Cell-based brain protection for infants with congenital heart defects
Official title: Cell Therapy for Neuroprotection in Congenital Heart Disease
Funded at Boston University Medical Campus · NIH-11375931 · Data updated 2026-11-12
This project uses bone marrow-derived mesenchymal cells given through the heart-lung machine during surgery to help protect the brains of infants and young children with congenital heart defects.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston University Medical Campus NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | December 15, 2017 |
| Project end | April 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $684,944 |
| NIH project number | 5R01HL139712-08 |
| Project ID | NIH-11375931 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are infants and young children with congenital heart defects who will undergo cardiac surgery requiring cardiopulmonary bypass.
✕Less likely to help
Children who do not undergo bypass-based heart surgery, older patients, or those whose brain injury stems from unrelated causes are unlikely to benefit from this specific intervention.
What this research studies
From a parent's view, the team is developing a way to deliver therapeutic bone marrow-derived cells (BM-MSCs) through the cardiopulmonary bypass machine used in infant heart surgery to protect the developing brain. Laboratory piglet experiments showed cellular, structural, and behavioral improvements after this delivery method, and a phase 1 clinical protocol (MeDCaP) was launched to test the approach in infants. The renewed work focuses on understanding how the cells and their exosomes affect white matter and the brain's subventricular zone so the treatment can be optimized. The goal is to make the cell therapy safer and more effective for babies who need open-heart surgery.
Why it matters
Potential benefit: If successful, this approach could reduce developmental delays and improve brain growth and long-term neurobehavioral outcomes in children with congenital heart disease.
How similar studies have gone: Preclinical piglet studies and an early phase 1 trial (MeDCaP) have shown promising safety signals and preliminary benefits, but larger and mechanism-focused studies remain needed.
Where this research is happening
-
Boston University Medical Campus
Boston, United States Active
Researchers
- Principal investigator: Ishibashi, Nobuyuki — Boston University Medical Campus
- Study coordinator: Ishibashi, Nobuyuki
Related research
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Multimodal AI to improve genetic diagnosis of congenital heart disease
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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.