Abstract #1359

Hyperpolarized Helium-3 MRI Detects the Effects of a Cftr Potentiator (Ivacaftor) Therapy in Subjects with Cystic Fibrosis and the G551D Mutation

Talissa A. Altes¹, Mac Johnson², John P. Mugler¹, Deborah Froh³, Lucia Flors¹, Grady Wilson Miller¹, Jaime H. Mata¹, Carlos L. Salinas¹, Nick Tustison¹, Po-Shun Lee², Tao Song², Karl Yen², Eduard E. de Lange¹, Martyn C. Botfield²

¹Radiology, University of Virginia, Charlottesville, VA, United States; ²Vertex Pharmaceuticals Incorporated, Cambridge, MA, United States; ³Pediatric Pulmonology, University of Virginia, Charlottesville, VA, United States

This study to evaluate the ability of hyperpolarized helium-3 MR ventilation imaging to detect the effects of treatment with ivacaftor, a CFTR potentiator, demonstrated a rapid onset of improvement in lung ventilation with treatment and equally rapid return to near baseline ventilation following cessation of treatment. Eight subjects with CF were imaged on 5 different days, 2 weeks apart (Days 0, 14, 28, 42, and 56), and were treated with Ivacaftor from Days 14 to 42. Interim data analysis has been conducted on the first 4 subjects in the study, although all 8 subjects have completed the trial.