Dr. Simon Gibbs,
The development of new drugs for pulmonary arterial hypertension (PAH) represents a particular challenge in a disease where the number of patients is few. The formula for success to get a new drug to licensing takes a good idea followed by testing in cell culture, proof of concept studies in at least two complimentary animal models, target verification in human tissue and clinical trials up to and including phase III, according to Professor Ardi Ghofrani from Giessen. Not all classes of drug have been efficacious in humans: serotonin antagonists and vasoactive intestinal polypeptide may have failed because they did not adhere to this formula for success. Professor Ralph Schermuly described how many new candidate agents are being investigated including growth factor inhibitors, micro RNAs, molecules which alter metabolism and anti-inflammatory agents which incorporate Spiegelmeyer technology. While imatanib (a tyrosine kinase inhibitor) may be an effective treatment for PAH, dasatinib causes pulmonary hypertension which is reversible after discontinuation. Current drugs for PAH reduce mortality probably by increasing cardiac output. Professor Nazzareno Galie stressed that failure of therapy in PAH can be minimized by implementing guidelines, treating early, treating to goal, implementing combination therapy and when appropriate lung transplantation. Professor Marc Humbert discussed PAH where pulmonary vascular resistance could be normalized. In particular HIV associated PAH retroviral drugs may contribute to treatment of PAH, immunosuppressive agents may be effective in SLE and mixed connective tissue disease and calcium channel blockers in vasoreactive idiopathic PAH. There are many lessons to learn from treatment successes and failures and it is clear that there are subgroups of PAH patients who require special management of their disease. The complexity of delivering best treatment is increasing.
From bench to practice: pulmonary arterial hypertension
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