Repeated Intrathecal Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis.

ABSTRACT:

Objective

To assess the safety and efficacy of 2 repeated intrathecal injections of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs) in amyotrophic lateral sclerosis (ALS).

Methods

In a phase 2 randomized controlled trial (NCT01363401), 64 participants with ALS were randomly assigned treatments (1:1) of riluzole alone (control group, n = 31) or combined with 2 BM-MSC injections (MSC group, n = 33). Safety was assessed based on the occurrence of adverse events. The primary efficacy outcome was changes in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score from baseline to 4 and 6 months postinjection. Post hoc analysis includes investigation of cerebrospinal fluid biomarkers and long-term survival analysis.

Results

Safety ratin

SUBMITTER: Oh KW 

PROVIDER: S-EPMC6175096 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature