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

Experimental Compound Shows Promise in Reversing Skin Disease Associated with Systemic Sclerosis

Author: Collee Labbe
Date Published: January-2016
Source: NIAMS Spotlight on Research

A new drug appears to alter the expression of certain genes associated with systemic sclerosis by blocking a key protein, and also leads to clinical improvements in the skin, according to a study funded in part by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The study was published in the Journal of Clinical Investigation. Systemic sclerosis, a severe form of scleroderma, is a chronic connective tissue disease that affects multiple organs and tissues in the body. It can lead to fibrosis, or hardening, of the skin and other organs such as the lungs, kidneys and heart. Currently, no effective treatments exist. Because the disease affects everyone differently, finding therapies has been challenging.

SteadyMed’s Treyvent Awarded Orphan Drug Designation from FDA

Author: Dainela Semedo
Date Published: January-2016
Source: Pulmonary Hypertension News

SteadyMed, Ltd., a company that develops products to treat orphan and high-value conditions with unmet parenteral delivery needs, recently announced that its lead product candidate, Trevyent® has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH).

Idiopathic Pulmonary Fibrosis Drugs Makes Inroads in Systemic Sclerosis Market

Author:
Date Published: January-2016
Source: Clinical Trials Arena

Pharmaceutical giant, Boehringer Ingelheim (BI), is poised to begin a Phase III trial of its idiopathic pulmonary fibrosis (IPF) drug, Ofev (nintedanib) as a treatment for systemic sclerosis-related interstitial lung disease (SSc-ILD). Ofev initially gained approval by the Food and Drug Administration (FDA) in October 2014 and by the European Medicines Agency (EMA) in January 2015. Roche and Genentech's Esbriet, arguably BI's closest competitor in the field, is more widely known than Ofev. Despite being available in Europe since 2011, Esbriet gained FDA approval on the same day as Ofev.

EADV: Fresolimumab shows early promise in scleroderma

Author: Bruce Jancin
Date Published: December-2015
Source: Rheumatology News

The recent success of fresolimumab in treating early diffuse cutaneous systemic sclerosis in a proof-of-concept study signals better days ahead in the treatment of scleroderma, Dr. Thomas Krieg predicted at the annual congress of the European Academy of Dermatology and Venereology. “There will be new and better treatments based upon our improved understanding of the pathophysiology of this complex multifaceted disease. New targeted therapies are likely,” according to Dr. Krieg, chairman of the department of dermatology and venereology and dean of the medical faculty at the University of Cologne (Germany).

Recent developments in the classification, evaluation, pathophysiology, and management of scleroderma renal crisis

Author: Cybele Ghossein, John Varga, Andrew Z. Fenves
Date Published: December-2015
Source: Current Rheumatology Reports

Scleroderma renal crisis (SRC) is an uncommon complication of systemic sclerosis. Despite the advent of angiotensin-converting inhibitor therapy, SRC remains a life-threatening complication. Recent studies have contributed to a better understanding of SRC, but much remains unknown regarding its pathophysiology, risk factors, and optimal management. Genetic studies provide evidence that immune dysregulation might be a contributing factor, providing hope that further research in this direction might illuminate pathogenesis and provide novel predictors for this complication.

News for Patients

Experimental Compound Shows Promise in Reversing Skin Disease Associated with Systemic Sclerosis

Author: Collee Labbe
Date Published: January-2016
Source: NIAMS Spotlight on Research

A new drug appears to alter the expression of certain genes associated with systemic sclerosis by blocking a key protein, and also leads to clinical improvements in the skin, according to a study funded in part by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The study was published in the Journal of Clinical Investigation. Systemic sclerosis, a severe form of scleroderma, is a chronic connective tissue disease that affects multiple organs and tissues in the body. It can lead to fibrosis, or hardening, of the skin and other organs such as the lungs, kidneys and heart. Currently, no effective treatments exist. Because the disease affects everyone differently, finding therapies has been challenging.

SteadyMed’s Treyvent Awarded Orphan Drug Designation from FDA

Author: Dainela Semedo
Date Published: January-2016
Source: Pulmonary Hypertension News

SteadyMed, Ltd., a company that develops products to treat orphan and high-value conditions with unmet parenteral delivery needs, recently announced that its lead product candidate, Trevyent® has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH).

Idiopathic Pulmonary Fibrosis Drugs Makes Inroads in Systemic Sclerosis Market

Author:
Date Published: January-2016
Source: Clinical Trials Arena

Pharmaceutical giant, Boehringer Ingelheim (BI), is poised to begin a Phase III trial of its idiopathic pulmonary fibrosis (IPF) drug, Ofev (nintedanib) as a treatment for systemic sclerosis-related interstitial lung disease (SSc-ILD). Ofev initially gained approval by the Food and Drug Administration (FDA) in October 2014 and by the European Medicines Agency (EMA) in January 2015. Roche and Genentech's Esbriet, arguably BI's closest competitor in the field, is more widely known than Ofev. Despite being available in Europe since 2011, Esbriet gained FDA approval on the same day as Ofev.

EADV: Fresolimumab shows early promise in scleroderma

Author: Bruce Jancin
Date Published: December-2015
Source: Rheumatology News

The recent success of fresolimumab in treating early diffuse cutaneous systemic sclerosis in a proof-of-concept study signals better days ahead in the treatment of scleroderma, Dr. Thomas Krieg predicted at the annual congress of the European Academy of Dermatology and Venereology. “There will be new and better treatments based upon our improved understanding of the pathophysiology of this complex multifaceted disease. New targeted therapies are likely,” according to Dr. Krieg, chairman of the department of dermatology and venereology and dean of the medical faculty at the University of Cologne (Germany).

Recent developments in the classification, evaluation, pathophysiology, and management of scleroderma renal crisis

Author: Cybele Ghossein, John Varga, Andrew Z. Fenves
Date Published: December-2015
Source: Current Rheumatology Reports

Scleroderma renal crisis (SRC) is an uncommon complication of systemic sclerosis. Despite the advent of angiotensin-converting inhibitor therapy, SRC remains a life-threatening complication. Recent studies have contributed to a better understanding of SRC, but much remains unknown regarding its pathophysiology, risk factors, and optimal management. Genetic studies provide evidence that immune dysregulation might be a contributing factor, providing hope that further research in this direction might illuminate pathogenesis and provide novel predictors for this complication.

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