Lab-Grown cells may unlock lifesaving CF drug for thousands

This study tested whether a new lab method could predict if people with rare cystic fibrosis mutations would benefit from the drug Trikafta. Researchers gave Trikafta to 42 patients with specific rare mutations not normally approved for the drug. They collected skin or blood samples to grow airway cells in the lab, then checked if those lab cells' response to Trikafta matched the patients' actual lung improvement. The goal was to create a tool to help identify rare mutation patients who could benefit from existing treatments.