Extracellular Vesicle–Enhanced Mitochondrial Therapy for ALS and Neurodegeneration
Official title: Extracellular Vesicle-Enhanced Mitochondrial Therapy for ALS and Neurodegeneration
Funded at University of Georgia · NIH-11514491 · Data updated 2026-11-12
A new approach uses tiny cell-made bubbles to deliver healthy mitochondria to nerve cells affected by ALS and other neurodegenerative diseases, aiming to slow disease progression and protect brain and spinal cord function.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Georgia NIH-funded |
| Lab location | 1 site (Athens, United States) |
| Project start | September 2, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $402,347 |
| NIH project number | 1R21NS148841-01A1 |
| Project ID | NIH-11514491 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with ALS, Alzheimer’s disease/dementia, or related neurodegenerative conditions who are studying or near future participation in trials exploring novel CNS-delivery therapies.
✕Less likely to help
Patients with other non-neurodegenerative conditions or very advanced disease stages not suitable for CNS-delivery therapy may not benefit.
What this research studies
This project aims to overcome the delivery barriers to mitochondria by using extracellular vesicles from neural progenitor cells to transport healthy mitochondria into damaged neurons. The approach targets neurodegenerative diseases like ALS, Alzheimer’s, and Parkinson’s by leveraging the protective and regenerative signals carried by the vesicles. Researchers will develop and test the EV-based delivery platform to cross the blood–brain barrier and improve cellular uptake in preclinical models, with the goal of translating findings toward patient care. The study combines neuroscience, cell biology, and delivery technology to address a critical bottleneck in treating central nervous system diseases.
Why it matters
Potential benefit: If successful, patients could experience slower disease progression and preservation of movement and cognition through a more effective way to deliver mitochondrial therapy to affected brain regions.
How similar studies have gone: Previous preclinical work suggests mitochondrial transplantation can be beneficial, but CNS delivery remains a major hurdle; this EV-based strategy is a novel extension with promising early signals.
Where this research is happening
-
University of Georgia
Athens, United States Active
Researchers
- Principal investigator: Yao, Yao — University of Georgia
- Study coordinator: Yao, Yao
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.