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Targeting Trafficking in Drug Development pp 385–425Cite as

Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator and Drugs: Insights from Cellular Trafficking

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Part of the book series: Handbook of Experimental Pharmacology ((HEP,volume 245))

Abstract

The eukaryotic cell is organized into membrane-delineated compartments that are characterized by specific cadres of proteins sustaining biochemically distinct cellular processes. The appropriate subcellular localization of proteins is key to proper organelle function and provides a physiological context for cellular processes. Disruption of normal trafficking pathways for proteins is seen in several genetic diseases, where a protein’s absence for a specific subcellular compartment leads to organelle disruption, and in the context of an individual, a disruption of normal physiology. Importantly, several drug therapies can also alter protein trafficking, causing unwanted side effects. Thus, a deeper understanding of trafficking pathways needs to be appreciated as novel therapeutic modalities are proposed. Despite the promising efficacy of novel therapeutic agents, the intracellular bioavailability of these compounds has proved to be a potential barrier, leading to failures in treatments for various diseases and disorders. While endocytosis of drug moieties provides an efficient means of getting material into cells, the subsequent release and endosomal escape of materials into the cytosol where they need to act has been a barrier. An understanding of cellular protein/lipid trafficking pathways has opened up strategies for increasing drug bioavailability. Approaches to enhance endosomal exit have greatly increased the cytosolic bioavailability of drugs and will provide a means of investigating previous drugs that may have been shelved due to their low cytosolic concentration.

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  1. Department of Physiology and Biophysics, Chicago Medical School, North Chicago, IL, USA

    Robert J. Bridges & Neil A. Bradbury

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  1. Robert J. Bridges
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  2. Neil A. Bradbury
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Correspondence to Neil A. Bradbury .

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  1. Red de Apoyo a la Investigación, National Autonomous University of Mexico and Instituto Nacional de Ciencias, Médicas y Nutrición SZ, Mexico City, Mexico

    Alfredo Ulloa-Aguirre

  2. Dep of Anatomy, Physiology and Pharmacology, Auburn University, College of Veterinary Medicine, Auburn, Alabama, USA

    Ya-Xiong Tao

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Bridges, R.J., Bradbury, N.A. (2018). Cystic Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator and Drugs: Insights from Cellular Trafficking. In: Ulloa-Aguirre, A., Tao, YX. (eds) Targeting Trafficking in Drug Development. Handbook of Experimental Pharmacology, vol 245. Springer, Cham. https://doi.org/10.1007/164_2018_103

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