News for Patients

Prognosis in Scleroderma: Sex and the Joints

Author: Nancy Walsh
Date Published: October-2014
Source: MedPage Today

Creation of the European League Against Rheumatism (EULAR)'s scleroderma registry -- the largest in the world -- has permitted researchers to examine risk factors for disease progression and outcomes, including the effects of male sex and early clinical signs in two recent reports. Scleroderma's Gender Gap Men affected with systemic sclerosis (SSc) have a strikingly worse disease phenotype than women, European researchers reported online in Annals of the Rheumatic Diseases.


New Study Pinpoints Complex Genetic Origins for Autoimmune Diseases

Author: Jeffrey Norris
Date Published: October-2014
Source: UCSF

Scores of autoimmune diseases afflicting one in 12 Americans — ranging from type 1 diabetes, to multiple sclerosis (MS), to rheumatoid arthritis, to asthma — mysteriously cause the immune system to harm tissues within our own bodies. Now, a new study pinpoints the complex genetic origins for many of these diseases, a discovery that may lead to better diagnosis and ultimately to improved treatments. A team of scientists from UC San Francisco, the Broad Institute of MIT and Harvard, and Yale School of Medicine developed a new mathematical tool to more deeply probe existing DNA databases. In so doing they discovered how certain DNA variations, when inherited, are likely to contribute to disease.


FDA Approves Esbriet® (pirfenidone) for the Treatment of Idiopathic Pulmonary Fibrosis (IPF) in the United States

Author: InterMune
Date Published: October-2014
Source: InterMune Press Release

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced that the U.S. Food and Drug Administration (FDA) has approved Esbriet® (pirfenidone) as a treatment for idiopathic pulmonary fibrosis (IPF) in the United States. IPF is a fatal disease caused by progressive scarring (fibrosis) of the lungs, which makes breathing difficult and prevents the heart, muscles and vital organs from receiving enough oxygen to work properly. The disease can advance quickly or slowly, but eventually the lungs will harden and stop working altogether.


Boehringer Ingelheim’s OFEV™ (nintedanib*) approved by the FDA for the treatment of idiopathic pulmonary fibrosis

Author: Dr. Kristin Jakobs
Date Published: October-2014
Source: BusinessWire

Boehringer Ingelheim announced that the US Food and Drug Administration (FDA) has approved OFEV™ (nintedanib*) for the treatment of idiopathic pulmonary fibrosis (IPF), a debilitating and fatal lung disease, which has a median survival of 2-3 years after diagnosis. Until today there were no FDA-approved treatments for IPF. Granted Breakthrough Therapy designation during its review by the FDA, nintedanib* is the first and only tyrosine kinase inhibitor (TKI) approved to treat IPF. Nintedanib* is taken as one capsule twice daily and will be available to patients within 10 days.


Scientists make important breakthrough in fight against debilitating autoimmune diseases

Author: University of Bristol
Date Published: September-2014
Source: News Medical

cientists have made an important breakthrough in the fight against debilitating autoimmune diseases such as multiple sclerosis by revealing how to stop cells attacking healthy body tissue. Rather than the body's immune system destroying its own tissue by mistake, researchers at the University of Bristol have discovered how cells convert from being aggressive to actually protecting against disease.


Extra Help With Medicare Prescription Drug Plan Costs

Author: Social Security Administration
Date Published: October-2014
Source: Social Security Administration

Welcome! The Medicare Prescription Drug program gives you a choice of prescription plans that offer various types of coverage. You may be able to get extra help to pay for the monthly premiums, annual deductibles, and co-payments related to the Medicare Prescription Drug program. However, you must be enrolled in a Medicare Prescription Drug plan to get this extra help.


Interstitial Pneumonia? Rheumatic Disease Has Better Odds Than Idiopathic

Author: Rita Baron-Faust
Date Published: October-2014
Source: Rheumatology Network

Patients who have connective tissue disease (CTD) survive longer with interstitial pneumonia than those with idiopathic pulmonary fibrosis (IPF), according to new research, although declines in pulmonary function over time appear similar in both groups. Researchers from the National Jewish Health center in Denver speculate that the worse outcome in IPF patients is due to a higher rate of fatal acute exacerbations of pulmonary fibrosis than among CTD patients with “usual” interstitial pneumonia.


The Brighter Side of Living With Chronic Illness: 6 Amazing Things You Know Better Than Most

Author: Lottie V. Ryan
Date Published: October-2014
Source: HuffPost Healthy Living

The diagnosis of any chronic illness comes with much you wish you didn't have to carry, and suffer with, for the rest of your life. You learn the scales of pain, you learn the bureaucracy of the health care system, you learn to grieve for abilities and opportunities lost, and so much more. I know this; I am living with chronic illness too. Yet all is not lost. Your chronic illness also teaches you many great things that offer enormous reward in life, and sometimes it's good to take a moment to acknowledge this brighter side of your existence.


Data on Systemic Sclerosis Drug Candidate To Be Presented at Rare Disease Roundtable

Author: L. Ferreira
Date Published: October-2014
Source: Scleroderma

The chief executive officer of clinical stage biopharmaceutical company Corbus Pharmaceuticals Holdings, Yuval Cohen, Ph.D., is presenting an update on the company’s research, as well as revision of previous clinical developments regarding Corbus’s lead product candidate, Resunab, at the Leerink Partners Rare Disease Roundtable. The product candidate is being studied by the drug developer as a possible treatment for rare, life-threatening inflammatory diseases, such as Systemic Sclerosis and Cystic Fibrosis.


Reversing the effects of pulmonary fibrosis with a microRNA mimic

Author: Jim Shelton
Date Published: September-2014
Source: YaleNews

Yale University researchers are studying a potential new treatment that reverses the effects of pulmonary fibrosis, a respiratory disease in which scars develop in the lungs and severely hamper breathing. The treatment uses a microRNA mimic, miR-29, which is delivered to lung tissue intravenously. In mouse models, miR-29 not only blocked pulmonary fibrosis, it reversed fibrosis after several days. The findings were published Sept. 19 in the journal EMBO Molecular Medicine.


New Report Identifies Key Features in Systemic Sclerosis

Author: Ana de Barros
Date Published: September-2014
Source: Pulmonary Hypertension News

Systemic sclerosis (SSc) is a disease that affects multiple organs, with the heart being frequently affected and correlating with a poor outcome for the patient. Different heart structures can be affected leading to pericardial disease, arrhythmias, conduction system abnormalities, direct myocardial disease such as pulmonary arterial hypertension, myositis, cardiac failure, cardiac fibrosis, coronary artery diseases and, sometimes, primary valvular involvement. A group of researchers has recently reviewed key studies concerning cardiac arrhythmias and conduction defects in patients suffering from SSc, a wide-ranging report entitled, “Cardiac arrhythmias and conduction defects in systemic sclerosis,” and published in the journal Rheumatology.


Screening for interstitial lung disease in systemic sclerosis: performance of high-resolution CT with limited number of slices: a prospective study

Author: T. Frauenfelder, A. Winklehner, T. Nguyen, R. Dobrota, S. Baumueller, B. Maurer, O. Distler
Date Published: September-2014
Source: Annals of the Rheumatic Diseases

Early diagnosis of interstitial lung disease (ILD), currently the main cause of death in systemic sclerosis (SSc), is needed. The gold standard is high-resolution CT (HRCT) of the chest, but regular screening faces the risk of increased radiation exposure. We performed a prospective validation of a dedicated, 9-slice HRCT protocol with reduced radiation dose for the detection of ILD in patients with SSc.


Recent advances in scleroderma-associated pulmonary hypertension.

Author: Kristen Highland
Date Published: September-2014
Source: Current Opinion in Rheumatology

Purpose of review: This review summarizes recent advances in pulmonary hypertension, a leading cause of morbidity and mortality in scleroderma (SSc). Recent findings: Although WHO Group I pulmonary arterial hypertension (PAH) is the most common cause of pulmonary hypertension, all WHO Groups can occur. PAH is now a criterion for the diagnosis of SSc. Results of recent research have resulted in greater insight into the epidemiology of SSc-pulmonary hypertension with regard to prevalence, incidence and clinical risk factors.


Patient-derived stem cells shed light on pulmonary hypertension

Author: Leigh MacMillan
Date Published: September-2014
Source: Vanderbilt University

Pulmonary arterial hypertension (PAH) – high blood pressure in the lungs – has been linked to both heritable and idiopathic (spontaneous) causes. The underlying vascular changes that cause PAH are unclear and previous attempts to study changes in patient tissues have been limited by end-stage disease effects.


New Targets For Pulmonary Arterial Hypertension Treatments Identified By Researchers

Author: Patricia Inacio
Date Published: September-2014
Source: Pulmonary Hypertension News

A new study published in September’s issue of the American Journal of Respiratory and Critical Care Medicine, entitled, “The Sphingosine Kinase 1 / Sphingosine-1-Phosphate Pathway in Pulmonary Arterial Hypertension” reports the discovery of two novel therapeutic targets to treat pulmonary arterial hypertension.


Editorial: update on scleroderma and other fibrosing syndromes.

Author: Soumya Chatterjee
Date Published: September-2014
Source: Current Opinion in Rheumatology

With the turn of the century, the knowledge of the pathogenesis of fibrosing disorders has revolutionized, largely because of better understanding of the molecular mechanisms of fibrosis. This section update gives us a glimpse of the recent advances in the field from internationally renowned experts in scleroderma (SSc) and other fibrosing syndromes.


Scleroderma: the role of serum autoantibodies in defining specific clinical phenotypes and organ system involvement.

Author: Robyn Domsic
Date Published: September-2014
Source: Current Opinion in Rheumatology

Purpose of review: To discuss recent advances in serologic testing for systemic sclerosis (SSc)-associated antibodies with respect to the diagnosis and prognosis of the disease. Recent findings: The importance of SSc antibodies for diagnosis has become increasingly recognized, as evidenced by incorporation into the 2013 American College of Rheumatology/the European League Against Rheumatism clinical classification criteria for SSc.


Emerging cellular and molecular targets in fibrosis: implications for scleroderma pathogenesis and targeted therapy.

Author: Castelino FV, Varga J.
Date Published: September-2014
Source: Current Opinion in Rheumatology

PURPOSE OF REVIEW: To summarize the recent advances in understanding the novel cytokine pathways, intracellular signaling molecules, cell-fate decisions, cellular aging and senescence, and the cross-talk of effector cells and the extracellular matrix (ECM) in the pathogenesis of systemic sclerosis (SSc) fibrosis. RECENT FINDINGS: Studies from the animal models and human beings implicate novel molecular pathways such as Wnts, the chemokines, chemokine (C-X-C motif) ligand 4 and chemokine (C-C motif) ligand 2, and the lipid mediators lysophosphatidic acid and sphingosine-1-phosphate in the pathogenesis of SSc. These signals, coupled with the mesenchymal cell-fate decisions, contribute to aberrant fibroblast activation and myofibroblast accumulation.


Update on scleroderma-associated interstitial lung disease.

Author: Fan MH, Feghali-Bostwick CA, Silver RM.
Date Published: September-2014
Source: Current Opinion in Rheumatology

PURPOSE OF REVIEW: Systemic sclerosis (SSc), or scleroderma, is a heterogeneous and complex autoimmune disease characterized by varying degrees of skin and organ fibrosis and obliterative vasculopathy. The disease results in significant morbidity and mortality, and to date, available treatments are limited. Lung involvement is the leading cause of death of patients with SSc. Over the past year, significant advances have been made in our understanding of SSc-associated lung disease, and this review attempts to encapsulate these most recent findings and place them in context.


FDA Grants Arena Pharmaceuticals’ PAH Investigational Drug Orphan Drug Status

Author: Leonor Mateus Ferreira
Date Published: September-2014
Source: Pulmonary Hypertension News

The U.S. Food and Drug Administration has granted APD811, an investigational drug candidate internally developed by Arena Pharmaceuticals, orphan drug status. APD811 is an oral agonist of the prostacyclin (IP) receptor, which is designed for the treatment of vasospastic diseases, such as pulmonary arterial hypertension (PAH). “The FDA Office of Orphan Products Development (OOPD) evaluates scientific and clinical data submissions from sponsors to identify and designate drug candidates that could potentially treat rare diseases to help advance the evaluation and development of such products,” explained Craig M. Audet, Ph.D., Arena’s senior vice president of operations and head of global regulatory affairs.