Cardiac Progenitor Cell Therapy for Heart Repair — Regenerative Therapies
Heart-derived progenitor cells and cardiosphere-derived cells (CDCs) for myocardial regeneration after infarction and in cardiomyopathy.
Overview
Cardiac Progenitor Cell (CPC) therapy encompasses several approaches to regenerate damaged myocardium, primarily using cardiosphere-derived cells (CDCs) and c-kit+ cardiac progenitor cells. CDCs, pioneered by Eduardo Marban at Cedars-Sinai, are harvested from endomyocardial biopsy, grown as self-assembling cardiospheres, and expanded as CDCs. The CADUCEUS trial (2012) demonstrated that intracoronary CDC infusion reduced scar size by 42% at 12 months post-MI. The ALLSTAR trial, a larger Phase 2 study, showed safety but did not meet its primary endpoint of scar reduction. The c-kit+ cardiac stem cell field was severely damaged by the HARVARD/Piero Anversa scandal - 31 papers retracted for fabrication, and Brigham and Women's Hospital paid $10 million to settle.
Indications
- Post-myocardial infarction (scar reduction and remodeling)
- Ischemic cardiomyopathy with reduced ejection fraction
- Duchenne muscular dystrophy cardiomyopathy (HOPE-Duchenne trial)
- Heart failure with reduced ejection fraction (HFrEF)
- Non-ischemic dilated cardiomyopathy (investigational)
Mechanism of Action
Endomyocardial biopsy tissue explanted and cultured. Cardiac progenitors migrate out and form self-assembling cardiospheres - 3D clusters containing c-kit+ cells, MSC-like cells, and cardiac progenitors
Dosing
| Compound | Dose | Frequency | Notes |
|---|---|---|---|
| Cardiosphere-Derived Cells (CDCs) | 25 million CDCs | Single infusion 1.5-3 months post-MI | Intracoronary infusion via balloon catheter (CADUCEUS trial) |
| CAP-1002 (Allogeneic CDCs) | 150 million allogeneic CDCs | Single infusion, may repeat quarterly | CAP-1002 for DMD cardiomyopathy (HOPE-Duchenne) |
Evidence Grade
GRADE C
Safety & Contraindications
- Intracoronary delivery requires cardiac catheterization
- Risk of coronary microembolization and slow flow/no-reflow phenomenon
- Endomyocardial biopsy for autologous harvest carries perforation risk
- c-kit+ cardiac stem cell field tainted by Anversa data fabrication scandal
- Allogeneic CDCs may trigger immune response
- Arrhythmia risk from cell transplantation into scarred myocardium
- ALLSTAR Phase 2 trial failed primary endpoint - efficacy uncertain
- Long-term durability of scar reduction benefits unclear
- Limited to specialized cardiac centers with interventional capabilities