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 $1,000,000 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.
 
E-mail Print PDF
 

Research News

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…

Review: Cancer-Induced Autoimmunity in the Rheumatic Diseases

Author: Ami A. Shah, Livia Casciola-Rosen and Antony Rosen
Date Published: January-2015
Source: Arthritis & Rheumatology

Tantalizing connections between autoimmune rheumatic diseases and cancer have become increasingly evident over the past several decades. These connections are complex, with different relationships in frequency, timing, and types of cancers observed in different diseases or disease subgroups. Several recent advances from disparate fields have begun to illuminate the dynamic and bidirectional interactions occurring at the cancer–immune system interface which may be relevant to understanding the origins of autoimmunity ([1]). These interactions include the existence of potent anticancer immune responses that limit tumor growth, as well as multiple immune and inflammatory pathways that can contribute to tumor growth and robustness. The striking ability of immune checkpoint inhibitors to reveal powerful anticancer immune responses in patients with cancer highlights the fact that natural immune responses to cancers occur, and may regulate the emergence of cancer ([2]).

Autologous Fat Grafting in the Treatment of Fibrotic Perioral Changes in Patients With Systemic Sclerosis

Author: Del Papa, Nicoletta; Caviggioli, Fabio; Sambataro, Domenico; et al
Date Published: January-2015
Source: Cell Transplantation

Autologous fat tissue grafting (AFTG) has been successfully used in the treatment of different sclerotic conditions, including localized scleroderma. Patients with advanced systemic sclerosis (SSc)-related perioral thickening and mouth opening limitation are candidates for this therapeutic approach. AFTG of the lips was performed to improve mouth opening in patients with SSc. We enrolled in the study 20 female patients with diffuse SSc (median age 35 ± 15 years and 11 ± 10 years of disease duration). Two-milliliter fractions of autologous fat drawn from trochanteric or periumbilical areas were injected in eight different sites around the mouth.

Impaired BMPRII Signalling in a TGFβ Dependent Mouse Model of Pulmonary Hypertension and in Systemic Sclerosis.

Author: A. Gilbane, E. Derrett-Smith, S. Trinder, R. Good, A. Pearce , C. Denton , and A. Holmes
Date Published: January-2015
Source: American Journal of Respiratory and Critical Care Medicine

Rationale: Up to 10 percent of systemic sclerosis (SSc) patients develop pulmonary arterial hypertension (PAH). This risk persists throughout the disease and is time-dependent, suggesting that SSc is a susceptibility factor. Outcome for SSc-PAH is poor compared with heritable (hPAH) or idiopathic (iPAH) forms, despite clinical and pathological similarities. Whereas susceptibility in hPAH and iPAH is strongly associated with gene mutations leading to reduced expression of bone morphogenetic protein type II receptor (BMPRII), these mutations have not been observed in SSc-PAH.

News for Patients

Actemra Looks Promising in Scleroderma

Author: Nancy Walsh
Date Published: April-2015
Source: MedPage Today

Encouraging results were seen for tocilizumab (Actemra) in the treatment of diffuse systemic sclerosis (SSc) in a proof-of-concept study, an investigator reported here. At 24 weeks, a numerically favorable response was seen on modified Rodnan skin scores among patients randomized to tocilizumab compared with those receiving placebo, with changes of -3.9 units versus -1.2 units, for a difference of -2.70 (95% CI -5.85 to 0.45, P=0.09), according to Christopher P. Denton, MBBS, of University College London.

Systemic Sclerosis Patients Are More Prone to Atherosclerosis

Author: Patricia Silva
Date Published: April-2015
Source: Scleroderma News

In a new study entitled “Subclinical atherosclerosis and peripheral vascular disease in systemic sclerosis patients: Relation to potential risk factors,” authors suggest that patients with Systemic sclerosis may have increased risk of developing subclinical atherosclerotic macro vascular disease. The team highlights that further research is necessary to tackle both the cause for increased risk of atherosclerosis and the vessel damage in the SSc patient population. The study was published in the journal The Egyptian Rheumatologist.

Researchers Find New Small Molecule Capable of Triggering the Immune System

Author: Patricia Silva
Date Published: April-2015
Source: Scleroderma News

Researchers at the University of Colorado Boulder and Tsinghua University in China recently published in the journal Science Advances that a specific small molecule is capable of triggering an immune response, having potential biomedical applications for treating diseases such as Scleroderma. The study is entitled “Specific activation of the TLR1-TLR2 heterodimer by small-molecule agonists.”

7 Things You Should Know About Autoimmune Diseases

Author: Sarah Klein
Date Published: February-2015
Source: Huffington Post

As if living with chronic illness wasn't challenging enough, living with an autoimmune disease can be even more difficult. Still highly misunderstood by medical professionals and the public alike, autoimmune diseases are characterized by nebulous symptoms that can make diagnoses difficult to come by. Treatments vary, and in some cases rely entirely on behavior changes. In an effort to making living with -- or loving someone with -- an autoimmune disease just a little bit easier, here are seven important things to know about these health conditions.

Autoantibodies May Explain Link Between Cancer and Scleroderma Onset

Author: Patricia Silva
Date Published: February-2015
Source: Scleroderma News

Researchers at Johns Hopkins University School of Medicine in Baltimore investigated the association between autoantibodies, the risk of developing cancer and the possible link between cancer and scleroderma. The study is entitled “Examination of autoantibody status and clinical features that associate with cancer risk and cancer-associated scleroderma,” and is published in the journal Arthritis & Rheumatology.

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.

» Click here for details