Effect of RNS60 in amyotrophic lateral sclerosis: a phase II multicentre, randomized, double-­blind, placebo-­controlled trial

This study investigated the effects of RNS60, a potential treatment, on people living with Amyotrophic Lateral Sclerosis (ALS). RNS60 has shown promise in animal models of ALS and other neurodegenerative diseases. The trial involved 147 participants who were randomly assigned to receive either RNS60 or a placebo. The treatment was administered intravenously and via nebulization for 24 weeks, followed by a 24-week period without treatment. The study found that RNS60 did not significantly affect the levels of certain biological markers associated with inflammation and nerve damage. However, it did show some positive effects on measures of breathing and bulbar function (functions related to the throat and mouth).

• 1
  RNS60 administration was associated with slower rates of decline in respiratory function compared to placebo over a 24-week treatment period.
• 2
  The mean rate of change in the eating and drinking domains of the ALSAQ-40 were significantly slower in the RNS60 arm.
• 3
  Post hoc analysis in bulbar onset participants showed NfL levels were stable over the 24 weeks of treatment in the RNS60 arm, whilst in the placebo arm the levels increased.