Molecular Rescue in Pulmonary Arterial Hypertension

In this issue of the Journal, Humbert et al.1 report on a new approach to the treatment of pulmonary arterial hypertension, particularly the idiopathic and heritable forms of the disease. The treatment, which is given subcutaneously every 3 weeks, is a fusion protein that impairs activation of one limb of the proproliferative transforming growth factor β (TGF-β) pathways. Why would inactivation of a TGF-β pathway be an effective approach? The answer is in the genes. Approximately 70 to 80% of cases of heritable pulmonary arterial hypertension and roughly 20 to 30% of cases of idiopathic pulmonary arterial hypertension are consequences . . .