Grant: Operating Grant

Pulmonary hypertension is a deadly disease with unknown cause that kills patients within 3 years from the time of diagnosis if untreated. Available therapies improve the quality of life and alleviate some symptoms but they do not appear to prolong life and they do not reverse the disease. The disease is caused by excessive growth of cells that eventually occlude the lumen of the lung blood vessels, putting strain on the right chambers of the heart that eventually dilate, killing the patients. The lung vessels in this disease behave much like cancer also a disease of excessive cell growth. We have previously shown that an orphan drug called dichloroacetate can reverse the disease in many animal models. More recently we also published evidence that this drug may be effective in humans with one of the deadliest human cancers, brain cancer. This drug does not belong to any pharmaceutical industry and translating this knowledge into humans requires Federal funding. We proposed to conduct the first trials in humans with pulmonary hypertension focusing first on safety and tolerability of this new treatment and second on possible efficacy. If successful, these trials will be critical and the design of larger trials in the future that are required before such therapy is proven to be safe and effective for this condition. In such a case with this therapy might also be a quite cheap therapy since this drug is not owned by any for-profit pharmaceutical industry.