Investigational Drug May Prolong Survival in Patients With ALS

TUESDAY, Oct. 20, 2020 — Long-term survival analysis shows that patients with earlier initiation of sodium phenylbutyrate-taurursodiol (PB-TURSO) treatment for amyotrophic lateral sclerosis (ALS) have longer median overall survival, according to a study published online Oct. 16 in Muscle & Nerve.

Noting that PB-TURSO significantly slowed functional decline in a randomized, placebo-controlled phase 2 trial in ALS (CENTAUR), Sabrina Paganoni, M.D., Ph.D., from Massachusetts General Hospital in Boston, and colleagues report on the impact of the therapy on long-term survival. In the original study, adults with ALS were randomly assigned to PB-TURSO or placebo in a 2:1 ratio. Those who completed the six-month randomized phase were eligible to receive PB-TURSO in an open-label extension. An all-cause mortality analysis was performed incorporating all randomized participants.

The researchers obtained vital status for 135 of 137 participants originally randomized. Median overall survival was 25.0 and 18.5 months among participants originally randomly assigned to PB-TURSO and placebo, respectively (hazard ratio, 0.56).

“The results presented here demonstrate a long-term survival benefit from early initiation of PB-TURSO treatment in participants with ALS, adding to the previously reported functional benefit,” the authors write.

Several authors disclosed financial ties to biopharmaceutical companies, including Amylyx Pharmaceuticals, which funded the study.

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