(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
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
"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