New approaches to stop or reverse abnormal lung blood vessel connections (pulmonary AVMs)
Understanding and targeting molecular and cellular events responsible for pulmonary arteriovenous malformation development, growth and regression
Researchers are building lab and animal models to discover treatments that could prevent or shrink pulmonary arteriovenous malformations in people with hereditary hemorrhagic telangiectasia (HHT).
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project ID | NIH-11177921 on NIH RePORTER |
What this research studies
If you have HHT and pulmonary AVMs, this project aims to learn how those abnormal artery-to-vein connections form and change over time. The team will trace which part of the blood vessel network gives rise to PAVMs and identify the cellular signals that drive their growth or regression. They will create better animal and lab-grown models that mimic human PAVMs so potential drugs or molecular therapies can be tested before human use. The work focuses on genes already linked to HHT (like endoglin, ALK1, and SMAD4) to find actionable targets.
Who could benefit from this research
Good fit: Ideal candidates are people with hereditary hemorrhagic telangiectasia (HHT) who have known pulmonary AVMs or are at high risk for developing them.
Not a fit: People without HHT or those needing immediate clinical embolization should not expect direct or immediate personal benefit from this mostly laboratory-focused research.
Why it matters
Potential benefit: If successful, this work could lead to medical treatments that prevent, stop growth of, or even shrink pulmonary AVMs, reducing the need for repeat embolization and frequent imaging.
How similar studies have performed: There are currently no approved medications that reverse PAVMs and prior work is limited, so this approach is largely novel though grounded in known HHT genetics.
Where this research is happening
Stanford, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Spiekerkoetter, Edda Frauke — Stanford University
- Study coordinator: Spiekerkoetter, Edda Frauke
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.