Prevention of autism by drug therapy targeting mTOR system in developing brain

2016 Fiscal Year Final Research Report

• Project/Area Number: 26670491
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: The University of Tokyo
• Principal Investigator: MIZUGUCHI Masashi 東京大学, 大学院医学系研究科(医学部), 教授 (20209753)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 自閉症 / 結節性硬化症 / モデル動物 / 行動解析 / mTOR系 / 橋渡し研究

Outline of Final Research Achievements

Many patients with tuberous sclerosis complex (TSC), a hereditary disorder caused by an excessive activation of the mTOR system, have autism. As a translational research to develop drug therapy of autism, we conducted an experimental study using mouse models of TSC. When TSC mice were given an mTOR inhibitor, rapamycin, their autistic symptom, namely impairment of reciprocal social action, improved. The effect was comparable between adolescent and adult animals. On the other hand, animals who underwent chronic rapamycin treatment during the developmental period showed marked adverse effects, such as deterioration of systemic condition and atrophy of organs. The optimal dose and duration of drug therapy remained to be determined.

Free Research Field

医歯薬学