Amyotrophic lateral sclerosis (ALS) is a rare but fatal neurodegenerative disease that affects the nerve cells responsible for controlling the muscles in the body. There is currently no cure for ALS, and treatment options are limited. However, an investigational product called RNS60 has shown promise in treating ALS in animal models and early-phase clinical trials.
In a phase II clinical trial, researchers investigated the safety and efficacy of RNS60 in treating ALS. The trial involved 147 participants diagnosed with definite, probable, or probable laboratory-supported ALS. The participants were randomly assigned to receive either RNS60 or a placebo for 24 weeks. RNS60 was administered intravenously once a week and via nebulization (a way of inhaling medication) on non-infusion days. After the initial treatment period, the participants were followed up for an additional 24 weeks off-treatment.
The primary objective of the trial was to measure the effects of RNS60 on selected biomarkers of inflammation and neurodegeneration in peripheral blood. The secondary objectives were to measure the effect of RNS60 on functional impairment, self-sufficiency, respiratory function, quality of life, and survival. The safety and tolerability of RNS60 were also assessed.
The results of the trial showed that RNS60 did not differ from the placebo in terms of the assessed biomarkers of inflammation and neurodegeneration in peripheral blood. The trial found that people who took RNS60 had a slower decline in their breathing ability, measured by Forced Vital Capacity (FVC), and had less trouble with eating and drinking measured by ALS Assessment Questionnaire-40 (ALSAQ-40) than people who took the placebo. FVC measures the amount of air a person can exhale forcefully after taking a deep breath, while the ALSAQ-40 assesses the quality of life of people with ALS. These positive effects of RNS60 on respiratory and bulbar function warrant further investigation.
The safety and tolerability of RNS60 were also assessed in the trial, and the results showed that adverse events were similar in the two groups. In a post hoc analysis, the neurofilament light chain increased over time in bulbar onset placebo participants while remaining stable in those treated with RNS60. This suggests that RNS60 may have a protective effect on nerve cells in people with bulbar onset ALS.
The results of this phase II clinical trial suggest that RNS60 may be a promising treatment option for people with ALS. The positive effects of RNS60 on respiratory and bulbar function warrant further investigation. While there is still much to learn about RNS60 and its potential as a treatment for ALS, this trial provides hope for people living with this devastating disease.