Dichloroactate for Pulmonary Hypertension

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In health, blood flows freely from the heart through the lungs at low pressure. In a disease called pulmonary arterial hypertension (PAH), the blood vessels are narrow and this causes a back pressure on the heart, leading to premature death from heart failure. Idiopathic pulmonary arterial hypertension (IPAH) has no known cause and typically affects women of child bearing age. The current treatments are unsatisfactory. This project will examine an entirely new approach. It will investigate a drug called dichloroacetate (DCA) that acts by changing energy production in dividing cells. The study will seek early evidence that DCA might reduce the resistance to the passage of blood through the lungs of patients and improve heart function and include specific tests to confirm its mechanism of action. The drug is inexpensive and already available for human use. If the results of this study are encouraging, it will offer a new dimension to the management of IPAH and stimulate a further study in a larger patient population.