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.
 
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Research News

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.

Clinical Trial for MEDI-551 in Scleroderma Completed

Author: Mahoney JM, Taroni J, Martyanov V, Wood TA, Greene CS, Pioli PA, Hinchcliff ME, Whitfield ML
Date Published: January-2015
Source: PLoS One

Systemic sclerosis (SSc) is a rare systemic autoimmune disease characterized by skin and organ fibrosis. The pathogenesis of SSc and its progression are poorly understood. The SSc intrinsic gene expression subsets (inflammatory, fibroproliferative, normal-like, and limited) are observed in multiple clinical cohorts of patients with SSc. Analysis of longitudinal skin biopsies suggests that a patient's subset assignment is stable over 6-12 months. Genetically, SSc is multi-factorial with many genetic risk loci for SSc generally and for specific clinical manifestations.

Serum adhesion molecule levels as prognostic markers in patients with early systemic sclerosis: a multicentre, prospective, observational study.

Author: Hasegawa M, Asano Y, Endo H, Fujimoto M, Goto D, Ihn H
Date Published: January-2015
Source: PLoS One

To assess the utility of circulating adhesion molecule levels as a prognostic indicator of disease progression in systemic sclerosis (SSc) patients with early onset disease. METHODS: Ninety-two Japanese patients with early onset SSc presenting with diffuse skin sclerosis and/or interstitial lung disease were registered in a multicentre, observational study. Concentrations of intercellular adhesion molecule (ICAM) -1, E-selectin, L-selectin, and P-selectin in serum samples from all patients were measured by enzyme-linked immunosorbent asssay (ELISA). In 39 patients, adhesion molecule levels were measured each year for four years. The ability of baseline adhesion molecule levels to predict subsequent progression and severity in clinical and laboratory features were evaluated statistically.

News for Patients

SSc Patients May Improve with Grafts of Their Own Fat

Author: Alisa Woods
Date Published: January-2015
Source: Scleroderma News

Italian scientists may have identified a way to improve systemic sclerosis (SSc) symptoms with fat grafts. A recent study titled “Autologous Fat Grafting in the Treatment of Fibrotic Perioral Changes in Patients with Systemic Sclerosis” published in the journal Cell Transplantation showed that the fat grafts improved mouth movement, improved blood vessel regrowth, and helped to restore the skin.

DETECT, ASIG Algorithms Outperform ESC/ERS Guidelines for Pulmonary Arterial Hypertension in Systemic Sclerosis

Author: Daniela Semedo
Date Published: January-2015
Source: Scleroderma News

Systemic sclerosis (SSc) is a multisystem connective tissue disease characterized by vasculopathy and fibrosis. Pulmonary arterial hypertension (PAH) is one of the most severe organ complications and a leading cause of death in SSc. Estimations are that patients with SSc that have associated PAH have a 3-year survival of just 50%. Since early identification has been found to improve the outcome, researchers have been working on pinpointing the optimal care management for patients with SSc.

Genetic Markers Aid in Tracking CVD Risk in SSc - Two markers appear to be useful for identifying SSc patients at risk for pulmonary hypertension.

Author: Diana Swift
Date Published: January-2015
Source: MedPage Today

Patients with systemic sclerosis (SSc) who are free of traditional cardiovascular risk factors can still have increased levels of two biomarkers indicative of myocyte injury: high-sensitivity cardiac troponin (HS-cTnT) and N-terminal fragment of pro-brain natriuretic peptide (NT-proBNP). "Elevated levels of these markers may reflect subclinical myocardial damage and dysfunction caused by the disease [SSc] itself," wrote French investigators online in Arthritis Care and Research.

Is Rituximab Ready for Prime Time in SSc? Experts: new data support rituximab for reducing lung fibrosis in systemic sclerosis.

Author: Diana Swift
Date Published: January-2015
Source: MedPage Today

B-cell depletion via rituximab (Rituxan) infusion reduced the progression of skin thickening and lung fibrosis in systemic sclerosis (SSc), according to a review article, and clinicians need to consider rituximab therapy in these patients. Fiona McQueen, MD, of the University of Auckland, and Kamal Solanki, MD, of Waikato Hospital in Hamilton, both in New Zealand, looked at observational, case-control data from the European League Against Rheumatism Scleroderma Trials and Research (EUSTAR) group and summarized their findings in Rheumatology.

ASSET (Abatacept Systemic SclErosis Trial)

Author:
Date Published: January-2015
Source: University of Michigan Scleroderma Program

ASSET (Abatacept Systemic SclErosis Trial) is an international, placebo-controlled, double blind, randomized trial of early diffuse cutaneous systemic sclerosis (SSc). This trial assesses abatacept (Orencia®, a recombinant fusion protein consisting of the extracellular domain of human CTLA4 is FDA approved biologic medication for rheumatoid arthritis and juvenile arthritis) in patients with early diffuse SSc with less than or equal to 36 months.

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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