Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. RCT (n=137) found slower functional decline than placebo as measured by ALSFRS-R score over 24 weeks (mean rate of change −1.24 vs. −1.66 points/month, respectively; p=0.03).

SPS commentary:

An editorial discusses how patients with ALS and their treating physicians should interpret these data, as the observed therapeutic effect is modest, and although formal comparisons with previous studies alone are difficult, it is unclear whether either taurursodiol alone or sodium phenylbutyrate alone might yield similar benefits or whether the doses were the most effective. It notes one ongoing phase 3 trial of tauroursodeoxycholic acid alone, with results expected in mid-2021, may provide partial answers. It highlights that ~ 19% of the patients in the active-drug group discontinued the intervention due to adverse events, and in light of the residual questions about efficacy and the ability of patients to continue taking the drug, it agrees with the authors’ conclusion on the need for longer and larger trials,


New England Journal of Medicine

Resource links: