Randomized, double-blind, placebo-controlled trial of rapamycin in amyotrophic lateral sclerosis.

ABSTRACT: In preclinical studies rapamycin was found to target neuroinflammation, by expanding regulatory T cells, and affecting autophagy, two pillars of amyotrophic lateral sclerosis (ALS) pathogenesis. Herein we report a multicenter, randomized, double-blind trial, in 63 ALS patients who were randomly assigned in a 1:1:1 ratio to receive rapamycin 2 mg/m²/day,1 mg/m²/day or placebo (EUDRACT 2016-002399-28; NCT03359538). The primary outcome, the number of patients exhibiting an increase >30% in regulatory T cells from baseline to treatment end, was not attained. Secondary outcomes were changes from baseline of T, B, NK cell subpopulations, inflammasome mRNA expression and activation status, S6-ribosomal protein phosphorylation, neurofilaments; clinical outcome measures of dis

SUBMITTER: Mandrioli J 

PROVIDER: S-EPMC10435464 | biostudies-literature | 2023 Aug

REPOSITORIES: biostudies-literature