NACFC 2020 | S27: Challenges of Precision Medicine in Cystic Fibrosis

The use of CFTR modulators in cystic fibrosis is emblematic of precision medicine — with specific CFTR variants being addressed by tailored small molecules. Individual patient response to modulators can be quite variable, although reasons for this heterogeneity remain unknown. Environmental factors, systemic metabolism, tissue levels of drugs, complex CFTR alleles, genetic modifiers, and other factors all may contribute to patient response in vivo.

New information in this area and model systems that predict clinical benefit are needed to optimize CF precision therapeutics. The same approaches can be used for improving access among individuals with rarest forms of the disease.