NHLBI IN THE PRESS

Researchers hone in on compound that could treat pulmonary arterial hypertension in newborns, children with heart defects

Premature newborn baby

Researchers have identified a compound that could one day treat pulmonary arterial hypertension (PAH) – a rare and dangerous health condition that can occur among premature newborns and children born with heart defects.

Using stem cells with genetic signatures of actual patients with the condition, researchers screened 4,500 compounds at different stages of clinical development in eight different doses. Only six compounds improved cell survival, but a tyrosine kinase inhibitor (TKI) – a type of drug that treats certain cancers – was a lead compound for further investigation.

This TKI, called AG1296, outperformed several other similar drugs. More so, the compound reversed key biomarkers of the disease in human cells and improved vascular function when tested in mice. The study, partly-funded by NHLBI, appears in the journal Science Translational Medicine.