Safety of intrathecal autologous adipose-derived mesenchymal stromal cells in patients with ALS.

ABSTRACT:

Objective

To determine the safety of intrathecal autologous adipose-derived mesenchymal stromal cell treatment for amyotrophic lateral sclerosis (ALS).

Methods

Participants with ALS were enrolled and treated in this phase I dose-escalation safety trial, ranging from 1 × 10⁷ (single dose) to 1 × 10⁸ cells (2 monthly doses). After intrathecal treatments, participants underwent standardized follow-up, which included clinical examinations, revised ALS Functional Rating Scale (ALSFRS-R) questionnaire, blood and CSF sampling, and MRI of the neuroaxis.

Results

Twenty-seven patients with ALS were enrolled and treated in this study. The safety profile was positive, with the most common side effects reported being temporary low back and radicular leg pain

SUBMITTER: Staff NP 

PROVIDER: S-EPMC5123559 | biostudies-literature | 2016 Nov

REPOSITORIES: biostudies-literature