脳神経内科領域における開発途上の薬剤

Drugs under Development (Neurology)

従来，脳神経内科領域の疾患，特に変性疾患に対しては，低分子医薬による対症療法が中心であった。しかし近年，疾患の発症機序の根本に働きかけ病理・病態を改善させる疾患修飾薬として，特定の蛋白質，RNA，DNAに選択的に作用する抗体医薬，核酸医薬，遺伝子治療薬の開発が進行している。神経免疫疾患や機能性疾患のみならず，蛋白質の機能喪失や異常蛋白質の蓄積による神経変性疾患も根治治療が可能になることが期待される。

Abstract

Neurological diseases, especially degenerative diseases, have been mainly treated symptomatically with small molecule drugs. In recent years, however, the development of antibody therapeutics, nucleic acid therapeutics, and gene therapies that selectively act on specific proteins, RNA, and DNA has been underway to identify disease-modifying drugs that improve disease outcomes by acting on the underlying pathogenic mechanisms of diseases. It is expected to enable disease-modifying therapy not only for neuroimmunological and functional diseases, but also for neurodegenerative diseases caused by loss of protein function and accumulation of abnormal proteins.