Stem Cell Therapy for HLHS – First Pivotal Trial
Stem Cell Therapy for HLHS – First Pivotal Trial
Source: Medic News , Sept 09, 2025
1. HLHS: Hypoplastic Left Heart Syndrome is a rare congenital defect where the left side of the heart is underdeveloped, forcing the weaker right ventricle to pump for the whole body, and is fatal without staged surgery in early life.; standard 3-stage surgeries improve survival but only ~50% reach age 15 without transplant.
2. New Therapy: Lomecel-B (stem cell injection) aims to strengthen the right ventricle and reduce heart failure/transplant.
3. Trial (ELPIS II): 40 infants, 12 U.S. centers, randomized during stage-2 surgery to surgery alone vs surgery + Lomecel-B.
4. Early Results: In first 10 treated, all alive and transplant-free at 5 years; no safety issues.
5. Next Step: Pivotal trial results expected after 1 year, with follow-up up to 5 years.
Takeaway : This is the first major U.S. trial of stem cell therapy for HLHS, offering hope to extend survival beyond current limits.
https://clinicaltrials.gov/ct2/show/NCT04925024
