New Trial Designs and Potential Therapies for Pulmonary Artery Hypertension

• Published in: Journal of the American College of Cardiology Vol. 62; no. 25; pp. D82 - D91
• Main Authors: Gomberg-Maitland, Mardi, MD, MSc, Bull, Todd M., MD, Saggar, Rajeev, MD, Barst, Robyn J., MD, Elgazayerly, Amany, MD, PhD, Fleming, Thomas R., PhD, Grimminger, Friedrich, MD, PhD, Rainisio, Maurizio, PhD, Stewart, Duncan J., MD, Stockbridge, Norman, MD, PhD, Ventura, Carlo, MD, PhD, Ghofrani, Ardeschir H., MD, Rubin, Lewis J., MD
• Format: Journal Article Conference Proceeding
• Language: English
• Published: New York, NY Elsevier Inc 24-12-2013; Elsevier
• Subjects: Animals; Biological and medical sciences; Cardiology. Vascular system; Cardiovascular; Clinical Trials as Topic - methods; Clinical Trials as Topic - trends; Drug Discovery - trends; ethics; Familial Primary Pulmonary Hypertension; Heart; Humans; Hypertension, Pulmonary - diagnosis; Hypertension, Pulmonary - therapy; Internal Medicine; Medical sciences; Pneumology; pulmonary arterial hypertension; Pulmonary hypertension. Acute cor pulmonale. Pulmonary embolism. Pulmonary vascular diseases; Research Design - trends; therapeutics; trial designs; NO; therapeutics; eNOS; RV; trial designs; pulmonary hypertension; RAAS; endothelial progenitor cell; platelet-derived growth factor; LV; ethics; PRO; PVR; time to clinical worsening; renin-angiotensin-aldosterone system; right ventricle/ventricular; TTCW; patient-reported outcome; endothelial nitric oxide synthase; extracorporeal membrane oxygenation; left ventricle/ventricular; MSC; pulmonary arterial hypertension; ECMO; mesenchymal stem cell; nitric oxide; fatty acid oxygenation; 6-min walk distance; VEGF; FAO; PAH; PDGF; vascular endothelial growth factor; PH; EPC; pulmonary vascular resistance; 6MWD; Respiratory disease; Cardiovascular disease; Potential; Pulmonary artery; Pulmonary hypertension; Design; Goal; Treatment; Pulmonary vessel; Blood vessel; Clinical trial; Circulatory system; Cardiology
• ISSN: 0735-1097; 1558-3597
• DOI: 10.1016/j.jacc.2013.10.026

A greater understanding of the epidemiology, pathogenesis, and pathophysiology of pulmonary artery hypertension (PAH) has led to significant advances, but the disease remains fatal. Treatment options are neither universally available nor always effective, underscoring the need for development of novel therapies and therapeutic strategies. Clinical trials to date have provided evidence of efficacy, but were limited in evaluating the scope and duration of treatment effects. Numerous potential targets in varied stages of drug development exist, in addition to novel uses of familiar therapies. The pursuit of gene and cell-based therapy continues, and device use to help acute deterioration and chronic management is emerging. This rapid surge of drug development has led to multicenter pivotal clinical trials and has resulted in novel ethical and global clinical trial concerns. This paper will provide an overview of the opportunities and challenges that await the development of novel treatments for PAH.