Gene correction combined with lung macrophage transplant for hereditary pulmonary alveolar proteinosis
Human gene transfer & macrophage cell transplantation therapy of hereditary PAP (hPAP)
This offers people with hereditary pulmonary alveolar proteinosis a one-time approach that corrects the faulty gene in their own stem cells and then delivers corrected lung immune cells to help clear excess lung surfactant and improve breathing.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11170668 on NIH RePORTER |
What this research studies
If you join, doctors will collect your blood or bone-marrow stem cells (CD34+), use a safe viral vector to add a working CSF2RA gene, grow the corrected cells into lung macrophages, and then place those macrophages into your lungs. The team has tested this approach in human cells and validated animal models, and now plans a Phase I effort to check safety and find useful clinical and lab measures for future trials. You would have detailed breathing tests, imaging, lung-fluid sampling, and safety monitoring over time to track effects and side effects.
Who could benefit from this research
Good fit: Ideal candidates are people diagnosed with hereditary PAP due to GM‑CSF receptor (CSF2RA/CSF2RB) defects who are medically stable enough for cell collection and bronchoscopic or instillation procedures.
Not a fit: People without hPAP caused by GM‑CSF receptor mutations, or those too medically unstable for cell collection or lung procedures, are unlikely to benefit from this therapy.
Why it matters
Potential benefit: If successful, this could restore lung macrophage function, clear accumulated surfactant, and reduce or prevent progressive respiratory failure in people with hPAP.
How similar studies have performed: Related gene-complementation and pulmonary macrophage transplant work has corrected cells and cured disease in validated animal models and restored function in human cells, but direct human PMT therapy is only now entering early clinical testing.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Trapnell, Bruce C — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Trapnell, Bruce C
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.