Basic research for the development of a new treatment for myasthenia gravis using designed artificial receptors and iPS cells

2021 Fiscal Year Final Research Report

• Project/Area Number: 19K16495
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 48020:Physiology-related
• Research Institution: Osaka Medical and Pharmaceutical University
• Principal Investigator: Yamashita Manami 大阪医科薬科大学, 医学部, 助教 (80750637)
• Project Period (FY): 2019-04-01 – 2022-03-31
• Keywords: 重症筋無力症 / 神経筋接合部 / アセチルコリン受容体 / iPS細胞

Outline of Final Research Achievements

An abnormality at the neuromuscular junction, the synapse between motor nerves and muscles, causes a disease called myasthenia gravis. At the neuromuscular junction, the motor nerve releases acetylcholine, which is received by acetylcholine receptors in the muscles, which transmit information and allow normal muscle movement. In patients with myasthenia gravis, an autoimmune disease, autoantibodies attack acetylcholine receptors, resulting in impaired synaptic function, which in turn causes muscle weakness and other abnormalities. Until now, treatment and research have been conducted in the direction of suppressing immunity, but we have conducted basic research to propose a new treatment method by gene transfer of receptors that are not attacked by the immune system, based on a completely new concept.

Free Research Field

生理学

Academic Significance and Societal Importance of the Research Achievements

本研究対象である重症筋無力症は重篤な症状をきたす神経疾患であり、特に今までの治療が奏効しない、もしくは治療法の変更を必要とする患者のために、新規の治療法を開発しようとするものである。重症筋無力症は国内だけでも2万名以上の患者がいると推定されており、年々数が増えている。また、ガン治療で大きな話題となっている免疫チェックポイント阻害剤でも副作用として重症筋無力症が発生することが報告されており、その意味でも臨床的意味付けは今後増大していくものと考えられる。