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

SRF-funded Harvard Researchers Explore Cellular Self-destruction as Scleroderma Treatment

Author: Magdalena Kegel
Date Published: January-2018
Source: Scleroderma News

Organ fibrosis in scleroderma patients might be reversed with treatments that allow fibrotic cells to self-destruct, Harvard scientists suggest.

Their study, funded by the SRF, “Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis,” appeared in the journal Science Translational Medicine. It demonstrated that the survival–self-destruction balance of fibrosis-producing myofibroblast cells can be tipped by using a compound now being explored as a cancer treatment.

Getting Started on Methotrexate: What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Methotrexate is a medication commonly used to treat rheumatoid arthritis and many other autoimmune and inflammatory conditions. Nurse Victoria Ruffing talks about what you should know when getting started on Methotrexate, as well as the side effects of Methotrexate.

Pathogenesis of systemic sclerosis: recent insights of molecular and cellular mechanisms and therapeutic opportunities

Author: John Varga, Maria Trojanowska, Masataka Kuwana
Date Published: July-2017
Source: Journal of Scleroderma and Related Disorders

Systemic sclerosis (SSc) is a complex disease characterized by early microvascular abnormalities, immune dysregulation and chronic inflammation, and subsequent fibrosis of the skin and internal organs. Excessive fibrosis, distinguishing hallmark of SSc, is the end result of a complex series of interlinked vascular injury and immune activation, and represents a maladaptive repair process. Activated vascular, epithelial, and immune cells generate pro-fibrotic cytokines, chemokines, growth factors, lipid mediators, autoantibodies, and reactive oxygen species. These paracrine and autocrine cues in turn induce activation, differentiation, and survival of mesenchymal cells, ensuing tissue fibrosis through increased collagen synthesis, matrix deposition, tissue rigidity and remodeling, and vascular rarefaction.

The mighty fibroblast and its utility in scleroderma research

Author: Sara M. Garrett, DeAnna Baker Frost, Carol Feghali-Bostwick
Date Published: May-2017
Source: Journal of Scleroderma and Related Disorders

Fibroblasts are the effector cells of fibrosis characteristic of systemic sclerosis (SSc, scleroderma) and other fibrosing conditions. The excess production of extracellular matrix (ECM) proteins is the hallmark of fibrosis in different organs, such as skin and lung. Experiments designed to assess the pro-fibrotic capacity of factors, their signaling pathways, and potential inhibitors of their effects that are conducted in fibroblasts have paved the way for planning clinical trials in SSc. As such, fibroblasts have proven to be valuable tools in the search for effective anti-fibrotic therapies for fibrosis. Herein we highlight the characteristics of fibroblasts, their role in the etiology of fibrosis, utility in experimental assays, and contribution to drug development and clinical trials in SSc.This article is available for you to download for free for 1 week until 2nd August 2017.

Systemic Sclerosis Linked With Altered Gut Microbiome

Author: Gregory M. Weiss, MD
Date Published: July-2017
Source: Rheumatology Network

The numbers of Bacteroidetes bacteria in the GI tracts of 2 separate cohorts of patients with systemic sclerosis were significantly reduced when compared with healthy controls. American patients with systemic sclerosis had more extensive alterations in their intestinal microbiota than those in a Norwegian cohort. An abundance of Prevotella species was associated with moderate-to-severe GI symptoms in patients with systemic sclerosis. Clostridium species abundance was associated with low GI symptom severity, and Lactobacillus with none-to-mild constipation.

News for Patients

SRF-funded Harvard Researchers Explore Cellular Self-destruction as Scleroderma Treatment

Author: Magdalena Kegel
Date Published: January-2018
Source: Scleroderma News

Organ fibrosis in scleroderma patients might be reversed with treatments that allow fibrotic cells to self-destruct, Harvard scientists suggest.

Their study, funded by the SRF, “Targeted apoptosis of myofibroblasts with the BH3 mimetic ABT-263 reverses established fibrosis,” appeared in the journal Science Translational Medicine. It demonstrated that the survival–self-destruction balance of fibrosis-producing myofibroblast cells can be tipped by using a compound now being explored as a cancer treatment.

Getting Started on Methotrexate: What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Methotrexate is a medication commonly used to treat rheumatoid arthritis and many other autoimmune and inflammatory conditions. Nurse Victoria Ruffing talks about what you should know when getting started on Methotrexate, as well as the side effects of Methotrexate.

Raynaud’s Phenomenon : What You Should Know

Author: Johns Hopkins
Date Published: January-2018
Source: Johns Hopkins Rheumatology

Raynaud’s Phenomenon is a condition that results in decreased blood flow to your fingers and toes. There are two types of Raynaud’s: primary and secondary. SRF-funded investigator, Dr. Zsuzsanna McMahan from the Johns Hopkins Scleroderma Center dives into what this condition is, things to look out for, how to manage it, and when you should speak with a doctor.

Uptravi Slows Progression of Pulmonary Hypertension Stemming from Connective Tissue Disease, Study Finds

Author: Iqra Mumal
Date Published: August-2017
Source: Pulmonary Hypertension News

Actelion’s Uptravi (selexipag) delays the progression of pulmonary arterial hypertension that stems from connective tissue disease, a follow-up analysis of Phase 3 clinical trial findings indicates. People with PAH-CTD also tolerate the therapy well, according to the study. The PAH part of PAH-CTD refers to high blood pressure in the lungs’ arteries.

Prucalopride Can Help Improve Gastrointestinal Symptoms in SSc Patients, Study Shows

Author: Alice Melao
Date Published: August-2017
Source: Scleroderma News

Prucalopride, marketed under the name Resolor in Europe and Resotran in Canada, can safely manage mild to severe gastrointestinal (GI) symptoms associated with systemic sclerosis (SSc), including bowel movement and transit, reflux disease, and bloating, finds a study published in the journal Arthritis Research and Therapy. Doctors have proposed using medicines to improve prokinetics, or motility of the GI tract, in treating SSc patients. But the lack of proper clinical trials demonstrating the therapeutic potential of such drugs in the SSc population discouraged their use in clinics.

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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