mTOR signaling and heart changes in adult polycystic kidney disease
Official title: mTORC1/2 Signaling in the Heart in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Funded at VA Eastern Colorado Health Care System · NIH-11212743 · Data updated 2026-11-12
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 start | July 1, 2018 |
| Project end | September 30, 2026 |
| Funding year | 2026 |
| NIH project number | 5I01BX003803-08 |
| Project ID | NIH-11212743 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
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.
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.
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 gone: 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
-
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
Related research
Other NIH-funded projects on the same topics.
-
Personalized CKD-MBD care using systems biology and AI
A patient-facing program aims to tailor mineral-balance therapy for people with chronic kidney disease by using a computer model and smart decision rules to predict who will respond best to treatments.
-
Mitigating DKA in people with type 1 diabetes and kidney disease using SGLT1/2 inhibitors
This project tests a new way to prevent dangerous diabetic ketoacidosis in people with type 1 diabetes and chronic kidney disease who are using SGLT inhibitors, by tracking ketones in real time and combining glucose-keton data for safer therapy.
-
Chronic Kidney Disease in Children: following young people as they grow up with CKD
A long-term study in kids with chronic kidney disease tracks kidney function and heart health as they transition into adulthood.
-
Chronic Kidney Disease in Children follow-up study
A long-term study that follows kids with kidney disease as they grow into young adults, collecting health data from home and official records.
-
KDARC: Building a research backbone to study aging and kidney disease
Aimed at creating a dedicated infrastructure to advance aging-related kidney disease research so clinicians can better care for older adults.
-
Malaria-related kidney injury and its link to chronic kidney disease in children
This grant looks at why some children who have malaria develop kidney injury and later chronic kidney disease, focusing on how severe malaria affects the kidneys over time.
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.