Present and future of disease-modifying therapies in multiple system atrophy

• Published in: Autonomic neuroscience 2018-05, Vol.211, p.31-38
• Main Authors: Lopez-Cuina, Miguel, Foubert-Samier, Alexandra, Tison, François, Meissner, Wassilios G.
• Format: Article
• Language: English
• Subjects: Alpha-Synuclein; alpha-Synuclein - metabolism; Clinical trials; Clinical Trials as Topic; Disease modifying therapies; Disease Notification - methods; Humans; Multiple System Atrophy - diagnosis; Multiple System Atrophy - therapy; Translational Medical Research; Treatment; Treatment Outcome
• ISSN: 1566-0702; 1872-7484
• DOI: 10.1016/j.autneu.2017.12.008

Through the last decade seven clinical trials on Multiple System Atrophy have been published, virtually all of them reported negative results. Patients and family remain hopeful while facing this devastating disease, but as doctors we still cannot offer them disease-modifying therapies. The field has seen many advances regarding pathophysiology, translational research, diagnostic accuracy, natural history and imaging, but successful treatment remains elusive. This review provides an overview of the available tools for designing clinical trials, critically analyzes the past studies and describes the knowledge obtained from them, and finally gives some orientation for future trials that could meet the current needs of patients and clinicians, overcoming the hurdles met by previous studies. •All but one disease-modifying clinical trials have reported negative results.•The field is ready for conducting large randomized clinical trials.•Neuroimaging holds clear promise for the development of objective outcome measures.•At least five molecules in the pipeline have shown promise for future trials.