Improving metabolic treatment for neurological mitochondrial disorders
Official title: Metabolic treatment of neurological mitochondrial disorders
Funded at University of Washington · NIH-11053548 · Data updated 2026-11-12
This study is looking at how a drug called rapamycin might help improve symptoms for people with Leigh Syndrome and MELAS by blocking a specific pathway in the body, and it aims to find new ways to make treatments better for patients like you.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Washington NIH-funded |
| Lab location | 1 site (Seattle, United States) |
| Project start | April 1, 2024 |
| Project end | March 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $194,375 |
| NIH project number | 5R21NS136915-02 |
| Project ID | NIH-11053548 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Leigh Syndrome or MELAS.
✕Less likely to help
Patients with mitochondrial disorders not related to Leigh Syndrome or MELAS may not benefit from this research.
What this research studies
This research investigates how inhibiting a specific pathway in the body, known as mTOR, can improve conditions in patients with Leigh Syndrome and MELAS, two serious neurological mitochondrial disorders. The study will explore the effects of a drug called rapamycin on metabolic changes in animal models and a small group of patients. By understanding how this drug works, researchers aim to identify new treatment strategies that could enhance patient outcomes. The research will also examine the role of certain proteins in the body that may influence the effectiveness of these treatments.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options and better quality of life for patients with mitochondrial disorders.
How similar studies have gone: Previous research has shown promising results with mTOR inhibitors in similar conditions, indicating potential for success in this approach.
Where this research is happening
-
University of Washington
Seattle, United States Active
Researchers
- Principal investigator: Bitto, Alessandro — University of Washington
- Study coordinator: Bitto, Alessandro
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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.