Skip to Content

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.

Abstract/Full Text (subscription or payment may be required)

© 2020 HealthDay. All rights reserved.

Posted: October 2020

Read this next

AHA Meeting Addresses Latest Research, COVID-19, Health Care Disparities

MONDAY, Nov. 23, 2020 -- Fresh takes on the efficacy of fish oil and vitamin D, new treatments for heart failure patients, the cardiovascular effects of COVID-19, and structural...

AHA: Blacks, Hispanics Make Up 53 Percent of COVID-19 Deaths

MONDAY, Nov. 23, 2020 -- Due to disproportionate representation, Black and Hispanic patients bear much of the burden of mortality and morbidity related to COVID-19, according to a...

Predictors of Late Effects in Young AML Survivors Explored

MONDAY, Nov. 23, 2020 -- Many adolescent and young adult (AYA) survivors of acute myeloid leukemia (AML) experience late effects, with the most common being endocrine,...