Autologous transplantation of P63+ lung progenitor cells in patients with bronchiectasis: a randomized, single-blind, controlled trial

<bold>Background:</bold> Non-cystic fibrosis bronchiectasis is a severe respiratory disease characterized by progressive loss of lung function, which can hardly be rescued by conventional therapy and encouraged the development of stem/progenitor cell-based therapy. P63+ airway basal progenitor cells have been identified as a promising candidate for lung epithelium repair, providing new therapeutic option for bronchiectasis patients. <bold>Aim:</bold> To evaluate the safety and efficacy of an autologous P63+ basal progenitor cell product (REGEND001) in bronchiectasis patients, a randomized, single-blind, controlled, phase 1/2 trial was conducted. <bold>Methods:</bold> Autologous P63+ progenitor cells were cloned and expanded from the airway brushing tissues of patients. Totally 37 eligible patients were enrolled and randomly assigned to receive either bronchoscopic airway clearance (B-ACT) treatment only (n = 19) or B-ACT plus autologous P63+ progenitor cells transplantation at the dose of 1~3 × 10⁶ cells per kilogram body weight (n = 18). <bold>Results:</bold> Results showed that compared to the control group, the change in DLCO levels from baseline to 24 weeks post therapy was significantly higher in the cell treatment group (P-value = 0.039). Furthermore, the patients in cell treatment group also demonstrated significantly reduced lung damaged volume, improved SGRQ score, and ameliorated BSI and FACED scores within 4~12 weeks post cell transplantation. <bold>Conclusions:</bold> In patients with bronchiectasis, transplantation of REGEND001 was associated with increased lung gas exchange function, reduction of bronchiectasis severity scores and improved quality of life.