The rare lung disease is so complicated that its acronym is difficult to pronounce. However, for infants who were unfortunate enough to be born with this disorder, the outcome is generally fatal.

Nanoparticle-Based Therapy Could Treat Fatal Lung Disease in Newborns.

Left: normal mouse lung tissue showing a healthy density of blood vessels. Middle: example of abnormal, diseased tissue with reduced STAT3 activation. Right: tissue from a treated mouse. Green depicts blood vessels; Red shows activated STAT3. Image Credit: Cincinnati Children’s.

The disease is known as alveolar capillary dysplasia with misalignment of the pulmonary veins, or ACDMPV for short. According to a study, the disease is associated with mutations in the FOXF1 gene. Medical experts throughout the world have recorded around 200 cases, but an unestimated number of infants could have died without the disorder ever being diagnosed, stated the National Organization for Rare Disorders.

The disease is induced by genetic changes that inhibit the formation of proper blood vessels in the lungs. Within a few days or weeks post birth, infants turn blue because of the absence of oxygen while blood pressure increases inside their lungs. The few infants who get to live do so by receiving exceptionally rare infant-sized lung transplants.

Now, a new study headed by experts from Cincinnati Children’s and the University of Cincinnati has reported about helping mice (with a FOXF1 mutation that is identical to human ACDMPV patients) live longer with this fatal disease. This was done by applying high-tech nanoparticles to send a STAT3 gene into the lungs to trigger the growth of blood vessels.

STAT3 is a crucial downstream target of the FOXF1 gene and its delivery can rectify the vascular deficiency in ACDMPV mice. The study results were published online in the Circulation journal on June 11th, 2021.

If these findings can be matched in human analyses in the future, this success can potentially boost the speed of development for other nanoparticle-based treatments for many medical conditions, stated the study co-authors….