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].


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.


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.


Localized Scleroderma Characterized by T-Cell Cytokines and Chemokines

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

New Scleroderma-related findings have linked two variations of the disease, which in turn could lead to effective, new biomarkers and targeted treatments for patients. Two clinically different diseases, localized scleroderma (LS) and systemic sclerosis (SSc), can be characterized by similar T-cell produced cytokines and chemokines in pediatric patients, according to recent work presented at the 2014 American College of Rheumatology. At the meeting, Dr. Kathryn S. Torok, from the Pediatric Rheumatology Department of Children’s Hospital of Pittsburgh of the University of Pittsburgh Medical Center, presented “Circulating T-Helper Cell- Associated Cytokines and Chemokines in Localized Scleroderma,” which delved further into the biological characteristics of the only partially-investigated disease LS.


New Scleroderma Criteria Improves Upon Disease Classification

Author: Patricia Inacio
Date Published: December-2014
Source: Scleroderma News

The American College of Rheumatology (ACR), together with the European League Against Rheumatism (EULAR) published new criteria for classification of systemic sclerosis. The new guidelines were introduced to allow earlier diagnosis and facilitate more targeted therapeutic treatments. The criteria were published in the journal of Arthritis & Rheumatism in a report entitled “2013 Classification Criteria for Systemic Sclerosis. An American College of Rheumatology and European League Against Rheumatism Collaborative Initiative.”


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.


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).


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.