Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis

Study answer

What this study found

TUDCA was well tolerated and was associated with signals of slower functional decline versus placebo. The primary responder outcome favored TUDCA: 13 (87%) versus 6 (43%) patients, P = 0.021, and baseline-adjusted ALSFRS-R at study end was higher with TUDCA (23.3 [19.9-26.6] vs 16.3 [12.9-19.7]; P = 0.007). The ALS-FRS-R slope during treatment was less steep with TUDCA (-0.262 vs -0.388; P < 0.01), and the bulbar subscore also favored TUDCA at end of study (3.8 [2.2-5.4] vs 1.9 [0.8-2.9]; P = 0.037). Study-related adverse events were reported in 2 events (13.3%) with TUDCA and 3 events…

Study & population

Multicenter, double-blind, placebo-controlled trial at three Italian centers in adults aged 18-75 years with spinal-onset, clinically probable or definite ALS by revised El Escorial criteria.

Intervention

Tauroursodeoxycholic acid (TUDCA) dihydrate was given orally at 1 g twice daily for 54 weeks, added to background riluzole.

Key limitation

This was a small pilot study with only 34 randomized patients and fewer analyzed for safety, which limits precision and generalizability.

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Original abstract