mTOR signaling and heart changes in adult polycystic kidney disease
mTORC1/2 Signaling in the Heart in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Testing newer mTOR-targeting drugs and heart-penetrating antisense therapies to prevent or reduce heart enlargement in people with autosomal dominant polycystic kidney disease.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | VA Eastern Colorado Health Care System NIH-funded |
| Lab location | 1 site (Aurora, UNITED STATES) |
| Project ID | NIH-11212743 on NIH RePORTER |
What this research studies
This project looks at why people with ADPKD can develop heart enlargement and diastolic dysfunction even before high blood pressure appears. The team will use genetic PKD models and give newer mTORC1-selective drugs (such as DL-001) and palmitate-conjugated antisense oligonucleotides designed to get into heart muscle. They will compare heart size, function, and cell signaling changes after genetic or drug-based manipulation of mTORC1 and mTORC2. Results from these models could guide development of therapies aimed at protecting the heart in people with ADPKD.
Who could benefit from this research
Good fit: Adults with genetically confirmed autosomal dominant polycystic kidney disease, especially those with early signs of heart enlargement or diastolic dysfunction, would be the most relevant candidates.
Not a fit: People without ADPKD or those whose heart disease is driven solely by long-standing hypertension or advanced, irreversible heart failure are less likely to benefit from these specific mTOR-targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to treatments that prevent or reverse heart enlargement and diastolic dysfunction in people with ADPKD.
How similar studies have performed: Prior preclinical work with rapamycin and newer mTORC1-selective compounds like DL-001 has shown promising effects in cells and animals, but human benefits have not yet been proven.
Where this research is happening
Aurora, UNITED STATES
- VA Eastern Colorado Health Care System — Aurora, United States (Active)
Researchers
- Principal investigator: Edelstein, Charles Louis — VA Eastern Colorado Health Care System
- Study coordinator: Edelstein, Charles Louis
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.