First-in-human study of inhaled Azacitidine in patients with advanced non-small cell lung cancer

•In heavily-pretreated lung cancer patients with predominantly lung involvement, inhaled Azacitidine was feasible and well-tolerated.•Disease control was observed in 3 out of 8 patients receiving inhaled Azacitidine.•Demethylating effects were noted in 2 out of 3 of patients whose bronchial epithelium was sampled pre- and post-treatment.•Inhaled Azacitidine may exemplify a new approach for managing patients with lung-confined malignant and/or premalignant conditions.