TRPC6 blocker to protect the heart in Duchenne muscular dystrophy
Official title: TRPC6 inhibition therapy to rescue cardiac muscle dysfunction in muscular dystrophy
Funded at Medical College of Wisconsin · NIH-11291309 · Data updated 2026-11-12
This project uses a drug that blocks the TRPC6 channel to protect heart and skeletal muscles in adults with Duchenne muscular dystrophy.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | March 18, 2024 |
| Project end | February 28, 2027 |
| Funding year | 2026 |
| Award (this year) | $249,000 |
| NIH project number | 5R00HL155840-05 |
| Project ID | NIH-11291309 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for future human testing would be adults (age 21+) with Duchenne muscular dystrophy, especially those showing signs of cardiac involvement or arrhythmia.
✕Less likely to help
People without DMD, children under 21, or patients whose heart damage is already advanced and irreversible are unlikely to benefit directly from this preclinical work.
What this research studies
From a patient point of view, researchers are testing a small, pill-like drug that blocks a muscle channel called TRPC6, which is overactive in Duchenne muscular dystrophy (DMD). They will treat mouse models of DMD and use engineered heart tissue to see if the drug reduces scarring, abnormal calcium handling, arrhythmias, and muscle weakness. The team will measure heart function, muscle strength, and survival and will study the cellular signals that cause damage. These lab and preclinical results are meant to guide whether this approach could move into human trials.
Why it matters
Potential benefit: If successful, this approach could slow or prevent heart muscle failure and reduce muscle scarring in people with DMD, potentially improving lifespan and quality of life.
How similar studies have gone: Early animal studies reported by the team showed that genetic or drug-based TRPC6 blockade prolonged lifespan and improved heart and muscle function in severe DMD models, and related TRPC6 inhibitors are already in human trials for other conditions.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Lin, Brian Leei — Medical College of Wisconsin
- Study coordinator: Lin, Brian Leei
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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.