News for Patients


Being Disfigured by Scleroderma Gave Me the Chance to Love Myself

Author: Lisa Goodman-Helfand
Date Published: June-2016
Source: Dr. Oz, The Good Life

Nobody escapes adolescence unscarred by the agony of bullies, peer pressure, acne, and hormones. Along with every survivor of that awkward era, I, too, faced these obstacles. But I had the added bonus of navigating a newly diagnosed autoimmune disease: scleroderma.

I was 10 years old when doctors introduced me to the unknown world of scleroderma. It was 1985, long before Google could tell you every horrifying detail about your illness. I had no idea scleroderma could affect a patient externally, causing the skin to tighten and result in disfigurement and other painful physical abnormalities. Nor did I know that in its worst form, scleroderma attacks one's internal organs and can be fatal. In an effort to shield me from the complexities of scleroderma, my mom told me that my skin was tighter than normal, but that I was perfectly fine otherwise.

 

Scleroderma Patients and Lung Transplantation

Author: John Hansen-Flaschen, MD
Date Published: June-2016
Source: ATS News

Two studies published in the June issue of Annals of the American Thoracic Society look at scleroderma and lung transplantation. Patients with advanced lung disease due to systemic sclerosis (SSC) have long been considered poor candidates for lung transplantation. However, in a retrospective study of lung transplant outcomes over an eight-year period at the University of Pittsburgh, Maria M. Crespo. MD, and colleagues found that one- and five-year survival in patients with scleroderma are similar to pulmonary fibrosis. The results, they wrote, “indicate that lung transplant is a reasonable treatment option in selected patients with scleroderma.”

 

Phase 3 Trial to Begin for Terguride in Diffuse Cutaneous Scleroderma

Author: Magdalena Kegel
Date Published: June-2016
Source: Scleroderma News

The German pharmaceutical company Medac, in August, will begin Phase 3 of a clinical trial to access the therapeutic potential of terguride, a disease-modifying drug for the treatment of diffuse cutaneous systemic sclerosis.

Terguride is a serotonin receptor blocker, acting at two particular serotonin receptors called 5-HT2A and 5-HT2B. Medac is convinced that stopping the nerve signaling in serotonin neurons will block the progression of fibrotic tissue development and blood vessel remodeling in organs affected by diffuse cutaneous systemic sclerosis.

 

Pfizer, Novartis, BMS join $45M round for early fibrotic disease startup

Author: Stacy Lawrence
Date Published: June-2016
Source: Fierce Biotech

A trio of pharmas have bought into the early vision of startup Blade Therapeutics. Pfizer Venture Investments, Novartis Institutes for Biomedical Research and Bristol-Myers Squibb ($BMY) all came in to the $45 million Series B round for the startup, which aims to address novel targets in fibrotic disease. It expects to get into the clinic with this financing.

Founded in the fall of 2015, Blade is based upon technology in-licensed from Johns Hopkins University, in particular, on work from the lab of Dr. Harry Dietz--a professor of genetics and medicine at the university and the company’s founder. His work focuses on the cause of a pair of fibrotic diseases: Marfan Syndrome and Stiff Skin Syndrome, which forms the basis of Blade’s investigation into new biological pathways involved in tissue fibrosis and dysfunction.

 

Progression of Lung Fibrosis in Scleroderma May Be Predicted by Antibody-Specific T-cells

Author: Magdalena Kegel
Date Published: May-2016
Source: Scleroderma News

Immune T-cells induced by the presence of autoantibodies against topoisomerase-I were linked to lung fibrosis and found to predict disease progression in scleroderma patients. The finding may open new avenues of research into treatments that selectively target the various tissue-specific disease manifestations of scleroderma — a substantial improvement to the nonselective immunosuppression used today.

In scleroderma, autoantibodies targeting specific structures are linked to various clinical representations of the disease. One such antibody against the enzyme topoisomerase-I, crucial for the winding and unwinding of DNA molecules during gene expression and cell divisions, is present in 20 percent to 45 percent of scleroderma patients and linked to diffuse skin disease and lung fibrosis. Patients with anti-topoisomerase-I antibodies also have more severe disease.

 

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial

Author: H. Ghofrani, F. Grimminger, E. Grünig, Y. Huang, et al
Date Published: April-2016
Source: The Lancet

Pulmonary arterial hypertension is a chronic disease associated with poor long-term outcomes. Identifying predictors of long-term outcome in pulmonary arterial hypertension is important to assess disease severity and guide treatment. We investigate associations between efficacy parameters and long-term outcomes in patients with pulmonary arterial hypertension receiving riociguat in the PATENT-2 study. We also present safety and efficacy data from the final data cutoff of PATENT-2, where most patients had received at least 2 years of riociguat treatment.

 

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial

Author: H. Ghofrani, F. Grimminger, E. Grünig, Y. Huang, et al
Date Published: April-2016
Source: The Lancet

Pulmonary arterial hypertension is a chronic disease associated with poor long-term outcomes. Identifying predictors of long-term outcome in pulmonary arterial hypertension is important to assess disease severity and guide treatment. We investigate associations between efficacy parameters and long-term outcomes in patients with pulmonary arterial hypertension receiving riociguat in the PATENT-2 study. We also present safety and efficacy data from the final data cutoff of PATENT-2, where most patients had received at least 2 years of riociguat treatment.

 

Predictors of long-term outcomes in patients treated with riociguat for pulmonary arterial hypertension: data from the PATENT-2 open-label, randomised, long-term extension trial

Author: Patricia Inacio
Date Published: April-2016
Source: Scleroderma News

Researchers recently discovered a natural molecule, the M10 peptide, that can significantly decrease fibrosis in a mouse model of scleroderma. The study, “M10, a caspase cleavage product of the hepatocyte growth factor receptor, interacts with Smad2 and demonstrates antifibrotic properties in vitro and in vivo,” was published in the journal Translational Research. Systemic sclerosis, also known as scleroderma, is a chronic systemic autoimmune disease characterized by excessive deposition of collagen, leading to fibrosis in several organs, which can ultimately result in organ failure. Effective treatments targeting fibrosis in systemic sclerosis patients are lacking.

 

Large Scleroderma Study Offers Genetic Map, Identifies Patients at Risk for Pulmonary Hypertension

Author: Margarida Azevdeo
Date Published: April-2016
Source: Scleroderma News Today

A research team with scientists from the University of Granada in Spain and the Spanish National Research Council (CSIC) has carried out the largest scleroderma study to date, creating a complete genetic map of the disease with the genetic analysis of more than 5,000 patients.

 

FDA Approves Corbus Phase 2 Trial Extension of Systemic Sclerosis Drug Resunab

Author: Kara Elam
Date Published: April-2016
Source: Lung Disease News

Corbus Pharmaceuticals, a clinical stage company targeting rare, chronic inflammatory and fibrotic diseases, recently received approval by the U.S. Food and Drug Administration (FDA) for a 12-month open-label extension trial of the ongoing Phase 2 study testing Resunab as a therapy for diffuse cutaneous systemic sclerosis.

 

Cardiovascular Focus: Scleroderma Presentations from FESCA

Author: FESCA
Date Published: February-2016
Source: 4th Systemic Sclerosis World Congress

In the nearly hour-long video, linked below, several engaging experts detail the ways that scleroderma can impact cardiovascular function. Professor Oliver Distler, University Hospital Zurich, brings participants through pulmonary changes arising from fibrosis, detailing testing regimen, and identifying targets for anti-fibrotic medications in clinical trials.

 

Prediction of improvement in skin fibrosis in diffuse cutaneous systemic sclerosis: a EUSTAR analysis

Author: Rucsandra Dobrota, Britta Maurer1, Nicole Graf, Suzana Jordan, Carina Mihai, Otylia Kowal-Bielecka, Yannick Allanore, Oliver Distler
Date Published: March-2016
Source: Annals of the Rheumatic Diseases

Improvement of skin fibrosis is part of the natural course of diffuse cutaneous systemic sclerosis (dcSSc). Recognising those patients most likely to improve could help tailoring clinical management and cohort enrichment for clinical trials. In this study, we aimed to identify predictors for improvement of skin fibrosis in patients with dcSSc.

 

2016 FESCA Video Lecture - What Do We Know About Scleroderma Now & What Will Help Digestive Problems

Author: C. Denton / J. Clarke
Date Published: February-2016
Source: FESCA

Video from the 4th Systemic Sclerosis World Congress held February 18-20 in Lisbon, Portugal, this video features Dr. Chris Denton with the Royal Free Hospital in the UK and Dr. John Clarke with the Johns Hopkins Scleroderma Center.

 

Scleroderma Patients Needed for Research Study to Evaluate Internet Self-Management Program

Researchers from the Universities of New Mexico and Michigan and the Medical University of South Carolina are seeking participants for a study to evaluate a self-management program for people with systemic sclerosis.

To be eligible, you must have been diagnosed with limited or diffuse systemic sclerosis, be 18 years of age, possess basic computer literacy and have access to a computer with Internet and email capabilities, be able to communicate in written English, reside in the United States, and be willing to compete the study protocol.

You will complete a packet of questionnaires at 3 points in time: before the intervention, after the intervention is completed and 6 months later. You will be randomly assigned to an internet intervention group or a control group. If you are assigned to the internet group, you will access and complete the internet self-management program at home over a 16-week time period and participate in the Discussion board. If you are assigned to the control group, you will receive an educational book written by an expert on systemic sclerosis; at the end of the study, you will also have the chance to access the internet self-management program.

This study was approved by the UNM Human Research Review Committee (HRRC#14 -369). For more information about the study and the eligibility requirements please contact Jennifer Serrano at scleroderma-selfmanagement@umich.edu (preferred) or 734-232-2119 or Janet Poole at jpoole@salud.unm.edu.

 

 

New Cannabinoid-based Drug Slows Fibrosis, Blocks Inflammation in Early Study

Author: Magadelna Kegel
Date Published: March-2016
Source: Scleroderma News

A research team has developed a cannabinoid-based drug that stimulates not only one, but two, receptors believed to be involved in fibrosis development in scleroderma. The drug, VCE-004.8, prevented the formation of fibrosis-promoting myofibroblasts in culture and stopped fibrotic changes in a mouse model of dermal fibrosis. Findings from these pre-clinical studies, while early, are promising and may lead to a new therapy for SSc. Scientists have increasingly realizing that the PPAR-γ receptor is involved in inflammatory processes, and drugs stimulating the receptor can prevent inflammation and fibrosis in a mouse model of skin fibrosis. PPAR-γ is activated by cannabinoids, which have, in turn, also been shown to modulate fibrotic development.

 

Prediction of improvement in skin fibrosis in diffuse cutaneous systemic sclerosis: a EUSTAR analysis

Author: R. Dobrota1,B. Maurer, N. Graf, S. Jordan, C. Mihai, O. Kowal-Bielecka, Y. Allanore, O. Distler on behalf of EUSTAR coauthors
Date Published: March-2016
Source: Annals of the Rheumatic Diseases

Improvement of skin fibrosis is part of the natural course of diffuse cutaneous systemic sclerosis (dcSSc). Recognising those patients most likely to improve could help tailoring clinical management and cohort enrichment for clinical trials. In this study, we aimed to identify predictors for improvement of skin fibrosis in patients with dcSSc.

 

Stem Cell Treatment Shows Promise as Therapy for Multiple Sclerosis

Author: Rose Rimler
Date Published: March-2016
Source: Healthline

Twenty years ago, Dr. Alan Tyndall, a rheumatologist at University Hospital in Basel, Switzerland, was faced with giving a 37-year-old mother a grim diagnosis. Scleroderma, the autoimmune disease that pumps excess collagen into the body, was turning her pulmonary arteries into stone. The disease would be fatal. Even a lung transplant couldn’t save her. So Tyndall and his colleagues, including hematologist Dr. Alois Gratwohl, came up with a bold plan.

 

Cytori Update on Its U.S. Phase III Scleroderma Trial

Author: Cytori
Date Published: March-2016
Source: Cytori

Cytori Therapeutics, Inc. today announced that its U.S. FDA approved Phase III STAR trial has enrolled and treated its 40th patient (50% of target enrollment). In addition, a pre-specified independent data monitoring committee review of safety data from the initial 20 patients has been conducted and the committee has recommended that the study continue as planned. “We remain encouraged by how well the trial procedures have been tolerated and how the procedures have been instituted across the 20 study sites around the country. We are grateful to the investigators, research staff and especially the patients who have participated and enabled us to remain on track to complete enrollment in mid-2016,” said Dr. Steven Kesten, Chief Medical Officer, Cytori Therapeutics.

 

ProMetic Launches Phase II of Anti-Fibrotic PBI-4050

Author: ProMetic
Date Published: March-2016
Source: ProMetic

ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“ProMetic” or the “Corporation”) today announced that it will be initiating a double-blind, placebo-controlled phase 2 clinical trial in patients suffering from scleroderma. There is no cure for scleroderma, a chronic disorder characterized by an overproduction of collagen and abnormal growth of connective tissue. This collagen accumulation causes scarring (fibrosis) of the skin, and in the case of systemic scleroderma, also affects internal organs such as the lungs, the kidneys and gastrointestinal system. Scleroderma affects approximately 300,000 individuals in North America alone and varies in severity.

 

Muscle Weakness Predicts Disability in Scleroderma

Author: Wayne Kuznar
Date Published: March-2016
Source: MedPage Today

Muscle weakness was independently associated with disability in patients with scleroderma, and as muscle weakness worsened, disability also increased, according to findings from a retrospective, nested, case control study. Patients with muscle weakness had an average Health Assessment Questionnaire-Disability Index (HAQ-DI) of 1.4, which indicates moderate to severe disability, according to Julie J. Paik, MD, MHS, and colleagues from Johns Hopkins University in Baltimore.