神経領域の薬剤の現状を俯瞰する—真のDecade of Brainの到来か

Recent Advances in Novel Therapies for Neurological Diseases: An Overview and Future Scope

近年，多くの神経疾患に対して疾患修飾薬が開発された。その基盤技術は核酸医薬や抗体医薬，さらに酵素補充療法といった新規技術を用いたものから，従来薬の投与方法の工夫，さらに基礎研究によるプルーフオブコンセプト（POC）からドラッグリポジショニングを経て有効性が示されたものまで，多種多様な創薬戦略が結実している。「治る脳神経内科」に向けた最新の神経疾患治療薬を概観する。

Abstract

Disease-modifying therapies remain an important unmet medical need in many neurological diseases. However, recent advances in novel therapies, such as antisense oligonucleotides, antibodies, and enzyme supplementation have significantly improved the prognosis and delayed time until relapse of various neurological diseases. Nusinersen used for spinal muscular atrophy and patisiran for transthyretin-mediated familial amyloid polyneuropathy significantly suppress disease progression and prolong longevity. Antibodies against the CD antigen, interleukin, or complement significantly lessen the time until relapse of multiple sclerosis or neuromyelitis optica. Administration of antibodies has expanded for treatment of migraine and neurodegenerative diseases such as Alzheimer's disease. Therefore, a paradigm shift is being observed in therapeutic strategies for many neurological diseases, many of which are typically considered “intractable.”