Cell-based brain protection for infants with congenital heart defects
Cell Therapy for Neuroprotection in Congenital Heart Disease
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 ID | NIH-11375931 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates are infants and young children with congenital heart defects who will undergo cardiac surgery requiring cardiopulmonary bypass.
Not a fit: 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.
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 performed: 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, United States
- Boston University Medical Campus — Boston, United States (Active)
Researchers
- Principal investigator: Ishibashi, Nobuyuki — Boston University Medical Campus
- Study coordinator: Ishibashi, Nobuyuki
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.