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Updated: May 21, 2025

A Protocol for Comprehensive Assessment of Bulbar Dysfunction in Amyotrophic Lateral Sclerosis ALS

A Protocol for Comprehensive Assessment of Bulbar Dysfunction in Amyotrophic Lateral Sclerosis ALS

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An Expanded Access Protocol of RNS60 in Amyotrophic Lateral Sclerosis.

Grace Addy¹, Erica Scirocco¹, Dario Gelevski¹

¹Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.

|March 21, 2025

Summary

This summary is machine-generated.

RNS60, investigated for amyotrophic lateral sclerosis (ALS), showed a favorable safety profile in an expanded access protocol. Twice-daily nebulization was well-tolerated in patients ineligible for clinical trials.

Keywords:

RNS60 amyotrophic lateral sclerosis expanded access motor neuron disease neuroinflammation

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Area of Science:

Neuroscience
Clinical Pharmacology
Pulmonary Medicine

Background:

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
RNS60 is an investigational therapy for ALS, previously showing disease-slowing effects in a mouse model.
Previous studies indicated RNS60 was safe and well-tolerated in ALS patients.

Purpose of the Study:

To evaluate the safety and tolerability of RNS60 administered via twice-daily nebulization in ALS patients.
To provide RNS60 access to patients ineligible for controlled clinical trials.
To assess long-term dosing feasibility in advanced ALS.

Main Methods:

An ongoing expanded access protocol (EAP) treated 84 participants with ALS.
Participants received RNS60 via twice-daily home nebulization for up to 48 months.
Safety was monitored through adverse events and laboratory tests.

Main Results:

The most frequent treatment-related adverse event was increased secretions (32%).
Serious adverse events and deaths were not considered related to RNS60 treatment.
The study demonstrated the feasibility of long-term RNS60 administration.

Conclusions:

Twice-daily nebulization of RNS60 exhibits a benign side effect profile in ALS patients.
Expanded access protocols are a viable complementary approach to clinical trials for advanced ALS.
RNS60 shows promise for long-term management in ALS patients outside of traditional trial settings.