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Developing treatments for pulmonary arterial hypertension.

ABSTRACT: Developing new treatments for pulmonary arterial hypertension (PAH) is a challenge. We have enjoyed success with regulatory approvals for three drug classes-prostanoids, endothelin receptor antagonists and phosphodiesterase type 5 inhibitors. But we have also seen some disappointing results, for example, from studies with vasoactive intestinal polypeptide, statins and tergolide. Animal models are an unreliable predictor of efficacy in humans. The best model for the disease is the patient. This review discusses three major issues facing the evaluation of drugs in PAH patients-target validation, choosing the right dose, and early trial design.

SUBMITTER: Wilkins MR

PROVIDER: S-EPMC3641724 | biostudies-literature | 2013 Jan

REPOSITORIES: biostudies-literature

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Developing treatments for pulmonary arterial hypertension.

Wilkins Martin R MR

Pulmonary circulation 20130101 1

Developing new treatments for pulmonary arterial hypertension (PAH) is a challenge. We have enjoyed success with regulatory approvals for three drug classes-prostanoids, endothelin receptor antagonists and phosphodiesterase type 5 inhibitors. But we have also seen some disappointing results, for example, from studies with vasoactive intestinal polypeptide, statins and tergolide. Animal models are an unreliable predictor of efficacy in humans. The best model for the disease is the patient. This r ...[more]

PMID: 23662193

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