Developing a treatment for CADASIL using a specialized antibody
Humanization of a Notch 3 Agonist Antibody for Pre-Clinical Development of a CADASIL Treatment
['FUNDING_U01'] · SCHEPENS EYE RESEARCH INSTITUTE · NIH-11035208
This study is working on a new treatment for CADASIL, a genetic condition that can cause strokes and memory problems, by developing a special antibody that helps protect the blood vessels in the brain, and they’re testing it in mice to see if it can prevent damage to important cells.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SCHEPENS EYE RESEARCH INSTITUTE (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11035208 on ClinicalTrials.gov |
What this research studies
This research focuses on creating a humanized version of a Notch 3 agonist antibody to treat CADASIL, a genetic condition that leads to strokes and cognitive decline. The approach involves optimizing this antibody to enhance its effectiveness in activating Notch 3 signaling, which is crucial for maintaining the health of blood vessels in the brain. By using transgenic mouse models, the researchers aim to demonstrate the antibody's ability to prevent the loss of important mural cells that contribute to vascular dysfunction. If successful, this could lead to a targeted therapy for patients suffering from CADASIL.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with CADASIL, particularly those with Notch 3 mutations.
Not a fit: Patients without CADASIL or those with other forms of vascular cognitive impairment may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a novel treatment option for patients with CADASIL, potentially improving their vascular health and cognitive function.
How similar studies have performed: Previous studies have shown promising results using similar antibody approaches in animal models, indicating potential for success in human applications.
Where this research is happening
BOSTON, UNITED STATES
- SCHEPENS EYE RESEARCH INSTITUTE — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ARBOLEDA-VELASQUEZ, JOSEPH — SCHEPENS EYE RESEARCH INSTITUTE
- Study coordinator: ARBOLEDA-VELASQUEZ, JOSEPH
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.