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

Charles Spaulding
Vice President, Communications

Alex Gonzalez
Director of Development

 

 
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Research News

EULAR: Updated scleroderma guidance focuses on new treatments, approaches

Author: Sara Freeman
Date Published: June-2015
Source: PM360

ROME (FRONTLINE MEDICAL NEWS) – Updated expert recommendations from the European League Against Rheumatism for the treatment of systemic sclerosis will focus on several new treatment options, although the use of biologic agents is not included in detail because there are still too few data on these drugs to give firm guidance on their use. The EULAR Scleroderma Trials and Research group (EUSTAR) recommendations for the treatment of scleroderma were first published 6 years ago ( Ann. Rheum. Dis. 2009;68:620-8 ) and considered data through December 2006. “Since then, a number of new drugs have become available and new and important information has been published concerning treatments already known before,” said Dr. Otylia Kowal-Bielecka of the Medical University of Bialystok, Poland, who discussed some of the main highlights of the revised recommendations (Ann. Rheum. Dis. 2015;74:90-1) at the European Congress of Rheumatology.

Fresolimumab treatment decreases biomarkers and improves clinical symptoms in systemic sclerosis patients

Author: L. Rice, Cristina M. Padilla, S. McLaughlin, A. Mathes, J. Ziemek, S. Goummih, S. Nakerakanti, M. York, G. Farina, M. Whitfield, R. Spiera, R. Christmann, J. Gordon, J. Weinberg, R. Simms, and R. Lafyatis
Date Published: June-2015
Source: Journal of Clinical Investigation

BACKGROUND. TGF-β has potent profibrotic activity in vitro and has long been implicated in systemic sclerosis (SSc), as expression of TGF-β–regulated genes is increased in the skin and lungs of patients with SSc. Therefore, inhibition of TGF-β may benefit these patients. METHODS. Patients with early, diffuse cutaneous SSc were enrolled in an open-label trial of fresolimumab, a high-affinity neutralizing antibody that targets all 3 TGF-β isoforms. Seven patients received two 1 mg/kg doses of fresolimumab, and eight patients received one 5 mg/kg dose of fresolimumab. Serial mid-forearm skin biopsies, performed before and after treatment, were analyzed for expression of the TGF-β–regulated biomarker genes thrombospondin-1 (THBS1) and cartilage oligomeric protein (COMP) and stained for myofibroblasts. Clinical skin disease was assessed using the modified Rodnan skin score (MRSS).

FDA Grants Breakthrough Therapy Designation for Actemra (tocilizumab) in Systemic Sclerosis

Author: Genentech
Date Published: June-2015
Source: Genentech

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for systemic sclerosis, also known as scleroderma.1 This designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible. Genentech has also initiated a Phase III study in systemic sclerosis (NCT02453256), a disease for which there are inadequate treatment options.3,4

Cardiac troponin testing in idiopathic inflammatory myopathies and systemic sclerosis-spectrum disorders: biomarkers to distinguish between primary cardiac involvement and low-grade skeletal muscle disease activity

Author: Michael Hughes, James B Lilleker, Ariane L Herrick, Hector Chinoy
Date Published: March-2015
Source: Annals of the Rheumatic Diseases

Primary cardiac involvement, an under-recognised manifestation of the idiopathic inflammatory myopathies (IIM) and systemic sclerosis (SSc)-spectrum disorders, is associated with significant mortality. Within these two conditions, traditional skeletal muscle enzyme testing may not effectively distinguish between skeletal and cardiac muscle involvement, especially in patients with subclinical cardiac disease.

Adipocyte–myofibroblast transition: linking intradermal fat loss to skin fibrosis in SSc

Author: Sarah Onuora
Date Published: January-2015
Source: Nature Reviews Rheumatology

Myofibroblasts are considered the primary fibrogenic effector cells in systemic sclerosis (SSc), but the origin of these cells within fibrotic lesions is a matter of debate. A new study published in Arthritis & Rheumatology shows that the majority of dermal myofibroblasts in fibrotic skin arise from adiponectin-positive progenitors resident in the…

News for Patients

Unique Microbial Signature Identified in Systemic Sclerosis

Author: Pam Harrison
Date Published: June-2015
Source: Medscape

Patients with systemic sclerosis have a distinct colonic microbiome that might contribute to clinical manifestations of the disease, according to a new study. "I see scleroderma patients exclusively in my practice, and gastrointestinal symptoms are sometimes the number one complaint," said Elizabeth Volkmann, MD, from the University of California at Los Angeles. "Patients often don't even feel comfortable disclosing these symptoms because they are so personal."

Tocilizumab and Systemic Sclerosis

Author: James Radke, PhD
Date Published: June-2015
Source: Rare Disease Report

t’s a busy week for Genentech and systemic sclerosis researchers. First, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation status to tocilizumab (Actemra) for treating patients with systemic sclerosis, or scleroderma.1 The designation should expedite the development and review of tocilizumab for this rare condition. Second, data from the phase 2 study that led to the Breakthrough Designation is being presented at EULAR 2015 this week as well. The developers of tocilizumab, Genentech also announced they have begun a phase 3 study to test the safety and efficacy of tocilizumab in patients with systemic sclerosis.

EULAR: Updated scleroderma guidance focuses on new treatments, approaches

Author: Sara Freeman
Date Published: June-2015
Source: PM360

ROME (FRONTLINE MEDICAL NEWS) – Updated expert recommendations from the European League Against Rheumatism for the treatment of systemic sclerosis will focus on several new treatment options, although the use of biologic agents is not included in detail because there are still too few data on these drugs to give firm guidance on their use. The EULAR Scleroderma Trials and Research group (EUSTAR) recommendations for the treatment of scleroderma were first published 6 years ago ( Ann. Rheum. Dis. 2009;68:620-8 ) and considered data through December 2006. “Since then, a number of new drugs have become available and new and important information has been published concerning treatments already known before,” said Dr. Otylia Kowal-Bielecka of the Medical University of Bialystok, Poland, who discussed some of the main highlights of the revised recommendations (Ann. Rheum. Dis. 2015;74:90-1) at the European Congress of Rheumatology.

Fresolimumab treatment decreases biomarkers and improves clinical symptoms in systemic sclerosis patients

Author: L. Rice, Cristina M. Padilla, S. McLaughlin, A. Mathes, J. Ziemek, S. Goummih, S. Nakerakanti, M. York, G. Farina, M. Whitfield, R. Spiera, R. Christmann, J. Gordon, J. Weinberg, R. Simms, and R. Lafyatis
Date Published: June-2015
Source: Journal of Clinical Investigation

BACKGROUND. TGF-β has potent profibrotic activity in vitro and has long been implicated in systemic sclerosis (SSc), as expression of TGF-β–regulated genes is increased in the skin and lungs of patients with SSc. Therefore, inhibition of TGF-β may benefit these patients. METHODS. Patients with early, diffuse cutaneous SSc were enrolled in an open-label trial of fresolimumab, a high-affinity neutralizing antibody that targets all 3 TGF-β isoforms. Seven patients received two 1 mg/kg doses of fresolimumab, and eight patients received one 5 mg/kg dose of fresolimumab. Serial mid-forearm skin biopsies, performed before and after treatment, were analyzed for expression of the TGF-β–regulated biomarker genes thrombospondin-1 (THBS1) and cartilage oligomeric protein (COMP) and stained for myofibroblasts. Clinical skin disease was assessed using the modified Rodnan skin score (MRSS).

FDA Grants Breakthrough Therapy Designation for Actemra (tocilizumab) in Systemic Sclerosis

Author: Genentech
Date Published: June-2015
Source: Genentech

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to Actemra® (tocilizumab) for systemic sclerosis, also known as scleroderma.1 This designation is intended to expedite the development and review of medicines with early signals of potential clinical benefit in serious diseases and to help ensure patients have access to them as soon as possible. Genentech has also initiated a Phase III study in systemic sclerosis (NCT02453256), a disease for which there are inadequate treatment options.3,4

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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