THE PROMISE REGENERATIVE MEDICINE AND STEM CELL THERAPY HOLDS FOR CONGENITAL HEART DEFECTS
Authored by Dr Swati Garekar, Senior Consultant-Pediatric Cardiology, Fortis Hospital Mulund
Congenital heart defects (CHD) are the most common birth defects and can adversely impact quality of life if left untreated. Diagnosis and treatment at the correct time is the right step towards an excellent outcome. Since the first successful open-heart surgery on September 2, 1952, done on a 3-year-old boy with a ventricular septal defect (VSD) at the University of Minnesota, significant advances have been achieved.
However, challenges remain with implantation of valves or conduits for replacing defective or missing parts of the heart. These replacements have several drawbacks such as the need for lifetime anticoagulants, risk of endocarditis, calcification over time, and size mismatch as the child grows.
Another challenge lies with complex heart defects that require staged surgeries; the long-term concern is the pumping ability of the heart’s ventricle. Can regenerative medicine and stem cell therapy help?
Regenerative medicine endeavors include tissue engineering — applying tissue-engineered conduits, valves, and patches, including chemically treated homografts and xenografts. The future hints at scaffolds acting as temporary structures that provide a ‘bed’ for native tissue growth, eventually absorbed by the body.
Stem cell therapy hopes to enable repair and regeneration of heart parts. Stem cells, derived from umbilical cord, bone marrow, or heart biopsy, can be injected into the coronaries or myocardium.
Numerous clinical trials are ongoing internationally, including the TICAP trial (trans coronary infusion of cardiac progenitor cells in single ventricle patients) and the PERSEUS trial for univentricular heart disease. These studies indicate safety, feasibility, and some improvement in ventricular function and growth.
India's regulations via ICMR provide guidelines for ethical stem cell research and evidence-based uses. While stem cell therapy is standard for blood cancers and applying CAR-T therapy for certain lymphomas is recent, usage in CHD remains limited to clinical trials, with none ongoing currently in India.