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Delivering drugs to the central nervous system: an overview

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Abstract

Developing therapies for the brain is perhaps the greatest challenge facing modern medicine today. While a great many potential therapies show promise in animal models, precious few make it to approval or are even studied in human patients. The particular challenges to the translation of neurotherapeutics to the clinic are many, but a major barrier is difficulty in delivering therapeutics into the brain. The goal of this workshop was to present ways to deliver therapeutics to the brain, including the limitations of each method, and describe ways to track their delivery, safety, and efficacy. Solving the problem of delivery will aid translation of therapeutics for patients suffering from neurodegeneration and other disorders of the brain.

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Acknowledgments

The author gives thanks to Pericles Calias, Ph.D., for reading the manuscript.

Conflict of interest

Patricia Dickson receives research support from BioMarin and Genyzme, LLC, which manufacture and distribute enzyme replacement therapy for lysosomal storage disorders.

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  1. Division of Medical Genetics, Department of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, 1124 W. Carson Street, HH1, Torrance, CA, 90502, USA

    Patricia I. Dickson

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Dickson, P.I. Delivering drugs to the central nervous system: an overview. Drug Deliv. and Transl. Res. 2, 145–151 (2012). https://doi.org/10.1007/s13346-012-0068-0

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