How cholesterol inside kidney cells may change adult polycystic kidney disease
Investigating intracellular cholesterol biosynthesis as a regulator of polycystic kidney disease progression
['FUNDING_R01'] · UT SOUTHWESTERN MEDICAL CENTER · NIH-11249595
The team will test whether increasing cholesterol production inside kidney cells can slow cyst growth in adults with autosomal dominant polycystic kidney disease (ADPKD).
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UT SOUTHWESTERN MEDICAL CENTER (nih funded) |
| Locations | 1 site (DALLAS, UNITED STATES) |
| Trial ID | NIH-11249595 on ClinicalTrials.gov |
What this research studies
Researchers will study how the SREBP proteins that control cholesterol and fat production inside cells affect cyst growth using mouse models and analyses of human kidney tissue. They will reduce or boost SREBP activity in animals to see how those changes alter cholesterol levels, cyst size, and survival. The team will measure cholesterol species and related genes in human ADPKD kidneys to confirm whether the findings in mice apply to people. Together these steps aim to determine if restoring cholesterol pathways in kidneys could be a new way to slow cyst progression.
Who could benefit from this research
Good fit: Adults with autosomal dominant polycystic kidney disease, especially those with early-to-moderate cyst growth, would be the most relevant group for future participation or trials based on this work.
Not a fit: People with end-stage kidney disease on long-term dialysis or those who have already had a kidney transplant are less likely to benefit from cholesterol-targeted approaches to slow cyst growth.
Why it matters
Potential benefit: If successful, this work could point to new treatments that slow cyst growth with different side-effect profiles than current options.
How similar studies have performed: Previous animal work from this group showed that activating SREBP proteins slowed cyst growth in mouse models, but targeting cholesterol specifically is a newer, less-tested approach in humans.
Where this research is happening
DALLAS, UNITED STATES
- UT SOUTHWESTERN MEDICAL CENTER — DALLAS, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LAKHIA, RONAK — UT SOUTHWESTERN MEDICAL CENTER
- Study coordinator: LAKHIA, RONAK
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.
Conditions: Adult Polycystic Kidney Disease