New treatments for rare mitochondrial diseases passed from mother to child
Official title: Emerging therapeutic candidates for rare maternally inherited mitochondrial diseases with shared etiologies
Funded at George Washington University · NIH-10915696 · Data updated 2026-11-12
This study is looking for new treatment options for people with two rare inherited mitochondrial diseases, MELAS and LHON-Plus, which cause serious health issues, and it aims to see if a shared treatment can help both groups of patients feel better.
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 | June 30, 2026 |
| Funding year | 2024 |
| Award (this year) | $932,694 |
| NIH project number | 5UH3TR003897-04 |
| Project ID | NIH-10915696 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with MELAS or LHON-Plus, particularly those experiencing severe symptoms related to these conditions.
✕Less likely to help
Patients with other types of mitochondrial diseases or those not affected by MELAS or LHON-Plus may not benefit from this research.
What this research studies
This research focuses on developing new therapeutic options for two rare mitochondrial diseases, MELAS and LHON-Plus, which are inherited from mothers. These conditions lead to severe neurological symptoms and significant health challenges due to a deficiency in ATP production, which is essential for energy in cells. The study aims to combine patients from both disease groups in a clinical trial to test the effectiveness of potential treatments, addressing a critical need for effective interventions. By utilizing a basket trial design, the research seeks to demonstrate that patients with different but related conditions can benefit from similar therapeutic approaches.
Why it matters
Potential benefit: If successful, this research could lead to effective treatments that significantly improve the quality of life for patients suffering from these debilitating mitochondrial diseases.
How similar studies have gone: While the approach of combining ultra-rare disease populations in clinical trials is innovative, similar strategies have shown promise in other rare disease research, indicating potential for success.
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
Related research
Other NIH-funded projects on the same topics.
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Emerging therapies for rare maternal mitochondrial diseases with shared causes
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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.