(F1293) HUMAN UMBILICAL CORD-DERIVED MESENCHYMAL STEM CELL THERAPY FOR SEVERE BRONCHOPULMONARY DYSPLASIA REFRACTORY TO CONVENTIONAL THERAPY: A PHASE I CLINICAL TRIAL

Abstract: Bronchopulmonary dysplasia (BPD) is a common and serious complication affecting very low birth weight preterm infants, characterized by impaired lung alveolarization and dysregulated vascularization. Infants with moderate-to-severe BPD face a significant risk of long-term respiratory and developmental disabilities. To date, there are no effective pharmacological treatments for BPD. Mesenchymal stem cells have been considered as a promising therapeutic option, offering potential to improve lung structure and function in BPD patients. The aim of this study is to explore a gap in BPD treatment by evaluating the safety and efficacy of intratracheal administration of human umbilical cord-derived mesenchymal stem cells (hUC-MSCs) in preterm infants with severe BPD. This prospective trial was conducted on preterm infants, born at 23 to 29 weeks of gestation with severe BPD, diagnosed at 36 weeks postmenstrual age. These infants remained difficult to wean from invasive mechanical ventilation, despite having received conventional therapy, including surfactants and steroid. Each infant received a single intratracheal dose of hUC-MSCs (1×10⁷ cells/kg). To date, three patients have been successfully enrolled in the study. No severe adverse events related to the intervention were observed at the time of this report. Following transplantation of MSCs, there was a progressive reduction in ventilator parameters, including fraction of inspiration O2 (FiO2) and mean airway pressure. All three patients were successfully extubated at a range of 10-19 days after MSCs transplantation, with no re-intubation required prior to discharge. The chest radiographs of the three patients showed a mitigation of atelectasis, diffuse haziness and reticular opacities in lungs, 2 months after MSCs transplantation, suggesting signs of lung recovery. In summary, intratracheal administration of allogeneic hUC-MSCs appears to be a safe and feasible therapy for preterm infants with severe BPD.

Funding Source: This work was supported by the Pediatrics development Fund of School of medicine from Zhejiang University Education Foundation.

Clinical Trial ID number: ClinicalTrials.gov ID: NCT06788470