The Search for Disease-Modifying Therapies in Pulmonary Hypertension

J Cardiovasc Pharmacol Ther. 2019 Jul;24(4):334-354. doi: 10.1177/1074248419829172. Epub 2019 Feb 17.

Abstract

Pulmonary hypertension (PH) and its severe subtype pulmonary arterial hypertension (PAH) encompass a set of multifactorial diseases defined by sustained elevation of pulmonary arterial pressure and pulmonary vascular resistance leading to right ventricular failure and subsequent death. Pulmonary hypertension is characterized by vascular remodeling in association with smooth muscle cell proliferation of the arterioles, medial thickening, and plexiform lesion formation. Despite our recent advances in understanding its pathogenesis and related therapeutic discoveries, PH still remains a progressive disease without a cure. Nevertheless, development of drugs that specifically target molecular pathways involved in disease pathogenesis has led to improvement in life quality and clinical outcomes in patients with PAH. There are presently more than 12 Food and Drug Administration-approved vasodilator drugs in the United States for the treatment of PAH; however, mortality with contemporary therapies remains high. More recently, there have been exuberant efforts to develop new pharmacologic therapies that target the fundamental origins of PH and thus could represent disease-modifying opportunities. This review aims to summarize recent developments on key signaling pathways and molecular targets that drive PH disease progression, with emphasis on new therapeutic options under development.

Keywords: DNA damage; epigenetics; inflammation; metabolism; molecular pathology; proliferation; pulmonary artery hypertension; vascular function.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antihypertensive Agents / adverse effects
  • Antihypertensive Agents / therapeutic use*
  • Arterial Pressure / drug effects*
  • Humans
  • Molecular Targeted Therapy
  • Pulmonary Arterial Hypertension / diagnosis
  • Pulmonary Arterial Hypertension / drug therapy*
  • Pulmonary Arterial Hypertension / metabolism
  • Pulmonary Arterial Hypertension / physiopathology
  • Pulmonary Artery / drug effects*
  • Pulmonary Artery / metabolism
  • Pulmonary Artery / physiopathology
  • Signal Transduction
  • Treatment Outcome
  • Vascular Remodeling / drug effects
  • Vasodilator Agents / adverse effects
  • Vasodilator Agents / therapeutic use*

Substances

  • Antihypertensive Agents
  • Vasodilator Agents