# Stem cell-based biomaterials for spinal regeneration in neural tube defects

> **NIH NIH R01** · JOHNS HOPKINS UNIVERSITY · 2020 · $330,757

## Abstract

PROJECT SUMMARY/ABSTRACT
Children with a severe form of spina bifida, known as myelomeningocele (MMC), suffer from substantial and
life-long morbidities secondary to lower limb weakness and paralysis, hydrocephalus, cognitive impairment,
bladder and bowel dysfunction, and orthopedic abnormalities. Although a randomized trial has shown a
reduction of postnatal hydrocephalus after prenatal surgery, there remains a critical need to provide these
children with an operative treatment that can better enhance neurologic function. Given the known
regenerative properties of neural progenitor cells transplanted in other models of spinal cord injury, the
application of neurons reprogrammed from amniotic fluid cells to treat MMC defects offers a novel, clinically
relevant, and potentially autologous alternative to conventional fetal MMC repair. Our central hypothesis is that
fetal neurosurgical treatment of spina bifida defects using a composite, cell-based neural patch with trophic
factor (sonic hedgehog, neurotrophin-3) functionality can maximally enhance neuronal regeneration within the
MMC spinal cord through engraftment and paracrine effects. In Specific Aim 1, we will investigate the short-
term paracrine effects of neural patches on the fetal MMC spinal cord. In Specific Aim 2, we will determine the
extent to which neural patches augment long-term MMC spinal cord regeneration and neurologic function in
vivo. The cornerstone of this proposal is the multidisciplinary team composed of an early-stage, fetal surgeon-
scientist (Dr. Kunisaki), academic neurosurgeon (Dr. Patil), senior developmental neurobiologist (Dr. O'Shea),
and senior materials science engineer (Dr. Shea). The expected outcomes will have validated a regenerative
medicine approach with high potential for clinical translation in the treatment of spina bifida and other spinal
cord injuries.

## Key facts

- **NIH application ID:** 10000197
- **Project number:** 5R01HD091323-04
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Shaun Michael Kunisaki
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $330,757
- **Award type:** 5
- **Project period:** 2017-09-01 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10000197

## Citation

> US National Institutes of Health, RePORTER application 10000197, Stem cell-based biomaterials for spinal regeneration in neural tube defects (5R01HD091323-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10000197. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*