Understanding why some patients with LAM do not respond to sirolimus treatment
Cellular determinants of responsiveness to mTOR inhibitors in LAM
This study is looking into why some people with Lymphangioleiomyomatosis (LAM) don’t respond well to sirolimus, the only approved treatment for this lung condition, by examining their cells to find out what makes them different, so we can improve how we treat LAM in the future.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11046114 on NIH RePORTER |
What this research studies
This research investigates the reasons behind the varying responses of patients with Lymphangioleiomyomatosis (LAM) to sirolimus, the only FDA-approved treatment for this lung disease. By analyzing specific cellular mechanisms and gene expressions in patients' cells, the study aims to identify why some individuals do not benefit from the medication. The approach includes advanced techniques like single-cell RNA sequencing to pinpoint resistant cell populations and their characteristics. The ultimate goal is to enhance treatment strategies for LAM patients by understanding these cellular determinants.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Lymphangioleiomyomatosis (LAM), particularly those who have experienced limited or no benefit from sirolimus treatment.
Not a fit: Patients with LAM who have not been treated with sirolimus or those with other unrelated lung conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options for LAM patients who currently do not respond to sirolimus.
How similar studies have performed: Previous research has shown promising results in understanding treatment responses in similar conditions, indicating that this approach could yield valuable insights.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Xu, Yan — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Xu, Yan
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.