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Format: 04/18/2014
Format: 04/18/2014

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AGH Pulmonary Hypertension Researchers Tap Unique Gathering of Patients in Orlando to Advance Understanding, Treatment of Deadly Lung Disease

Monday, June 25th, 2012

Difficulty Recruiting Study Participants Poses Big Challenge to Rare Disease Specialists

For physicians and medical researchers who specialize in rare diseases, a significant challenge to conducting effective basic and clinical research that may offer the hope of improved treatment, if not a cure, is often finding enough patients to participate in studies. This week in Orlando, Florida, a group of the nation’s leading specialists in one such disease, a deadly lung condition called pulmonary hypertension (PH), enjoyed the unique experience of having hundreds of willing patients come to them over the course of just a few days.

Teams representing nine of the country’s preeminent PH research centers, including Allegheny General Hospital (AGH) in Pittsburgh, convened at the Pulmonary Hypertension Association’s International Conference and Scientific Sessions on June 22-24 to promote the launch of major studies aimed at advancing knowledge about the disease. Other centers participating included Vanderbilt University, the Cleveland Clinic and Brigham and Women’s Hospital.

Though attendance at most scientific meetings is relegated almost exclusively to medical and research professionals, the PHA encourages patients to play an active role at its biannual meetings to help support critical research projects in the field. More than 200 patients who attended the PHA’s last conference in 2010 became research subjects.

The research teams collected data on-site from patients through blood draws, echocardiograms, medical histories, and questionnaires.

Pulmonary hypertension is a disease in which high blood pressure in the lungs causes the arteries between the heart and lungs to narrow and stiffen, ultimately resulting in heart failure. The disease is treatable but has no cure.

AGH’s study, led by Raymond L. Benza, MD, Medical Director of the hospital’s Advanced Heart Failure, Transplant and Pulmonary Vascular Disease Program, will involve performing a global proteomic analysis of plasma from PH patients with the goal of identifying better biomarkers for the disease and advancing the diagnostic capabilities of physicians.

Proteomics (the large scale study of proteins) is an emerging field that has the potential to uncover new therapeutic targets for the treatment and prevention of cardiovascular diseases, as well as new diagnostic biomarkers for early disease detection.

"The data obtained from our analyses will give us a better understanding of PH and hopefully lead to more effective diagnostic protocols and therapeutic options,” Dr. Benza said.

The AGH study aims to enroll 100 PH patients at the PHA conference as well as 100 healthy control participants. In addition to Dr. Benza, other key participants in the study include AGH cardiologists Srinivas Murali, MD and Amresh Raina, MD, study coordinator Joan Rossi, senior research assistant Laurie Machen and cardiovascular researcher Subrata Chakrabarti, PhD.

According to representatives from the PHA, patients’ willingness to volunteer for research projects has already made a difference in fighting the disease. Blood samples collected at an earlier PHA conference for instance were used in work leading to the discovery of the gene for PH -- the bone morphogenetic protein type II receptor (BMPR-II).

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