Recent Advances in Clinical Trials in Multiple System Atrophy - Archive ouverte HAL Accéder directement au contenu
Article Dans Une Revue Current Neurology and Neuroscience Reports Année : 2024

Recent Advances in Clinical Trials in Multiple System Atrophy

Résumé

PURPOSE OF REVIEW: This review summarizes previous and ongoing neuroprotection trials in multiple system atrophy (MSA), a rare and fatal neurodegenerative disease characterized by parkinsonism, cerebellar, and autonomic dysfunction. It also describes the preclinical therapeutic pipeline and provides some considerations relevant to successfully conducting clinical trials in MSA, i.e., diagnosis, endpoints, and trial design. RECENT FINDINGS: Over 30 compounds have been tested in clinical trials in MSA. While this illustrates a strong treatment pipeline, only two have reached their primary endpoint. Ongoing clinical trials primarily focus on targeting α-synuclein, the neuropathological hallmark of MSA being α-synuclein-bearing glial cytoplasmic inclusions. The mostly negative trial outcomes highlight the importance of better understanding underlying disease mechanisms and improving preclinical models. Together with efforts to refine clinical measurement tools, innovative statistical methods, and developments in biomarker research, this will enhance the design of future neuroprotection trials in MSA and the likelihood of positive outcomes.
Fichier non déposé

Dates et versions

hal-04501478 , version 1 (12-03-2024)

Identifiants

Citer

David Bendetowicz, Margherita Fabbri, Federico Sirna, Pierre-Olivier Fernagut, Alexandra Samier Foubert, et al.. Recent Advances in Clinical Trials in Multiple System Atrophy. Current Neurology and Neuroscience Reports, 2024, ⟨10.1007/s11910-024-01335-0⟩. ⟨hal-04501478⟩
0 Consultations
0 Téléchargements

Altmetric

Partager

Gmail Facebook X LinkedIn More