The development of the novel therapy on autism spectrum disorder: The direct delivery of oxytocin to the brain by nasal application

2023 Fiscal Year Final Research Report

• Project/Area Number: 21K06700
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 47060:Clinical pharmacy-related
• Research Institution: Kobe Pharmaceutical University
• Principal Investigator: Sakane Toshiyasu 神戸薬科大学, 薬学部, 教授 (50215638)
• Project Period (FY): 2021-04-01 – 2024-03-31
• Keywords: オキシトシン / 自閉症スペクトラム障害 / 脳 / glymphatic system / 鼻腔内投与

Outline of Final Research Achievements

The purpose of this study is to develop a more effective treatment for autism spectrum disorders by utilizing a direct drug transport pathway from the nasal cavity to the brain to achieve efficient brain delivery of oxytocin (OXT). The absorption, brain transfer, and brain dynamics of OXT after intranasal administration, as well as the factors that affect them, were examined. It was clarified from animal studies that OXT is efficiently transferred from the nasal cavity to the brain through the direct pathway, contribution of which to the total brain uptake of OXT is high. The regulation of the Glymphatic system, the circulatory system of extracellular fluid in the brain, allows increased and sustained concentration of OXT in the brain after nasal administration, through delaying the elimination of OXT from the brain.

Free Research Field

薬物動態学

Academic Significance and Societal Importance of the Research Achievements

現在、自閉症スペクトラム障害（ASD）の治療は、付随する不安やうつなどに対する対症薬物治療が実施されているに過ぎず、社会性の欠如、コミュニケーション障害、限定された興味等の中核症状に対する治療薬は存在しない。医師主導臨床試験で、オキシトシン（OXT）の鼻腔内投与製剤が、ASDの中核症状に対する治療薬となる可能性が指摘され、ASD治療薬の開発に対する期待が高まっている。本研究成果は、医師主導臨床試験の結果をもとに、OXTの鼻腔内投与製剤によるASDの治療システムの有効性を改善し、臨床応用を実現するための基礎的情報として、きわめて重要と考えられる。