Fabrication of enzyme reaction fields in the brain for treatment of central nerve system disease

2021 Fiscal Year Final Research Report

• Project/Area Number: 19K22953
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Review Section: Medium-sized Section 90:Biomedical engineering and related fields
• Research Institution: The University of Tokyo
• Principal Investigator: Anraku Yasutaka 東京大学, 大学院工学系研究科(工学部), 特任准教授 (60581585)
• Project Period (FY): 2019-06-28 – 2022-03-31
• Keywords: 高分子 / DDS / 脳 / 酵素補充療法

Outline of Final Research Achievements

Enzyme Replacement Therapy (ERT) is a treatment that aims to improve symptoms by replenishing the body's lacking enzymes. Drug delivery systems (DDS), which incorporate a system that delivers enzymes to the disease site and promotes metabolism, are expected to be an innovative therapy with low side effects. The objective of this project was to develop innovative therapeutic technologies for central nervous system (CNS) diseases by creating a "reaction field of enzymes in the brain. We will demonstrate these proposals by encapsulating the enzyme in DDS, equipped with optimal functions as a reaction field for the enzyme in vivo, and installing a ligand molecule for passing through the BBB on the surface layer. Through the synthesis of polymers to form DDS, construction of DDS encapsulating enzymes and evaluation of their fundamental physical properties, and in vitro and in vivo tests, it was demonstrated that the DDS constructed in this project could work as ERT in the brain.

Free Research Field

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Academic Significance and Societal Importance of the Research Achievements

本研究で得られた成果は、有効な治療法が未確立である中枢神経系(CNS)疾患に対して、薬剤送達に基づく分子治療という抜本的解決策を提供するものであり極めて大きな意義を有している。様々な機能分子を中枢系に送達する方法論確立は、CNS疾患に留まらず、脳腫瘍やアミノ酸代謝異常など広範な疾患治療に大きく貢献することが確信される。また高分子/材料設計の観点からは、生体適合性・標的指向性・環境応答性という異なる機能を空間的に制御された形で構造内部に配置する仕掛けを創り込むなど、独創性に秀でた生体材料設計プロセスを当該分野にもたらす意義を有していると確信する。