One of the dangerous health conditions that can occur among premature newborns, children born with heart defects, and some others is pulmonary arterial hypertension (PAH).
Commonly mistaken for asthma, this condition occurs when blood vessels in the lungs develop excessive resistance to blood flow. This forces the heart's right ventricle to work harder, causing it to enlarge, thicken and further elevate blood pressure. While early treatment usually succeeds, the condition can become persistent and progressive, which can lead to heart failure and death.
The exact incidence and prevalence of PAH remains unclear, but reviews of patient registries in Europe have estimated that the condition occurs in nearly 64 of every 1 million children, including transient cases. So far, there is no cure.
Now a team of scientists at Stanford University and Cincinnati Children's report finding a potentially effective needle in a haystack.
"In this study, we screened a library of 4500 compounds at different stages of clinical development in eight different doses. Of the six compounds that improved cell survival, we identified the tyrosine kinase inhibitor AG1296 as the lead compound for further investigation," says Mingxia Gu, MD, PhD, lead author of a study published May 5, 2021, in Science Translational Medicine.