ALK1-activating treatment for hereditary hemorrhagic telangiectasia (HHT)
Therapeutic Potential of ALK1 Activating Drugs in HHT Models
A new antibody approach that boosts ALK1 signaling to try to prevent or reverse abnormal blood vessels in people with HHT.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Feinstein Institute for Medical Research NIH-funded |
| Lab location | 1 site (Manhasset, United States) |
| Project ID | NIH-11252584 on NIH RePORTER |
What this research studies
Researchers are developing a bispecific antibody that links ALK1 and BMPRII receptors to turn on protective ALK1 signaling in blood vessel cells. They are testing whether this antibody prevents or reverses arteriovenous malformations (AVMs) in mouse models that mimic HHT and whether it restores signaling in blood vessel cells grown from patients with ALK1 mutations. The team will study how the antibody affects vessel growth, bleeding risk, and the molecular pathways that keep vessels normal. Positive results could support moving toward human testing or using patient samples to better target treatments.
Who could benefit from this research
Good fit: People diagnosed with HHT—especially those with confirmed ALK1 mutations or active arteriovenous malformations—would be the most likely candidates for related trials or sample donation.
Not a fit: Patients whose HHT is caused by other genes (such as ENG or SMAD4) or those without ALK1-related pathology may be less likely to benefit from an ALK1-targeted therapy.
Why it matters
Potential benefit: If successful, this approach could shrink or prevent dangerous AVMs and reduce bleeding for people with HHT.
How similar studies have performed: Preclinical work in mice and in patient-derived blood vessel cells has shown promising reversal of AVMs and activation of ALK1 signaling, but human testing remains novel.
Where this research is happening
Manhasset, United States
- Feinstein Institute for Medical Research — Manhasset, United States (Active)
Researchers
- Principal investigator: Marambaud, Philippe — Feinstein Institute for Medical Research
- Study coordinator: Marambaud, Philippe
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.