Rare Diseases

Biography

Biography: Suchismita Acharya

Abstract

Secondary to preterm birth, Bronchopulmonary Dysplasia (BPD) is the most common chronic respiratory disease in infants that disrupts the developmental program of the lung. BPD occurs due to an interaction between genetic and environmental factors (hyperoxia, invasive mechanical ventilation and sepsis). The pathologic hallmarks of BPD are pulmonary inflammation and increased cell death leading to impaired alveolarization and dysregulated vascularization. This can result in fibrosis, asymmetric aeration and respiratory insufficiency. Hyperoxia also induces vasoconstriction in the retina of the premature infants as an early response to oxygen leading to vasoobliteration, neovascularization and Retinopathy of Prematurity (ROP). To date, there are no effective drugs available to treat these conditions. AyuVis Research has developed nanoencapsulated formulation of novel small molecules that have demonstrated promising results in developmentally-appropriate mouse models of BPD and ROP. In BPD mice model, newborn pups (P0-saccular stage of lung development) were exposed to severe (100%) hyperoxia for 4 consecutive days (PN4-alveolar stage of lung development) followed by recovery in Room Air (RA) for next days till P14. On PN2 and PN4, the test compound was administered intranasally at a dose of 20 uM. On PN14, the pups were euthanized to collect the Bronchoalveolar Lavage (BAL), lungs, heart and serum for morphometry, flow cytometry, biochemical and physiological assays. In ROP mouse model, the PN7 pups were exposed to 75% oxygen for 10 days with or without test compounds via topical eye drops. On PN17, eyes were collected and retina was analyzed for angiogenesis markers. The small immunomodulatory compounds significantly decreased lung inflammation and lung dysfunction. Decrease in leaky blood vessels and angiogenesis was observed in treated eyes. The result demonstrated preventive and therapeutic potential of the compounds for these rare pediatric conditions.