Research Program

The best way to help people living with scleroderma is to fund the most promising research aimed at improved therapies and a cure.

Research is the cornerstone of the Scleroderma Research Foundation’s existence. Only from continued investment in top quality medical research will discoveries be made to help people living with scleroderma and improve their quality of life. To that end, we press forward with finding, funding and facilitating the most promising research projects at institutions around the world.

The Scleroderma Research Foundation (SRF) funds research aimed at understanding scleroderma pathogenesis (disease development), identifying markers for disease progression, developing new and more relevant animal models for scleroderma and developing new therapies. Through generous donations, the SRF awards research grants totaling more than $1,500,000 annually and is the largest nonprofit source of funds for scleroderma research.

Our core projects are aimed at understanding how the immune system and vasculature malfunction, how fibrosis begins and progresses as well as the interrelationships among these facets of the disease. Research relating to disease mechanisms provides a basis for identifying new therapeutic targets and the SRF actively promotes the exploration of new therapies.

The Foundation continues to focus significant energy on developing animal models that mimic aspects of scleroderma. These models will allow researchers to ask questions that cannot be asked in human studies and will complement experiments done with human tissue. Additionally, the SRF funds research aimed at identifying scleroderma biomarkers. Effective biomarkers could be used for early diagnosis, predicting and monitoring disease progression and assessing response to therapies.

The Scleroderma Research Foundation is dedicated to fostering the creation and continued success of Scleroderma Clinical Centers of Excellence. At these Centers, physicians representing many different specialties, such as rheumatology, pulmonology, cardiology, gastroenterology and dermatology are dedicated to clinical research and the care of scleroderma patients. Patients receive integrated care at the Centers and because all of the specialists are present and work closely together, standards of scleroderma care can be advanced. The Centers are also critical for training the next generation of scleroderma physicians and clinical investigators. Physicians and clinical investigators at the Centers play an integral role in other research projects funded by the SRF by providing vital clinical expertise.

With the expert guidance of our esteemed Scientific Advisory Board, our research projects are evaluated annually at the SRF Scientific Workshop, where intensive review and discussion of the next critical steps take place. The workshop is a forum for leading scientists from inside and outside the SRF program to provide new perspectives on the search for a cure, while promoting synergy among investigators and advancing the growing understanding of scleroderma.

Understanding of scleroderma at the cellular and molecular level is increasing thanks in part to partnerships the SRF has facilitated. Increasingly, SRF-funded scientists are exploring new opportunities that will translate laboratory advances into effective therapies to help patients live longer, fuller lives.

The Scleroderma Research Foundation is leading the scleroderma research effort by:

  • Promoting collaboration and cross-institutional cooperation among scientists in a variety of disciplines, through a strategic, integrated program.
  • Attracting promising new scientists to scleroderma research, through its Postdoctoral Fellowship Program.
  • Promoting and maintaining Scleroderma Centers of Excellence, such as the Scleroderma Center at Johns Hopkins University.
  • Bringing new experts, technology and forward thinking to the field of scleroderma research.
 
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Research News

Systematic autoantigen analysis identifies a distinct subtype of scleroderma with coincident cancer

Author: Robert Linda
Date Published: January-2017
Source: Johns Hopkins Rheumatology

A study by Livia Casciola-Rosen, Ph.D. and Aim Shah, M.D. from the Johns Hopkins Division of Rheumatology in collaboration with Steve J. Elledge, Ph.D. and collegues at MIT and Harvard, used cutting-edge technologies to identify a new subgroup of antibodies present in people without classical scleroderma-associated antibodies who develop cancer and scleroderma within a short period of time.

Immunosuppressive Therapy Helps Systemic Sclerosis Patients With Lung Disease

Author: Patricia Inacio
Date Published: January-2017
Source: Scleroderma News

Levels of the cytokine CXCl4 in the bloodstream drop sharply in response to immunosuppressive therapy, and are associated with improved lung function in systemic sclerosis (SSc) patients with interstitial lung disease (ILD), a study shows. The research, “Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease,” was published in the journal Arthritis Research & Therapy.

Gene Activity Predicts Progression of Autoimmune Disease, Researchers Find

Author: Jennie Dusheck
Date Published: December-2016
Source: Stanford Medicine

New research provides additional evidence that rituximab, a B-cell depletion therapy, improves lung fibrosis and reduces skin thickening in patients with systemic sclerosis (SSc).

Stanford researchers and their collaborators have found a way to tell whether patients with systemic sclerosis were improving during drug treatment a year before a standard clinical test could. Researchers at the Stanford University School of Medicine and six other institutions have designed a new diagnostic tool for a rare and deadly autoimmune disease that affects the skin and internal organs.

Systematic Autoantigen Analysis Identifies a Distinct Subtype of Scleroderma with Coincident Cancer

Author: George J. Xu, Ami A. Shahf, Mamie Z. Li, Qikai Xu, Antony Rosen, Livia Casciola-Rosen, and Stephen J. Elledge
Date Published: November-2016
Source: PNAS

In this study, we created a barcoded whole-genome ORF mRNA display library and combined it with phage-immunoprecipitation sequencing to look for autoantibodies in sera from patients with scleroderma who also had coincident cancer without a known autoantibody biomarker. Using these two technologies, we found that 25% of these patients had autoantibodies to RNA Binding Region Containing 3 (RNPC3) and multiple other components of the minor spliceosome. There was evidence of intra- and intermolecular epitope spreading within RNPC3 and the complex. These combined technologies are highly effective for rapidly discovering autoantibodies in patient subgroups, which will be useful tools for patient stratification and discovery of pathogenic pathways.

Targeted Therapy for Scleroderma Fibrosis

Author: Sara R. Schoenfeld, MD, & Flavia V. Castelino, MD
Date Published: October-2016
Source: The Rheumatologist

Scleroderma, or systemic sclerosis (SSc), is an autoimmune disease characterized by vasculopathy and fibrosis. Although relatively rare, with a prevalence in North America of approximately 300 per 1 million people, SSc is associated with significant morbidity and high rates of mortality.1 Patients with scleroderma have four times greater mortality than age- and sex-matched controls, with the majority of deaths related to interstitial lung disease (ILD) and pulmonary hypertension (PH).2

News for Patients

Immunosuppressive Therapy Helps Systemic Sclerosis Patients With Lung Disease

Author: Patricia Inacio
Date Published: January-2017
Source: Scleroderma News

Levels of the cytokine CXCl4 in the bloodstream drop sharply in response to immunosuppressive therapy, and are associated with improved lung function in systemic sclerosis (SSc) patients with interstitial lung disease (ILD), a study shows. The research, “Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease,” was published in the journal Arthritis Research & Therapy.

Systematic autoantigen analysis identifies a distinct subtype of scleroderma with coincident cancer

Author: Robert Linda
Date Published: January-2017
Source: Johns Hopkins Rheumatology

A study by Livia Casciola-Rosen, Ph.D. and Aim Shah, M.D. from the Johns Hopkins Division of Rheumatology in collaboration with Steve J. Elledge, Ph.D. and collegues at MIT and Harvard, used cutting-edge technologies to identify a new subgroup of antibodies present in people without classical scleroderma-associated antibodies who develop cancer and scleroderma within a short period of time.

Gene Activity Predicts Progression of Autoimmune Disease, Researchers Find

Author: Jennie Dusheck
Date Published: December-2016
Source: Stanford Medicine

New research provides additional evidence that rituximab, a B-cell depletion therapy, improves lung fibrosis and reduces skin thickening in patients with systemic sclerosis (SSc).

Stanford researchers and their collaborators have found a way to tell whether patients with systemic sclerosis were improving during drug treatment a year before a standard clinical test could. Researchers at the Stanford University School of Medicine and six other institutions have designed a new diagnostic tool for a rare and deadly autoimmune disease that affects the skin and internal organs.

Rituximab Effective Long-Term for Systemic Sclerosis

Author: Pauline Anderson
Date Published: November-2016
Source: MedPage Today

New research provides additional evidence that rituximab, a B-cell depletion therapy, improves lung fibrosis and reduces skin thickening in patients with systemic sclerosis (SSc).

Over 7 years, pulmonary function was stabilized or improved in SSc patients with interstitial lung disease receiving rituximab, and the drug also helped resolve skin thickening in these patients. As well, the study showed that cessation of rituximab therapy was associated with a decline in pulmonary function, and that the drug had an acceptable safety profile.

Parenting and Scleroderma: How Does That Work?

Author: Kim Tocker
Date Published: November-2016
Source: Scleroderma News

“If you didn’t have scleroderma then you’d be a normal Mum, aye?”

Our youngest was making a comment about how he loves going for bike rides with his Dad, and wished I could come, too. In the same conversation, he talked about missing me at the latest school sports afternoon, and described how lots of the other kids’ mums had participated in the parents’ running races.

Immediately I had visions of myself in slow motion. His mum crawling over the finish line about half an hour after the race actually finished, with a few of the parents who felt sorry for me obligingly clapping from the sidelines. I also could visualize my prednisone fat quivering in the sun as I lay there gasping for breath. No, it all would be awfully unmanageable, and also extremely traumatic, for those having to witness that sight.

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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