Gene Therapy May Treat Pulmonary Hypertension

Gene Therapy May Treat Pulmonary Hypertension

Reported May 20, 2010

(Ivanhoe Newswire) — Gene therapy has been shown to have positive effects in rat models of pulmonary arterial hypertension (PAH), a life-threatening disease in which pressure in the blood vessels of the lungs increases, causing a back-pressure strain on the heart.


In inherited forms of the disease, PAH is caused by a mutation in a receptor called bone morphogenetic protein receptor, type II (BMPR2). Even in some non-inherited forms of the disease, BMPR2 levels are low, according to researchers at the University of Adelaide in Australia, who conducted the study.


There have been many improvements in PAH treatment in recent years, but many patients still ultimately die of the disease, thus new treatments are still needed.



“We investigated whether increasing BMPR2 levels might improve pulmonary hypertension,” principal investigator Paul Reynolds, M.D., Ph.D, FRACP, was quoted as saying. “We used two rat models of pulmonary hypertension, and increased BMPR2 levels in the lungs using a gene therapy approach. We found in both models that BMPR2 gene delivery significantly reduced pulmonary hypertension and the strain it causes on the heart.”


The researchers found that the rats that were treated with BMPR2 compared to placebo had significantly reduced right ventricular hypertrophy, reduced pulmonary vascular resistance, and improved cardiac output.


“These findings were based on the knowledge that low BMPR2 levels are associated with pulmonary hypertension, but it has not previously been shown that increasing BMPR2 levels might be used as a therapy,” said Dr. Reynolds. “This research strongly suggests that increasing BMPR2 levels might offer a new therapeutic target in pulmonary hypertension.”


SOURCE: Presented at the American Thoracic Society (ATS) 2010 International Conference, New Orleans, May 19, 2010