Platelet-derived growth factor inhibition in idiopathic and familial pulmonary arterial hypertension

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Pulmonary arterial hypertension is an unusual but devastating condition. It typically affects young women who present with increasing breathlessness on exertion and leads to death within a few years from diagnosis. Current therapies can give some improvement in symptoms but most patients eventually die of the disease. Recent advances in our understanding of the genetic and molecular basis of pulmonary arterial hypertension have identified some key growth factor pathways which are abnormal in the lung blood vessels of these patients. Amongst these, the platelet-derived growth factor (PDGF) pathway shows promise as a new target for treatment. Orally available and well tolerated drugs already exist which target this pathway in other diseases. We propose to study the therapeutic potential of a drug which inhibits the PDGF pathway in patients with severe pulmonary hypertension. In addition, we will undertake studies in cells derived from these patients to increase our understanding of how this drug works in pulmonary hypertension.