Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy.

ABSTRACT:

Objective

This phase I/II study sought to explore intrathecal administration of mesenchymal stem cells (MSCs) as therapeutic approach to multiple system atrophy (MSA).

Methods

Utilizing a dose-escalation design, we delivered between 10 and 200 million adipose-derived autologous MSCs intrathecally to patients with early MSA. Patients were closely followed with clinical, laboratory, and imaging surveillance. Primary endpoints were frequency and type of adverse events; key secondary endpoint was the rate of disease progression assessed by the Unified MSA Rating Scale (UMSARS).

Results

Twenty-four patients received treatment. There were no attributable serious adverse events, and injections were generally well-tolerated. At the highest dose tier, 3 of 4 patients developed

SUBMITTER: Singer W 

PROVIDER: S-EPMC6659003 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature