Investigating a new approach to treat muscle degeneration in mitochondrial diseases
Official title: Targeting GDF15-GFRAL signaling in mitochondrial myopathies
Funded at Weill Medical Coll of Cornell Univ · NIH-11112908 · Data updated 2026-11-12
This study is looking at a way to help people with mitochondrial myopathies by blocking a specific pathway that causes muscle loss, hoping to improve their weight, energy, and overall muscle health.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Weill Medical Coll of Cornell Univ NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | June 1, 2025 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $216,113 |
| NIH project number | 1R21AR085715-01 |
| Project ID | NIH-11112908 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with mitochondrial myopathies who experience muscle degeneration and cachexia.
✕Less likely to help
Patients with mitochondrial diseases who do not exhibit symptoms of muscle degeneration or cachexia may not benefit from this research.
What this research studies
This research focuses on understanding and targeting the GDF15-GFRAL signaling pathway, which plays a role in muscle degeneration associated with mitochondrial myopathies. By using mouse models, the researchers aim to inhibit this signaling to prevent cachexia, a condition characterized by severe weight loss and muscle wasting. The study will explore the effects of established antibodies on caloric intake and energy expenditure, potentially leading to improved muscle function and overall health. Patients may benefit from insights gained about metabolic drivers of muscle degeneration and new therapeutic strategies.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that improve muscle function and prevent weight loss in patients with mitochondrial myopathies.
How similar studies have gone: Previous research has shown promise in targeting metabolic pathways in muscle degeneration, suggesting that this approach may yield beneficial results.
Where this research is happening
-
Weill Medical Coll of Cornell Univ
New York, United States Active
Researchers
- Principal investigator: D'aurelio, Marilena — Weill Medical Coll of Cornell Univ
- Study coordinator: D'aurelio, Marilena
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.