BioVAT Cardiac Muscle Patches Show Promise in Advanced Heart Failure: NEJM
Germany: Interim findings from an early-stage study suggest that BioVAT, a tissue-engineered cardiac muscle patch therapy, may help remuscularize the heart in patients with advanced heart failure. Surgically implanted BioVAT patches were associated with increased heart wall thickness, improved left ventricular ejection fraction, and better quality of life.
BioVAT consists of thin engineered cardiac muscle patches attached to damaged areas of the ventricle where cardiomyocytes have been lost. Researchers noted that while the initial results are encouraging, larger studies and longer follow-up are needed to confirm efficacy and assess potential risks such as electrical dysfunction or arrhythmias.
The findings are from a phase 1–2 open-label clinical study published in the New England Journal of Medicine and led by Wolfram-Hubertus Zimmermann and colleagues in Germany. The investigational therapy, termed biologic ventricular assist tissue (BioVAT), is designed to restore contractile function in patients with severe heart failure by implanting lab-grown cardiac muscle tissue.
BioVAT is created using engineered heart muscle composed of cardiomyocytes and supporting stromal cells derived from allogeneic induced pluripotent stem cells. The therapy is surgically implanted onto weakened or non-contracting regions of the left ventricle to rebuild functional myocardial tissue in areas where heart muscle cells have been lost.
The study enrolled 20 patients with heart failure and a reduced left ventricular ejection fraction of 35% or less, along with at least one segment of the heart showing reduced or absent movement. Patients received BioVAT grafts containing 5, 10, or 20 engineered-heart-muscle units, and all participants were placed on immunosuppressive therapy to reduce the risk of graft rejection.
Key Findings:Top of Form
- All participants experienced at least one adverse event during the follow-up period.
- Serious adverse outcomes included three deaths, attributed to vasoplegia, COVID-19 infection, and aortic dissection.
- One patient required heart transplantation during the study period.
- Immunosuppressive therapy was discontinued in four patients due to complications, including left ventricular assist device implantation, renal failure, and urothelial carcinoma.
- Efficacy analysis in the subgroup receiving the highest dose (20 engineered-heart-muscle units) included 12 patients who completed the 3-month interim assessment.
- These patients showed a significant increase in target heart-wall thickness, with a mean improvement of 4.5 mm.
- Left ventricular ejection fraction improved by 3.9 percentage points, indicating better cardiac pumping function.
- Quality-of-life scores on the Kansas City Cardiomyopathy Questionnaire increased, although the improvement did not reach strong statistical significance.
The investigators emphasized that these early findings provide initial evidence that cardiac remuscularization using engineered tissue is biologically feasible in humans with advanced heart failure. However, they cautioned that the study is limited by its small size, short follow-up period, and lack of a control group.
Overall, the results suggest that BioVAT therapy may represent a novel regenerative approach for end-stage heart failure, but further large-scale and long-term studies are essential to determine the durability of benefit, safety, and clinical applicability.
Reference:
DOI: 10.1056/NEJMoa2513525
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