News for Patients


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.

 

Systemic Sclerosis Mouse Study of Nintedanib in Fibrotic and Vascular Manifestations Looks Promising

Author: Daniela Semedo
Date Published: February-2016
Source: Scleroderma News

Nintedanib (OFEV) seems to inhibit the activation of fibroblasts and to exert an anti-fibrotic effect in mouse models of systemic sclerosis (SSc), according to data recently presented at the 4th Systemic Sclerosis World Congress in Lisbon, Portugal, last week. The presentation, “Effects of Nintedanib on Fibrotic and Vascular Manifestation in Pre-clinical Models of Systemic Sclerosis,” was given by J. Distler from the Department of International Medicine 3 and the Institute for Clinical Immunology at the University of Erlangen-Nuremberg in Germany.

 

Actelion Stresses Importance of PAH Early Detection, Life Quality at 2016 SSc World Congress and in Exclusive Interview

Author: Patricia Silva
Date Published: February-2016
Source: Scleroderma News

New data was recently presented regarding Actelion Pharmaceuticals’ therapies and strategies to improve clinical outcomes in patients with pulmonary arterial hypertension (PAH). The data were presented at the Actelion Satellite Symposium, titled “Improving outcome in pulmonary arterial hypertension,” which was part of the 4th Systemic Sclerosis World Congress held in Lisbon, Portugal, on Feb. 18-20, 2016.

 

Leading Researcher: Fibrosis Improved in Systemic Sclerosis and IPF Models With Monoclonal Antibody

Author: Margarida Azevedor
Date Published: February-2016
Source: Scleroderma News

Researchers at ChemomAb and Meir Medical Center in Tel Aviv, Israel, and the University of Florence, Italy, recently presented a study investigating the therapeutic effect of the monoclonal antibody CM-101 in animal models of systemic sclerosis (SSc) and idiopathic pulmonary fibrosis (IPF). The data was presented last week in a talk titled “CM-101, a Novel Monoclonal Antibody Blocking CCL24 Ameliorates Experimental Systemic Sclerosis (SSc) and Idiopathic Pulmonary Fibrosis (IPF)” at the 4th Systemic Sclerosis World Congress in Lisbon, Portugal. Systemic sclerosis and IPF are two conditions that involve the development and accumulation of fibrotic tissue and progressive decline of the patient’s health status. 

 

Typical Therapies Not Tough Enough to Curb Digital Ulcers

Author: Damian McNamara
Date Published: February-2016
Source: Medscape

Patients with systemic sclerosis and a recent history of digital ulcers remain at significantly higher risk of developing a new one, according to an observational study. The findings also suggest a common treatment might be insufficient for prevention in this high-risk subset. The study is one of five observational studies that make up the DeSScipher project, sponsored by the Scleroderma Society in London, United Kingdom.

 

PAH Drug, Opsumit, Seen to Prevent and Treat Lung Disease in a Mouse Model of Scleroderma

Author: Margarida Azevedor
Date Published: February-2016
Source: Pulmonary Hypertension News

Researchers at University College London (UCL) and Actelion Pharmaceuticals presented a study investigating the therapeutic effect of macitentan (Opsumit) in a mouse model of pulmonary hypertension associated with systemic scleroderma (SSc). The presentation, titled “Macitentan Responsiveness Supports the Validity of a Murine Model of Pulmonary Hypertension in Scleroderma Associated with altered TGFbeta/BMPRII Signalling,” took place at the recent 4th Systemic Sclerosis World Congress in Lisbon, Portugal.