New treatments for rare mitochondrial diseases passed from mother to child
Emerging therapeutic candidates for rare maternally inherited mitochondrial diseases with shared etiologies
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 ID | NIH-10915696 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with MELAS or LHON-Plus, particularly those experiencing severe symptoms related to these conditions.
Not a fit: Patients with other types of mitochondrial diseases or those not affected by MELAS or LHON-Plus may not benefit from this research.
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 performed: 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
Washington, United States
- George Washington University — Washington, United States (Active)
Researchers
- Principal investigator: Chiaramello, Anne Eliane — George Washington University
- Study coordinator: Chiaramello, Anne Eliane
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 below.