Viewpoint: activin signalling inhibitors for the treatment of pulmonary arterial hypertension

Extract Pulmonary vascular remodelling and dysfunction are hallmarks of pulmonary arterial hypertension (PAH), a rare and severe form of pre-capillary pulmonary hypertension, causing shortness of breath, fatigue, chest pain, exercise limitation, altered quality of life and markedly reduced life expectancy, despite guideline-supported management with approved medications [1–5]. In the past 30 years, several drugs with predominant vasodilator properties have been successfully developed for PAH, targeting the endothelin, nitric oxide and prostacyclin pathways (endothelin receptor antagonists, phosphodiesterase type-5 inhibitors, soluble guanylate cyclase stimulators, prostacyclin analogues and prostacyclin receptor agonists) [1, 2]. According to the guidelines, initial or sequential combination of two or three agents improves exercise capacity and delays time to clinical worsening, but such therapy does not truly qualify as disease modifying [1, 2, 6]. Therefore, it is a priority to discover novel approaches directly targeting the basic drivers of pulmonary vascular remodelling [6].
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Editorials Source Type: research