Nanofiber-based delivery of therapeutic peptides to the brain

Mazza, Mariarosa and Notman, Rebecca and Anwar, Jamshed and Rodger, Alison and Hicks, Matthew and Parkinson, Gary and McCarthy, Dave and Daviter, Tina and Moger, Julian and Garrett, Natalie and Mead, Tania and Briggs, Michael and Schätzlein, Andreas G. and Uchegbu, Ijeoma F. (2013) Nanofiber-based delivery of therapeutic peptides to the brain. ACS Nano, 7 (2). pp. 1016-26.

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The delivery of therapeutic peptides and proteins to the central nervous system is the biggest challenge when developing effective neuropharmaceuticals. The central issue is that the blood-brain barrier is impermeable to most molecules. Here we demonstrate the concept of employing an amphiphilic derivative of a peptide to deliver the peptide into the brain. The key to success is that the amphiphilic peptide should by design self-assemble into nanofibers wherein the active peptide epitope is tightly wrapped around the nanofiber core. The nanofiber form appears to protect the amphiphilic peptide from degradation while in the plasma, and the amphiphilic nature of the peptide promotes its transport across the blood-brain barrier. Therapeutic brain levels of the amphiphilic peptide are achieved with this strategy, compared with the absence of detectable peptide in the brain and the consequent lack of a therapeutic response when the underivatized peptide is administered.

Item Type:
Journal Article
Journal or Publication Title:
ACS Nano
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Deposited On:
21 Mar 2013 13:20
Last Modified:
17 Sep 2023 01:19