Finding existing medicines to treat Hereditary Hemorrhagic Telangiectasia using a blood-vessel-on-a-chip
Repurposing drugs to treat Hereditary Hemorrhagic Telangiectasia: identification using a vascularized HHT on-a-chip microphysiological system platform
This project tests whether already-approved drugs can correct the abnormal blood vessels that cause bleeding in people with Hereditary Hemorrhagic Telangiectasia using a lab-grown blood-vessel model.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California-Irvine NIH-funded |
| Lab location | 1 site (Irvine, United States) |
| Project ID | NIH-11251985 on NIH RePORTER |
What this research studies
Researchers will use a lab-grown 'blood-vessel-on-a-chip' that models the abnormal vessels seen in HHT to mimic how patient blood vessels behave. They will screen a library of de-risked (previously used) drugs to find compounds that restore normal endothelial cell behavior tied to ALK1 (ACVRL1) and endoglin (ENG) signaling. The team will prioritize drugs that reduce vessel overgrowth, leakiness, and other features that lead to nosebleeds and arteriovenous malformations. Promising candidates will undergo follow-up lab testing and could be advanced toward clinical testing for people with HHT.
Who could benefit from this research
Good fit: Ideal candidates are people with a clinical diagnosis of HHT, especially those with known ACVRL1 (ALK1) or ENG mutations and frequent nosebleeds or organ AVMs.
Not a fit: People without HHT, or whose bleeding is caused by unrelated conditions, are unlikely to benefit directly from this research.
Why it matters
Potential benefit: If successful, this work could speed up availability of treatments for HHT by identifying existing drugs that reduce bleeding and dangerous arteriovenous malformations.
How similar studies have performed: Some repurposed therapies such as anti-VEGF agents have helped HHT patients, but using a human vascular 'on-a-chip' platform for drug screening is a novel approach.
Where this research is happening
Irvine, United States
- University of California-Irvine — Irvine, United States (Active)
Researchers
- Principal investigator: Hughes, Christopher C. W. — University of California-Irvine
- Study coordinator: Hughes, Christopher C. W.
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.