University of Pittsburgh, Monash University, University of Florida, and University of Missouri–Kansas City researchers have identified a novel treatment approach for infants with moderate-to-severe bronchopulmonary dysplasia (BPD). Using an oral suspension of vamorolone (VAM), an FDA approved corticosteroid, it may be possible to treat BPD in infants without the adverse effects associated with current BPD treatments. Successful development and clinical application of this approach could have profound impacts on pediatric BPD patients, reducing the risk of adverse effects on physical growth or metabolic functions associated with existing BPD treatment.
VAM, a synthetic corticosteroid, has recently been approved by the FDA and European Medicines Agency for the treatment of children with Duchenne muscular dystrophy (DMD). Initial animal studies suggest VAM could treat moderate-to-severe BPD in infants in both the short- and long- term without the risk of adverse developmental effects associated with current BPD treatment.
Description
BPD is a form of chronic lung disease (CLD) and the one most found in premature babies. Prematurity, birth before 30 weeks gestation, considerably increases the risk of lung inflammation and injury from hyperoxia, infection, and manual ventilation resulting in alveolar remodeling which is characteristic of BPD and long-term lung dysfunction frequently observed in infants who survive BPD. Current treatment strategies involve glucocorticoids (GC) (e.g., prednisolone and dexamethasone). Long-term treatment (>30 days in the early weeks of life) is associated with adverse events including hyperglycemia, cerebral palsy, and impaired brain and linear growth. There remains a pressing clinical need to develop safer treatments for premature infants with BPD with less risk of adverse events. VAM could reduce or eliminate BPD pathologies without impacting on growth and development in these vulnerable patients.
Applications
• Bronchopulmonary dysplasia in infants and neonates
Advantages
Current GC treatment strategies for BPD in neonates and infants is associated with various adverse outcomes including reduced whole body and brain growth and white matter loss. VAM is a potent anti-inflammatory agent recently approved for the treatment of DMD in children over 2 years of age. Long term use in DMD patients has found a superior safety profile compared with prednisone, with improved linear growth. This novel treatment approach aims to harness the anti-inflammatory actions of VAM and improved safety profile to reduce the risk of adverse long-term effects associated with current BPD GC treatment.
Invention Readiness
In neonatal rats, treatment with VAM was compared to conventional BPD treatment. VAM did not impact on weight or blood glucose levels (unlike conventional treatments). Gene expression studies also reduce basal expression of key proinflammatory cytokines suggesting VAM can produce anti-inflammatory responses in neonatal lungs.
IP Status
Patent Pending
