Elia AE, Lalli, S, Monsurro MR, et al. Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis. Eur J Neurol 2016;23:45-52.
Submitted by Kathya Ramos, MD, News Science Editorial Board
Tauroursodeoxycholic acid (TUDCA) is a hepatically-synthesized hydrophilic bile acid used for treatment of chronic cholestatic liver diseases. Experimental studies suggest that TUDCA may have cytoprotective and anti-apoptotic effects, with potential neuroprotective activity.
In this pilot study, 34 ALS patients were randomized to receive either TUDCA (1 g twice daily for 54 weeks) or placebo after a lead-in period of 3 months. The primary outcome measure was the proportion of patients with with improvement of at least 15% in the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) slope during the treatment period as compared to the lead-in phase. Secondary outcomes included between-treatment comparison of ALSFRS-R at study end, comparison of the linear regression slopes for ALSFFRS-R mean scores, and the occurrence of adverse events.
The treatment group showed a higher proportion of responders compared to those who received placebo (87% vs. 43%, p = 0.021). At study end baseline-adjusted ALSFRS-R was signiﬁcantly higher (P = 0.007) in TUDCA than in placebo groups. Comparison of the slopes of regression analysis showed slower progression in the TUDCA than in the placebo group (P < 0.01). TUDCA was well tolerated; there were no between group diﬀerences in adverse events.
This pilot study provides preliminary clinical data indicating that Tauroursodeoxycholic acid (TUDCA) is safe and may be eﬀective in slowing the progression of ALS.
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