Nasal delivery of ALS treatment in tiny cell-made sacs delays disease onset in mice

by Charlene Betourney, University of Georgia

edited by Lisa Lock, reviewed by Alexander Pol

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One of the great challenges in treating diseases like amyotrophic lateral sclerosis (ALS) is not necessarily finding a promising drug. It is getting the drug to the right place.

Nature may already have the answer. New research from the University of Georgia's Regenerative Bioscience Center (RBC) is revealing how tiny, naturally produced carriers could help deliver medicines to the cells that need them most.

"Our research grew from a simple idea—instead of forcing a drug to find its way into the brain, give it a vehicle that already knows how to get there," said Yao Yao, an associate professor in UGA's College of Agricultural and Environmental Sciences and corresponding author of the study published in the Journal of Extracellular Vesicles.

Antisense oligonucleotides (ASOs) are drugs designed to stop specific disease-causing signals in cells, and they are emerging as a promising approach to treating ALS. But getting these treatments to the brain and spinal cord is no easy task.

ASOs do not readily cross the blood–brain barrier. The barrier can block them, cells may not readily take them up, and the body can remove or break them down before they have a chance to work. For these reasons, ASOs typically require delivery directly into the fluid surrounding the brain and spinal cord.

Neural stem cells naturally produce small fluid-filled sacs, or vesicles, that can carry molecules between cells. These tiny carriers may help treatments reach the brain and spinal cord despite the blood–brain barrier. The vesicles themselves also have biological effects that could complement the drug they carry.

To test nature's delivery system, the researchers turned to RBC director Steven Stice's specialized neural stem cell line, which produces the tiny vesicles used in AB126, an Aruna Bio investigational therapy that has advanced to clinical testing.

By pairing these types of biological couriers with ASOs, the researchers may have found a way around one of the biggest obstacles in treating neurological disease, at least in mice. "What sets this approach apart is that the vesicles do more than deliver a therapy. They have therapeutic properties of their own. We're harnessing both," said Yao.

Help delivered through the nose

In nerve cells grown in the laboratory, more of the vesicle-packaged treatment made its way inside test cells than when ASO was given on its own. But getting the treatment into the cells was only the first step. The researchers wanted to know whether the vesicles could carry the ASOs farther—into the nervous system through the nose.

The findings were encouraging. In mice, nasal delivery delayed disease onset, improved movement, protected motor neurons and reduced inflammation in the nervous system. Together, the results point to a potentially simpler way—think: no spinal tap—to deliver ASOs to the brain and spinal cord.

"When we packaged the ASO in these vesicles, we saw that it was more stable, reached neural tissue more effectively and stayed active longer than the ASO by itself," said Yao.

The RBC researchers also wanted to know where the drug itself was going, not simply whether the vesicles reached the right tissue.

"We put a tiny marker on the ASO drug itself, not the vesicle carrying it, so we could see that it was the drug reaching the target tissue, not just the carrier," said first author Yaochao Zheng, a doctoral student in UGA's Department of Animal and Dairy Science. "The signal showed us which tissues and cells the treatment reached, giving us a clearer picture of its distribution."

Yao, Zheng and their colleagues collaborated with the laboratory of Jie Jiang, associate professor in the Emory University School of Medicine, whose work focuses on developing therapeutic approaches for ALS. Jiang was part of the team's initial pilot research effort that was supported by Regenerative Engineering and Medicine, a joint research initiative between UGA, Emory and Georgia Tech.

"The next step is to fine-tune how much treatment reaches the brain and spinal cord and test it across multiple ALS models, including human motor neurons," said Jiang.

"We're using something cells already make themselves as the delivery vehicle," said Yao. "Rather than building a system from scratch, we're borrowing one that biology has already figured out."

More information

Yaochao Zheng et al., Neural Stem Cell‐Derived Extracellular Vesicles Improve CNS Delivery and Therapeutic Efficacy of Antisense Oligonucleotide in Amyotrophic Lateral Sclerosis, Journal of Extracellular Vesicles (2026). DOI: 10.1002/jev2.70370

Key medical concepts

Amyotrophic Lateral SclerosisOligonucleotides, Antisense

Clinical categories

NeurologyClinical pharmacology Provided by University of Georgia Who's behind this story?

Lisa Lock

BA art history, MA material culture. Former museum editor, paramedic, and transplant coordinator. Editing for Science X since 2021. Full profile →

Alexander Pol

PhD nano-engineering from Delft University. Published researcher and journal reviewer. Brings scientific insight to content standards. Full profile →

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