MD News and Information

Potential beneficial role for endothelin in scleroderma vasculopathy: inhibition of endothelial apoptosis by type B endothelin-receptor signaling

Author: Yongqing Wang, Nezam Altorok, Bashar Kahaleh
Date Published: June-2016
Source: Scleroderma Journal

Microvascular endothelial cell (MVEC) apoptosis is considered to be a key event in the pathogenesis of systemic sclerosis (SSc), an increased expression of endothelin-1 (ET1) is also well recognized in the disease. ET1 is thought to exert deleterious effects on the vasculature by virtue of its known vasospastic, proliferative and fibrotic effects, yet ET1 can act as a survival factor for a variety of cells, including MVEC. The aim of this study is to investigate if ET1 signaling protects SSc-MVECs from apoptosis.

 

iBio’s Endostatin-Related Peptides for Fibrosis, Scleroderma Issued US Patent

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

The pharmaceutical company iBio, which focuses on the clinical development of drugs for systemic sclerosis and other fibrotic diseases, was recently issued a U.S. patent protecting the company’s drug development attempts using endostatin-related peptides for the treatment of fibrosis.

According to a press release, the patent, US 9,365,616, titled “Use of Endostatin Peptides for the Treatment of Fibrosis,” complements earlier U.S. patents 8,507,441 and 8,716,232 covering this line of drug development. The patent covers both the composition of the drugs and the methods for which endostatin-related peptides might be used.

 

An Open-label, Phase II Study of the Safety and Tolerability of Pirfenidone in Patients with Scleroderma-associated Interstitial Lung Disease: the LOTUSS Trial

Author: Dinesh Khanna, Carlo Albera, Aryeh Fischer, Nader Khalidi, Ganesh Raghu, Lorinda Chung, Dan Chen, Elena Schiopu, Margit Tagliaferri, James R. Seiboldand Eduard Gorina
Date Published: June-2016
Source: The Journal of Rheumatology

Systemic sclerosis-associated interstitial lung disease (SSc-ILD) shares a number of clinical features and pathogenic mechanisms with idiopathic pulmonary fibrosis (IPF). This study was designed to evaluate the tolerability of the IPF treatment pirfenidone in SSc-ILD. The known gastrointestinal, skin, and liver adverse events (AE) of pirfenidone are of importance given the involvement of these organs in SSc.

 

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.”

 

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.

 

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.

 
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