# Novel Biologics to ablate choroid plexus for the treatment of neonatal hydrocephalus

> **NIH NIH R61** · CINCINNATI CHILDRENS HOSP MED CTR · 2024 · $394,544

## Abstract

Project Summary
Surgical treatment via ventriculoperitoneal shunt or endoscopic 3rd ventriculostomy is currently the most
commonly used strategy to treat pediatric hydrocephalus. However, shunt-treated hydrocephalic patients
continue to experience significant lifelong neurological problems and high malfunctioning shunt replacement
surgery rates. The endoscopic choroid plexus (ChP) coagulation helps manage cerebrospinal fluid (CSF)
volume by surgically removing the primary CSF production organ in the brain ventricles. However, this surgical
procedure requires neurosurgeons with extensive training and still achieves only partial ChP removal. This
study aims to evaluate a novel, potent, and less invasive ChP ablation tool in human ChP organoids and
rodent brains that can support new ways of treating neonatal hydrocephalus.
The NIH Funding Opportunity program announcement PAR-21-122 recognizes the unmet need for
translational studies for neurotherapeutics agents for treating neurological or neuromuscular disorders and
new therapeutics. In response to this opportunity, our proposal focuses on developing a new ChP ablation
tool via ChP-specific delivery of a clinically validated suicide gene. By utilizing cutting-edge techniques such
as recombinant adeno-associated virus (AAV) vector targeting ChP, human ChP organoids, robust rodent
models of neonatal hydrocephalus, and advanced MRI, including DTI and animal behavior assays, our project
aims to achieve rigorous efficacy and safety studies of this new tool.
The completion of this study will determine (1) pharmacokinetics and off-target effects and (2) the therapeutic
efficacy of this AAV-mediated ChP ablation tool, which has high potential to provide a new strategy for treating
pediatric hydrocephalus.

## Key facts

- **NIH application ID:** 10972862
- **Project number:** 1R61NS138647-01
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** JUNE GOTO
- **Activity code:** R61 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $394,544
- **Award type:** 1
- **Project period:** 2024-09-17 → 2026-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10972862

## Citation

> US National Institutes of Health, RePORTER application 10972862, Novel Biologics to ablate choroid plexus for the treatment of neonatal hydrocephalus (1R61NS138647-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10972862. Licensed CC0.

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