Autologous transplantation of airway basal stem cells in COPD patients: A randomized, single-blind, placebo-controlled phase 2 trial

Chronic obstructive pulmonary disease (COPD), a leading global cause of mortality, lacks therapies targeting irreversible lung damage. Airway basal progenitor cells (BCs) exhibit lung repair potential in preclinical and early clinical studies. This multicenter, randomized, single-blind, placebo-controlled phase 2 trial in China evaluated autologous BC transplantation in COPD patients with impaired gas transfer. BCs were isolated from healthy lung tissue via bronchoscopy, expanded under GMP conditions, and transplanted (2.5×10⁶ cells/kg) via bronchoscopy. Fifty patients (25 intervention, 25 placebo) were assessed over 24 weeks. Adverse event rates were comparable between groups. The intervention group showed improved lung gas transfer (DLCO: +0.117 vs. −0.159 mmol/min/kPa, p=0.0356) and reduced COPD symptoms (CAT score: −2.33 vs. +0.84, p=0.0397) versus controls. Exercise capacity (6MWD) increased by +46 meters in the intervention group. Autologous BC transplantation is safe and may offer therapeutic benefit for COPD patients with gas transfer deficiency. Approved by China National Medical Product Administration ( NCT05638776 ).