2022 AMI Online Salon

Ivacaftor for Cystic Fibrosis

Project Details

  • Entrant Name:  Mimi (Yuejun) Guo
  • Client: Faculty advisor:  Derek Ng
  • Copyright:  Mimi (Yuejun) Guo, 2021
  • Medium/software used: Maya, VMD, Photoshop, and Illustrator
  • Final presentation format: A two-page spread in a popular science magazine
  • Primary Audience: Educated lay audience

Project Description

Ivacaftor is the very first drug to directly treat the cause of cystic fibrosis, and it was granted the first Breakthrough Therapy designation by the FDA. With clinically proven safety and efficacy, it was approved in 2012, but it was not until 2019 that scientists finally knew the possible molecular basis of how it works. Using cryo-EM techniques, the Chen Lab published the structural data on Ivacafor interacting with the cystic fibrosis transmembrane conductance regulator (CFTR), the protein drug target that is misfolded in diseased individuals. These findings may serve as a roadmap for the future development of medications to treat other diseases caused by misfolded proteins. This two-page illustration spread explains how Ivacaftor potentiates the open state of CFTR through its interaction with three amino acid residues in the CFTR transmembrane helices. To provide further context and a cohesive story, this illustration also gives a brief overview of cystic fibrosis, comparing the physiological function of normal versus defective CFTR, to facilitate the molecular understanding of how Ivacaftor restores CFTR function.