2019 Volume 36 Issue 4 Pages 354-358
Multiple system atrophy (MSA) is a refractory neurodegenerative disease and at present no fundamental therapeutic method has been found, but due to the progress of research in recent years, development of new therapeutic regimens is expected in the future. Treatment methods that can be selected in Japan as of 2018 are symptomatic treatments such as TRH analogs, drugs for treating Parkinson's disease, vasopressors, drugs for dysuria disorder etc. Non–medication therapy includes treatment such as rehabilitation, non–invasive ventilation therapy, tracheostomy, ventilation, and gastrostomy. Clinical trials targeting humans have failed to demonstrate the inhibitory effect on progression, but as a future treatment, COQ10 treatment, mesenchymal stem cell transplantation, treatment targeting α–synuclein, etc. are expected. In future clinical trials, it is important to set up an appropriate clinical trial plan with the precise pathological condition modifying mechanism as the background, and appropriate clinical study plan such as severity, evaluation index, biomarker, treatment start time, drug dose setting etc.
