New MDS criteria時代の新しい治療

Novel Treatment of Multiple System Atrophy in the Era of the New MDS Criteria

多系統萎縮症（multiple system atrophy：MSA）の病態理解が進む中，疾患修飾療法開発に努力が注がれている。これまでにαシヌクレイン蛋白の凝集抑制，神経炎症，栄養因子低下を是正する複数の疾患修飾薬開発が試みられてきたが，ほとんどは失敗に終わっている。この度MSA診断基準が改訂され，より早期に信頼性の高い診断が可能となるとともに，新たに前駆期MSAの基準が制定され，トランスレーショナルギャップの縮小が期待されている。

Abstract

As our understanding of the pathogenesis of multiple system atrophy (MSA) continues to advance, significant research has focused on the development of disease-modifying therapies. In addition to inhibiting the aggregation of α-synuclein (αS) protein, a major culprit of MSA pathogenesis, disease-modifying drugs have been developed to mitigate cell-to-cell transmission of aggregated αS, thereby suppressing subsequent neuroinflammation, and release of trophic factors. However, most of these therapies have been unsuccessful. The diagnostic criteria for MSA have recently been revised to allow for earlier and more reliable diagnosis. In addition, clear criteria for prodromal MSA have been established, which is expected to narrow the translational gap.