New Hope for Patients PDF Print E-mail
Monday, 30 May 2011 20:40
taken from http://www.curecancerproject.orgPulmonary Fibrosis (PF) and Pulmonary Arterial Hypertension (PAH) are incurable rare lung diseases and are the leading cause of mortality in patients with Systemic Sclerosis (SSc). However patients might have a reason to breathe easier since researchers from the University of Pittsburgh now provide a novel insight that explains how Systemic Sclerosis develops.

Systemic Sclerosis (SSc) is a rare autoimmune disease which affects the connective tissue of the skin and internal organs, such as lungs, kidneys or heart. It is often accompanied by pulmonary complications, Pulmonary Fibrosis (PF) and Pulmonary Arterial Hypertension (PAH), for which therapies have only a low benefit and patients often require a lung transplantation. Despite previous research, how the disease develops remains poorly understood.

How SSc develops has eluded researchers, because they have lacked the necessary samples of affected tissue from patients. That is why Dr. Carol A. Feghali-Botswick’s research team from the University of Pittsburgh School of Medicine started collecting lung tissue samples from affected patients.

“The challenge was collecting a critical number of lung tissues from patients with SSc,” said Dr. Carol A. Feghali-Botswick. A certain number of lung tissue samples from patients with Systemic Sclerosis was necessary to obtain good results, but as lung biopsies are not routinely done the availability was fairly limited.

“We started banking these tissues in the mid-1990s and had to wait until we accrued a sufficient number,” explained Dr. Feghali-Botswick. “The University of Pittsburgh Medical Center has a vibrant lung transplant program and transplants more SSc patients than anywhere else in the country or the world, so we were uniquely poised to do this study once we had enough patients donate their lungs.” Finally they managed to analyze lung tissues samples from 33 patients with SSc who underwent lung transplantations. They created a list of characteristics, so called gene expression profiles, which were unique or shared throughout the samples.

With the data in hand, Dr. Feghali-Botswick’s team was able to generate signatures that were specific for SSC as well as signatures that were specific for the two complications: Pulmonary Fibrosis (PF) and Pulmonary Arterial Hypertension (PAH). These signatures provided Dr. Feghali-Botswick’s team with insights to genes that might be involved in causing SSc.

To follow up this successful work the researchers are continuing the next phase of the project. “We are currently looking at some of the genes identified through the analysis to determine if they can serve as markers of lung disease in SSc, and possibly even as predictors of lung involvement.“

Markers, such as biomarkers, are substances used to examine an organ’s function or other aspects of health. It is an important tool in diagnostics that allows for easy disease detection, limits the risks of misdiagnosis and also reduces the costs needed for treatment.

In the ongoing search for potential markers, Dr. Feghali-Botswick told CheckOrphan, “Some patients have serum samples obtained prior to the onset of their lung disease as well as following the diagnosis of lung involvement”. This way the researchers will be able to measure the proteins in the serum and to see if some of the protein levels increase prior to the onset of lung involvement or only following the development of lung disease.

“In the long run, this will allow us to develop a panel of proteins we can use for screening patients at risk of developing lung disease with the goal of initiating treatment very early when it's likely to be more effective,” Dr. Feghali-Botswick said.

“I would like to emphasize that none of this work would have been possible without the generosity of our patients who are always willing to donate blood, skin biopsies and lung tissues following lung transplantation surgery.“

Malisova, B. (2011), "New Hope for Patients With Rare Lung Diseases, Systemic Sclerosis (SSc)"; original article can be accessed here.
 
More articles :

» Anticoagulation of Systemic Sclerosis Patients In Question

Australia is in a state of ‘clinical equipoise’ over whether to anticoagulate systemic sclerosis patients with pulmonary hypertension, experts say. And with observational studies showing the treatment is associated with a  fivefold...

» Analytical Chemistry Used In Systemic Sclerosis Study

Scientists have used methods to identify new target autoantigens in patients with systemic sclerosis (SSc).The study, published by , stated that antinuclear antibodies (ANA) can normally be found in 90 per cent of SSc patients with the use of...

» Air Pollution Linked to Autoimmune Diseases

Many people with environmental illnesses have an assortment of autoimmune diseases which scientists are linking to air pollution.Though genetic factors have been found for autoimmune diseases, this knowledge has not helped to prevent them. In fact,...

» Positive Phase II Results for AIMSPRO In Scleroderma Announced

announced yesterday, positive results from its Double-Blind Placebo-Controlled Phase II Clinical Study evaluating the safety and tolerability of AIMSPRO given as a monotherapy to patients with Late Stage Established Diffuse Cutaneous Systemic...

» The Scleroderma Foundation Teams Up with Traitwise

The has teamed up with Austin, TX company, Incorporated, to launch a unique survey to poll thousands of patients and their caregivers. Data from the survey will be shared with researchers nationally and internationally in an effort to improve...

» PDE-5 Inhibitors in Scleroderma Raynaud Phenomenon and Digital Ulcers: Current Status of Clinical Trials

Ann J. Impens, Kristine Phillips, and Elena SchiopuReceived 2 June 2011; Accepted 2 August 2011Phosphodiesterases (PDEs) are isoenzymes that control the level of intracellular cyclic guanosine monophosphate (cGMP) and cyclic adenosine monophosphate...