Tauroursodeoxycholic acid in the treatment of patients with amyotrophic lateral sclerosis

• DOI: 10.1111/ene.12664
• Publication Date: 2015-02-09T08:09:38Z
• Authors: A. E. Elia; S. Lalli; M. R. Monsurrò; A. Sagnelli; A. C. Taiello; B. Reggiori; V. La Bella; G. Tedeschi; A. Albanese
• Keywords: Tauroursodeoxycholic acid; Tolerability; Riluzole

Background and purpose Tauroursodeoxycholic acid ( TUDCA ) is a hydrophilic bile that produced in the liver used for treatment of chronic cholestatic diseases. Experimental studies suggest may have cytoprotective anti‐apoptotic action, with potential neuroprotective activity. A proof principle approach was adopted to provide preliminary data regarding efficacy tolerability series patients amyotrophic lateral sclerosis ALS ). Methods As principle, using double‐blind placebo controlled design, 34 under riluzole who were randomized or (1 g twice daily 54 weeks) evaluated after lead‐in period 3 months. The examined every 6 weeks. primary outcome proportion responders [those subjects improvement at least 15% Amyotrophic Lateral Sclerosis Functional Rating Scale Revised ALSFRS ‐R) slope during compared phase]. Secondary outcomes included between‐treatment comparison ‐R study end, linear regression slopes ALSFFRS mean scores occurrence adverse events. Results well tolerated; there no between‐group differences higher (87%) than P = 0.021; 43%). At end baseline‐adjusted significantly 0.007) groups. Comparison analysis showed slower progression group < 0.01). Conclusions This pilot provides clinical indicating safe be effective .