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TUESDAY, Oct. 20, 2020 (HealthDay News) — 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<\/i>.<\/p>\n
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.<\/p>\n
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).<\/p>\n
“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.<\/p>\n
Several authors disclosed financial ties to biopharmaceutical companies, including Amylyx Pharmaceuticals, which funded the study.<\/p>\n
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