Abstract
Neuropeptides such as oxytocin (OT) have shown promise as treatments for many central nervous system (CNS) disorders, such as Dravet Syndrome and Autism Spectrum Disorder (ASD). The primary barriers to the use of OT as a treatment for ASD are that, due to its size and hydrophicity, it does not cross the blood brain barrier (BBB) and penetrate the CNS, and that it is rapidly metabolized in the blood stream, with a half‐life of 1–6 minutes following IV administration. Ongoing studies are attempting to overcome its poor brain penetrance using intranasal delivery of OT. Although the development of intranasal delivery of OT has been a seminal advance in ASD research, this administration method is an inefficient means of transferring drug to the brain and requires frequent dosing (because of the short half‐life of OT). As such, there is legitimate concern regarding the long‐term effects of high‐dose and frequent daily administration of intranasal OT. We have collected compelling data showing that a nanotechnology is a promising means of augments the brain penetrance of OT and protecting OT from metabolism and sustaining its delivery. We have found that our nanoscale drug carriers do not exhibit in vitro or in vivo toxicity, have sizes on the order of 100–200nm (which is highly amenable to brain delivery), increase the brain penetrance of an IR‐marker (of similar molecular size to OT) using bioimaging, engender stronger prosocial effects than OT alone given intranasally, and exhibit sustained release effects both in vitro and in vivo. Although we have focused on the development of intranasal OT, we believe that this new technology will be applicable to many other neuropeptides (such as neuropeptide Y) that may have promise for ASD and other disorders because of their anti‐inflammatory, neuroprotective, and/or anti‐seizure effects.Support or Funding InformationThis work was supported by R21NS100512.This abstract is from the Experimental Biology 2018 Meeting. There is no full text article associated with this abstract published in The FASEB Journal.
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