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

Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)

Author: A. Herrick, X. Pan, S. Peytrignet, M. Lunt, R. Hesselstrand, L. Mouthon, A. Silman, E. Brown, L. Czirják, J. Distler, O. Distler, et al
Date Published: February-2017
Source: Annals of the Rheumatic Diseases

Objectives The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches.

Methods This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or ‘no immunosuppressant’. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival.

Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease

Author: E. Volkmann, D. Tashkin, M. Roth, P. Clements, D. Khanna, D. Furst, M. Mayes, J. Charles, C. Tseng, R. Elashoff and S. Assassi
Date Published: January-2017
Source: Arthritis Research & Therapy

Increased circulatory levels of the chemokine CXCL4 have been associated with the presence of interstitial lung disease (ILD) in an observational study of patients with systemic sclerosis (SSc). The purpose of the present study was to evaluate the relationship between baseline CXCL4 level and extent of ILD in the context of a randomized controlled trial and to determine whether changes in CXCL4 levels in response to immunosuppression are associated with future progression of SSc-ILD.

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.

News for Patients

Combo Tx Aids in Scleroderma PAH

Author: Nancy Walsh
Date Published: January-2017
Source: MedPage Today

Initial combination therapy with ambrisentan (Letairis) and tadalafil (Cialis) was more effective than monotherapy with either agent alone among patients with pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH), a post-hoc subgroup analysis of the AMBITION trial found. Among patients with CTD-PAH randomized to receive both agents, the risk of having a first clinical event such as hospitalization for worsening PAH was 57% lower than in the two pooled monotherapy groups (HR 0.43, 95% CI 0.24-0.77), according to John Gerry Coghlan, MD, of the Royal Free Hospital in London, and colleagues.

Changes in plasma CXCL4 levels are associated with improvements in lung function in patients receiving immunosuppressive therapy for systemic sclerosis-related interstitial lung disease

Author: E. Volkmann, D. Tashkin, M. Roth, P. Clements, D. Khanna, D. Furst, M. Mayes, J. Charles, C. Tseng, R. Elashoff and S. Assassi
Date Published: January-2017
Source: Arthritis Research & Therapy

Increased circulatory levels of the chemokine CXCL4 have been associated with the presence of interstitial lung disease (ILD) in an observational study of patients with systemic sclerosis (SSc). The purpose of the present study was to evaluate the relationship between baseline CXCL4 level and extent of ILD in the context of a randomized controlled trial and to determine whether changes in CXCL4 levels in response to immunosuppression are associated with future progression of SSc-ILD.

Noninvasive Tests Can Help to Identify Those Most Likely, or Unlikely, to Have PH

Author: Magdalena Kegel
Date Published: January-2017
Source: Raynauds News

Noninvasive methods can help to determine which patients have a high probability of developing pulmonary hypertension (PH) and which are most unlikely to do so, researchers reported — offering clinicians further tools when examining people with suspected lung hypertension. The study, “Use of ECG and Other Simple Non-Invasive Tools to Assess Pulmonary Hypertension,” was published in the journal PLOS ONE.

Immunosuppressants Only of ‘Weak’ Benefit to Diffuse Scleroderma Patients, Study Reports

Author: Magdalena Kegel
Date Published: February-2017
Source: Scleroderma News

A large study following patients in the early stages of diffuse cutaneous scleroderma showed that immunosuppressants offered only a negligible benefit for those who took them, compared to those who didn’t. Since immunosuppressants produced numerically larger reductions in skin symptoms, the findings may support patients using such drugs from the onset of their disease. But since differences between treated and untreated patients were not statistically significant, the study may also point to the need for better treatments.

Estrogen Likely Contributes to Cold-induced Raynaud’s in Women

Author: Magdalena Kegel
Date Published: February-2017
Source: Raynauds

Estrogen may contribute to the development of Raynaud’s phenomenon in women, according to a study examining the relationship between palm blood flow and the hormone in mice. The findings might explain why Raynaud’s is more common in women of childbearing age. The study noted that reduced blood flow was mediated by estrogen’s effects on cell surface receptors. The study, “G-protein coupled estrogen receptor-mediated non-genomic facilitatory effect of estrogen on cooling-induced reduction of skin blood flow in mice,” was published in the European Journal of Pharmacology.

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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