INTRODUCTION: RNS60 is a novel immune-modulatory agent that has shown neuroprotective effects in amytrophic lateral sclerosis (ALS) preclinical models. RNS60 is administered by weekly intravenous infusion and daily nebulization. The objective of this pilot open-label trial was to test the feasibility, safety, and tolerability of long-term RNS60 administration in ALS patients.
METHODS: The planned treatment duration was 23 weeks and the primary outcomes were safety and tolerability. Secondary outcomes included PBR28 positron emission tomography (PET) imaging and plasma biomarkers of inflammation.
RESULTS: Sixteen participants with ALS received RNS60 and 13 (81%) completed 23 weeks of RNS60 treatment. There were no serious adverse events and no participants withdrew from the trial due to drug-related adverse events. There were no significant changes in the biomarkers.
DISCUSSION: Long-term RNS60 administration was safe and well-tolerated. A large, multicenter, phase II trial of RNS60 is currently enrolling participants to test the effects of RNS60 on ALS biomarkers and disease progression. Muscle Nerve 59:303-308, 2019.
- Administration, Inhalation
- Amyotrophic Lateral Sclerosis/diagnostic imaging
- Anti-Inflammatory Agents, Non-Steroidal/administration & dosage
- Brain/diagnostic imaging
- Healthy Volunteers
- Infusions, Intravenous
- Middle Aged
- Muscle Strength
- Pilot Projects
- Positron-Emission Tomography
- Sodium Chloride/adverse effects
- Treatment Outcome
- Young Adult