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

Borderline pulmonary arterial pressure in systemic sclerosis patients: a post-hoc analysis of the DETECT study

Author: S. Visovatti, O. Distler, J. Coghlan, C. Denton, et al
Date Published: December-2014
Source: Arthritis Research & Therapy

Patients with mean pulmonary artery pressures (mPAP) of 21 to 24mm Hg have a so-called borderline elevation of mPAP (BoPAP)?a condition thought to represent early-stage pulmonary arterial vasculopathy. Based on the DETECT study, this post-hoc analysis examined patient characteristics of systemic sclerosis (SSc) patients with normal mPAP, BoPAP and elevated mPAP, fulfilling pulmonary arterial hypertension (PAH) criteria.

Advances in the Evaluation and Management of Esophageal Disease of Systemic Sclerosis

Author: D. Carlson, M. Hinchcliff, J. Pandolfino
Date Published: December-2014
Source: Current Rheumatology Reports

Symptoms of heartburn and dysphagia as well as objective findings of abnormal esophageal acid exposure and esophageal dysmotility are common in patients with systemic sclerosis (SSc). Treatments for SSc esophageal disease are generally limited to gastroesophageal reflux disease (GERD) treatment with proton pump inhibitors. Progresses made in esophageal diagnostic testing offer the potential for improved clinical characterization of esophageal disease in SSc that may help direct management decisions.

Autotaxin Is Highly Expressed in Systemic Sclerosis (SSc) Skin, Mediates Dermal Fibrosis Via IL-6, and Is a Target for SSc Therapy

Author: F. Castelino, L. George, G. Bain, L. Goulet, R. Lafyatis and A. Tager
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin (ATX) is an enzyme present in biological fluids that is responsible for the production of the lipid mediator, lysophosphatidic acid (LPA). We previously implicated LPA and its receptor, LPA1 in SSc pathogenesis.1 Here we studied the role of ATX in SSc dermal fibrosis using the bleomycin (BLM) mouse model and skin biopsy samples from SSc patients and healthy controls. We evaluated the role of IL-6, a cytokine implicated in SSc, in mediating ATX-induced fibrosis. Additionally, we investigated the therapeutic potential of targeting ATX, by using a novel ATX inhibitor, PAT-048 in this model.

SAR100842, an Antagonist of Lysophaphatidic Acid Receptor 1, As a Potential Treatment for Patients with Systemic Sclerosis: Results from a Phase 2a Study

Author: C. Denton, A. Jagerschmidt, M. Jasson, O. Distler and Y. Allanore
Date Published: December-2014
Source: American College of Rheumatology

Preclinical genetic and pharmacological studies suggest a role for Lysophosphatidic acid (LPA) involvement in three key processes of systemic sclerosis: fibrosis, microangiopathy and immunoinflammation. We have assessed SAR100842, a potent selective orally available antagonist of LPA1 receptor and explored safety, skin biomarkers of disease activity and clinical efficacy in patients with diffuse cutaneous SSc (dcSSc) in a phase 2a clinical trial.

MY ACR2014 Favorites: Systemic Sclerosis Advances and a Challenge

Author: Kristine Lohr, MD
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin is responsible for production of lysophosphatidic acid (LPA), which is implicated in scleroderma pathogenesis. In the first report above, using a murine model of dermal fibrosis, an inhibitor of autotaxin attenuated dermal fibrosis and IL-6 expression. Autotaxin expression was increased in skin from systemic sclerosis patients, compared to healthy controls. The authors concluded that targeting autotaxin may be a therapeutic strategy for scleroderma fibrosis. Combining this information with the second report above, describing efficacy of an oral antagonist of the LPA receptor in a Phase 2a study of systemic sclerosis patients, I offered substituting tocilizumab for adalimumab to my patient with an overlap syndrome [RF/CCP+ RA, primary Sjogren syndrome (+ANA/SSA/SSB) and recent progression of morphea].

News for Patients

MY ACR2014 Favorites: Systemic Sclerosis Advances and a Challenge

Author: Kristine Lohr, MD
Date Published: December-2014
Source: American College of Rheumatology

Autotaxin is responsible for production of lysophosphatidic acid (LPA), which is implicated in scleroderma pathogenesis. In the first report above, using a murine model of dermal fibrosis, an inhibitor of autotaxin attenuated dermal fibrosis and IL-6 expression. Autotaxin expression was increased in skin from systemic sclerosis patients, compared to healthy controls. The authors concluded that targeting autotaxin may be a therapeutic strategy for scleroderma fibrosis. Combining this information with the second report above, describing efficacy of an oral antagonist of the LPA receptor in a Phase 2a study of systemic sclerosis patients, I offered substituting tocilizumab for adalimumab to my patient with an overlap syndrome [RF/CCP+ RA, primary Sjogren syndrome (+ANA/SSA/SSB) and recent progression of morphea].

Autotaxin Protein Can Be Targeted in SSc Therapy

Author: Maureen Newman
Date Published: December-2014
Source: Scleroderma News

A group from Massachusetts General Hospital is placing possible blame for systemic sclerosis (SSc) pathogenesis on autotaxin (ATX), an enzyme involved in the production of the lipid signaling molecule lysophophatidic acid (LPA). Dr. Falvia V. Castelino, a researcher in the Rheumatology Department, presented the group’s work, “Autotaxin is Highly Expressed in SSc Skin, Mediates Dermal Fibrosis Via IL-6, and Is a Target for SSc Therapy,” at the 2014 American College of Rheumatology Meeting in November.

Metabolism Influences Fibrosis in SSc

Author: Maureen Newman
Date Published: December-2014
Source: Scleroderma News

Fibrosis and metabolism: the two were recently linked in a study presented at the 2014 American College of Rheumatology Meeting in November. “Adiponectin Is an Endogenous Anti-Fibrotic and Target in Systemic Sclerosis: Novel Link Between Fibrosis and Metabolism,” presented by Dr. Feng Fang from Northwestern University and Feinberg School of Medicine, described a possible approach to a new pharmacological treatment of systemic sclerosis (SSc) based on these new findings.

Cureveda Receives NIH Grants for Systemic Sclerosis and COPD Drug

Author: Anna Tan, RN
Date Published: December-2014
Source: Lung Disease News

Biotechnology company Cureveda LLC just announced that it has received two National Institutes of Health (NIH) Phase I Small Business Technology Transfer (STTR) grants worth $450,000 in total. Cureveda specializes in treatments for inflammatory and fibrotic diseases, and the new funding will be used for VEDA-1209 preclinical testing as a breakthrough therapeutic for systemic sclerosis and chronic obstructive pulmonary disease (COPD).

FDA Grants Cytori Conditional Approval for a U.S. Pivotal Clinical Trial in Scleroderma

Author: Cytori
Date Published: December-2014
Source: BusinessWire

Cytori Therapeutics, Inc. (NASDAQ:CYTX) today announced that the U.S. Food and Drug Administration (FDA) has granted conditional approval for an Investigational Device Exemption (IDE) for a pivotal clinical trial, named the ‘STAR’ trial, to evaluate Cytori Cell TherapyTM as a potential treatment for impaired hand function in scleroderma, a rare autoimmune disease affecting approximately 50,000 patients in the United States.

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