Targeted biologic treatment to remove the cerebrospinal fluid–producing tissue in newborns with hydrocephalus
Official title: Novel Biologics to Ablate Choroid Plexus for the Treatment of Neonatal Hydrocephalus
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-11194484 · Data updated 2026-11-12
A gene-delivery approach aimed at newborns with hydrocephalus to selectively remove the choroid plexus tissue that makes excess spinal fluid.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project start | September 17, 2024 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $379,463 |
| NIH project number | 5R61NS138647-02 |
| Project ID | NIH-11194484 on NIH RePORTER |
Who could benefit from this research
✓Could help
Newborns and infants with hydrocephalus due primarily to CSF overproduction or who are candidates for choroid plexus intervention would be the ideal future participants.
✕Less likely to help
Patients whose hydrocephalus is caused mainly by obstructed CSF flow requiring mechanical correction, older children or adults, or people with unrelated brain conditions may not benefit from this approach.
What this research studies
If my newborn had hydrocephalus, this project aims to create a less invasive way to stop excess cerebrospinal fluid by delivering a 'suicide' gene specifically to the choroid plexus, the tissue that produces cerebrospinal fluid. Researchers will test adeno-associated virus (AAV) vectors that target the choroid plexus using lab-grown human choroid plexus organoids and rodent models of neonatal hydrocephalus. They will monitor outcomes with advanced MRI (including DTI) and animal behavior tests to check for safety and effects on the brain. The ultimate goal is to develop a therapy that could reduce or replace the need for shunts or complex neurosurgery in affected infants.
Why it matters
Potential benefit: Could reduce or eliminate the need for shunt surgeries and the lifelong complications associated with shunts by stopping the tissue that over-produces cerebrospinal fluid.
How similar studies have gone: Endoscopic choroid plexus coagulation is used clinically to reduce CSF production and AAV gene therapies are proven in other diseases, but choroid plexus ablation via a suicide gene is a novel approach currently tested only in lab and animal models.
Where this research is happening
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Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Goto, June — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Goto, June
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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.