P) directly after a cationic amino acid arginine (R) which favors the transport of the peptide through membranes. M10, when added to cell culture medium, remains in the cytoplasm and nuclei of cells for up to 24 hours.

Esophageal dilatation and interstitial lung disease in systemic sclerosis: a cross-sectional study

Author: C. Richardson, R. Agrawal, J. Lee, O. Almagor, R. Nelson, J. Varga, M. Cuttica, J. D′Amico Dematte, R. Chang, M. Hinchcliff,
Date Published: February-2016
Source: Science Direct

A patulous esophagus on high-resolution computed tomography (HRCT) of the thorax is frequently observed in patients with systemic sclerosis (SSc). Microaspiration has been purported to play a role in the development and progression of SSc interstitial lung disease (ILD), but studies examining the role of microaspiration in SSc-ILD have yielded conflicting results. This study was conducted to determine the association between esophageal diameter and SSc-ILD.

News for Patients

Being Disfigured by Scleroderma Gave Me the Chance to Love Myself

Author: Lisa Goodman-Helfand
Date Published: June-2016
Source: Dr. Oz, The Good Life

Nobody escapes adolescence unscarred by the agony of bullies, peer pressure, acne, and hormones. Along with every survivor of that awkward era, I, too, faced these obstacles. But I had the added bonus of navigating a newly diagnosed autoimmune disease: scleroderma.

I was 10 years old when doctors introduced me to the unknown world of scleroderma. It was 1985, long before Google could tell you every horrifying detail about your illness. I had no idea scleroderma could affect a patient externally, causing the skin to tighten and result in disfigurement and other painful physical abnormalities. Nor did I know that in its worst form, scleroderma attacks one's internal organs and can be fatal. In an effort to shield me from the complexities of scleroderma, my mom told me that my skin was tighter than normal, but that I was perfectly fine otherwise.

Scleroderma Patients and Lung Transplantation

Author: John Hansen-Flaschen, MD
Date Published: June-2016
Source: ATS News

Two studies published in the June issue of Annals of the American Thoracic Society look at scleroderma and lung transplantation. Patients with advanced lung disease due to systemic sclerosis (SSC) have long been considered poor candidates for lung transplantation. However, in a retrospective study of lung transplant outcomes over an eight-year period at the University of Pittsburgh, Maria M. Crespo. MD, and colleagues found that one- and five-year survival in patients with scleroderma are similar to pulmonary fibrosis. The results, they wrote, “indicate that lung transplant is a reasonable treatment option in selected patients with scleroderma.”

Phase 3 Trial to Begin for Terguride in Diffuse Cutaneous Scleroderma

Author: Magdalena Kegel
Date Published: June-2016
Source: Scleroderma News

The German pharmaceutical company Medac, in August, will begin Phase 3 of a clinical trial to access the therapeutic potential of terguride, a disease-modifying drug for the treatment of diffuse cutaneous systemic sclerosis.

Terguride is a serotonin receptor blocker, acting at two particular serotonin receptors called 5-HT2A and 5-HT2B. Medac is convinced that stopping the nerve signaling in serotonin neurons will block the progression of fibrotic tissue development and blood vessel remodeling in organs affected by diffuse cutaneous systemic sclerosis.

Pfizer, Novartis, BMS join $45M round for early fibrotic disease startup

Author: Stacy Lawrence
Date Published: June-2016
Source: Fierce Biotech

A trio of pharmas have bought into the early vision of startup Blade Therapeutics. Pfizer Venture Investments, Novartis Institutes for Biomedical Research and Bristol-Myers Squibb ($BMY) all came in to the $45 million Series B round for the startup, which aims to address novel targets in fibrotic disease. It expects to get into the clinic with this financing.

Founded in the fall of 2015, Blade is based upon technology in-licensed from Johns Hopkins University, in particular, on work from the lab of Dr. Harry Dietz--a professor of genetics and medicine at the university and the company’s founder. His work focuses on the cause of a pair of fibrotic diseases: Marfan Syndrome and Stiff Skin Syndrome, which forms the basis of Blade’s investigation into new biological pathways involved in tissue fibrosis and dysfunction.

Progression of Lung Fibrosis in Scleroderma May Be Predicted by Antibody-Specific T-cells

Author: Magdalena Kegel
Date Published: May-2016
Source: Scleroderma News

Immune T-cells induced by the presence of autoantibodies against topoisomerase-I were linked to lung fibrosis and found to predict disease progression in scleroderma patients. The finding may open new avenues of research into treatments that selectively target the various tissue-specific disease manifestations of scleroderma — a substantial improvement to the nonselective immunosuppression used today.

In scleroderma, autoantibodies targeting specific structures are linked to various clinical representations of the disease. One such antibody against the enzyme topoisomerase-I, crucial for the winding and unwinding of DNA molecules during gene expression and cell divisions, is present in 20 percent to 45 percent of scleroderma patients and linked to diffuse skin disease and lung fibrosis. Patients with anti-topoisomerase-I antibodies also have more severe 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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