The HEAL-CHF trial — the world's largest randomized controlled trial of iPSC-derived cardiomyocyte therapy for severe heart failure — demonstrates that cell therapy can repair damaged myocardium in patients with advanced heart failure.
• Published in Nature Medicine (2026)
• 25 patients enrolled in the world's largest iPSC-cardiomyocyte RCT for heart failure
• Global first — largest population of human iPSC-cardiomyocyte transplantation
• Proven safety and efficacy — enhanced cardiac function and quality of life
• Multicenter trial — led by Chinese researchers with international collaboration
Introduction
Heart failure remains a leading cause of morbidity and mortality worldwide, with an estimated 64 million people affected globally. Despite advances in pharmacological and device-based therapies, the prognosis for patients with advanced heart failure remains poor, with 5-year mortality rates exceeding 50%.
Cardiomyocytes have limited regenerative capacity, and the heart cannot repair itself after significant injury. This has driven interest in cell-based therapies that could regenerate damaged myocardium.
In 2026, the HEAL-CHF (Human Embryonic stem cell-derived cardiomyocyte therapy for ischEmic heArt faiLure) trial — the world's largest randomized controlled trial of iPSC-derived cardiomyocyte therapy — published its landmark results in Nature Medicine.
Trial Design and Participants
The HEAL-CHF trial was a multicenter, randomized, open-label, controlled study conducted across multiple centers in China with international academic collaboration.
| Parameter | Details |
|---|---|
| Number of patients | 25 patients with severe ischemic heart failure |
| Intervention | iPSC-derived cardiomyocyte patches transplanted into the infarcted myocardium |
| Primary endpoint | Change in left ventricular ejection fraction (LVEF) at 6 months |
| Secondary endpoints | 6-minute walk test, quality of life, safety |
Key Results
The trial demonstrated that iPSC-derived cardiomyocyte therapy is both safe and effective in treating severe heart failure:
- Cardiac function — significant improvement in LVEF in treated patients compared to controls
- Exercise capacity — 6-minute walk distance increased substantially
- Quality of life — Minnesota Living with Heart Failure Questionnaire scores improved significantly
- Safety — no tumorigenicity or severe arrhythmias were observed in the transplanted patients
The study represents the largest cohort of patients ever transplanted with human iPSC-derived cardiomyocytes, providing critical evidence for the safety and efficacy of this regenerative approach.
Significance
This trial marks a major milestone in cardiovascular regenerative medicine:
- Proof of concept — demonstrates that iPSC-derived cardiomyocyte transplantation can improve cardiac function in advanced heart failure
- Safety validation — the largest safety dataset for iPSC-derived cardiomyocyte therapy to date
- Regenerative potential — offers a new paradigm beyond symptom management and device therapy
- Global leadership — positions China at the forefront of cardiac regenerative medicine