Brain-targeted enzyme therapy for neuronopathic Gaucher disease
Systemic Enzyme Delivery by a Brain Targeted SapC-DOPS Nanocarrier for Treatment of Neuronopathic Gaucher Disease
This project uses an IV-delivered, brain-penetrating nanocarrier carrying a stable enzyme to reduce harmful glycolipid buildup in people with neuronopathic Gaucher disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-11126812 on NIH RePORTER |
What this research studies
If you or your child joined this work, doctors would give an IV infusion of a SapC-DOPS nanocarrier carrying a new, long-lasting enzyme called fGCase intended to reach the brain. In laboratory and mouse tests this carrier has crossed the blood-brain barrier, kept the enzyme active for longer, and lowered the harmful glycolipids that accumulate in neuronopathic Gaucher disease. The team will measure where the packaged enzyme goes in the body, how long it remains functional, and whether it safely lowers disease markers in the brain. If results are favorable, the data would support moving toward safety and dosing studies in people to see if neurological symptoms can be prevented or improved.
Who could benefit from this research
Good fit: People with neuronopathic Gaucher disease who have neurological symptoms or are at high risk for CNS progression would be the most relevant candidates.
Not a fit: Patients with non-neuronopathic (visceral-only) Gaucher disease, those with advanced irreversible brain damage, or those who cannot receive IV therapy may not directly benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could reduce brain glycolipid accumulation and could help prevent or lessen neurological decline in people with neuronopathic Gaucher disease.
How similar studies have performed: Current enzyme replacement therapies help visceral Gaucher disease but do not cross the blood-brain barrier; this SapC-DOPS nanocarrier approach is novel with promising animal results but has not yet been proven in people.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Sun, Ying — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Sun, Ying
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.