New treatments for maternally inherited mitochondrial diseases MELAS and LHON-Plus
Official title: Emerging therapeutic candidates for rare maternally inherited mitochondrial diseases with shared etiologies
Funded at George Washington University · NIH-11191529 · Data updated 2026-11-12
New drug candidates aim to boost cellular energy for people with MELAS or LHON-Plus, rare maternally inherited mitochondrial diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | George Washington University NIH-funded |
| Lab location | 1 site (Washington, United States) |
| Project start | July 1, 2021 |
| Project end | August 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $916,686 |
| NIH project number | 5UH3TR003897-05 |
| Project ID | NIH-11191529 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with a confirmed genetic diagnosis of MELAS or LHON‑Plus due to mitochondrial Complex I defects who are willing and able to attend visits at participating clinical sites.
✕Less likely to help
People with other types of mitochondrial disease not caused by Complex I defects, those without a confirmed genetic diagnosis, or those unable to travel to trial sites are unlikely to benefit from this trial.
What this research studies
You would join a basket clinical trial that combines people with MELAS and LHON‑Plus so researchers can study both groups together. The trial targets a shared molecular problem called Complex I deficiency that lowers mitochondrial ATP production and causes chronic energy shortage in cells. The project moves from early development into clinical testing to look at safety, feasibility, and whether symptoms like stroke‑like episodes, muscle weakness, or vision loss improve or stabilize. Because these conditions are ultra‑rare, the study works through the Rare Diseases Clinical Research Network to recruit patients and make the trial possible.
Why it matters
Potential benefit: If successful, the treatment could raise cellular energy production and reduce major symptoms such as stroke‑like episodes, muscle weakness, or vision loss.
How similar studies have gone: Some mitochondrial‑targeting drugs have shown mixed results (for example, idebenone in LHON), so this approach builds on limited prior successes but remains experimental.
Where this research is happening
-
George Washington University
Washington, United States Active
Researchers
- Principal investigator: Chiaramello, Anne Eliane — George Washington University
- Study coordinator: Chiaramello, Anne Eliane
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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.