Neural stem cell vesicles slowed disease progression in an ALS mouse model
Original source
Neural stem cell-derived small extracellular vesicles ameliorate disease progression in the SOD1 G93A murine model of amyotrophic lateral sclerosis. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human, Mouse
Population inferred from the title and abstract by Compass.
Related topics
Repeated treatment with small extracellular vesicles from neural stem cells improved movement and protected motor neurons, neuromuscular junctions and muscle in SOD1 G93A mice. The findings were linked to reduced activity of the p53 pathway in treated mice and to muscle RNA patterns from people with ALS.
Why this matters
The results identify a possible treatment approach for ALS, but they come from a mouse model and do not show that the vesicles are safe or effective in people. They therefore do not currently change treatment for people living with ALS.
Limitations and context
This was primary research in the SOD1 G93A transgenic mouse model, not a human clinical trial. The study used repeated administration and reported supporting RNA-sequencing analyses from people with ALS, but those analyses do not establish that the vesicles work in humans. Human safety, dosing and effectiveness still need to be tested.