Stem Cell Therapy for Hypoplastic Left Heart Syndrome (HLHS)

Stem Cell Therapy Research

Sunjay Kaushal, MD, PhD, division head of Cardiovascular-Thoracic Surgery at Lurie Children's, is the Principal Investigator of a National Institute of Health (NIH) clinical trial investigating allogeneic stem cells’ (human stem cells not obtained from the patient) ability to remodel the right ventricle of the heart (ELPIS trial, NCT #03525418).   

There are typically four phases to a clinical trial. The ELPIS trial is currently in its second phase. 

• Phase 1 of the trial, which determined the safety of the stem cell therapy, is completed.  
• Phase 2, which aims to determine whether stem cells are effective, studies patients with hypoplastic left heart syndrome who are administered the stem cells during scheduled stage II bidirectional Glenn surgery. Funding for Phase 2 is supported by NIH and the Marcus Foundation. 

An additional clinical trial (CHILD trial, NCT #03406884) is also underway at Lurie Children’s involving administering the patient’s own (autologous) stem cells. Infants potentially eligible for the study are those who are waiting for a Norwood operation after diagnosis of hypoplastic left heart syndrome. Learn more about the research studies being conducted by Lurie Children's Heart Center.  

Stem Cell Therapy Research Publications 

• Stem Cell Therapy in Single-Ventricle Physiology: Recent Progress and Future Directions 
• Human Neonatal Cardiac Mesenchymal Stem Cells Demonstrates Superior Cardiac Regenerative Abilities Compared to Other Stem Cells 
• Anti-Inflammatory and Immuno-Modulatory Properties of Neonatal MSC 
• Intravenous Administration of Neonatal Mesenchymal Stem Cells Mediates Functional Recovery in a Renal Ischemia-reperfusion Injury Rodent Model 
• Neonatal mesenchymal stem cells used for anti-inflammatory and neuroprotective therapy for neonates undergoing cardiopulmonary bypass 
• Human mesenchymal stem cells attenuate hypertrophy in induced pluripotent stem cells derived cardiomyocytes from hypoplastic left heart syndrome patients 
• Stem cell therapy for hypoplastic left heart syndrome: mechanism, clinical application, and future directions 
• Regenerative medicine therapy for single ventricle congenital heart disease 
• Study design and rationale for ELPIS: A phase I/IIb randomized pilot study of allogeneic human mesenchymal stem cell injection in patients with hypoplastic left heart syndrome 

The Lurie Children's Difference

The Stem Cell Therapy Program team at Lurie Children’s, led by Dr. Kaushal, provides the potential for innovative management of hypoplastic left heart syndrome with access to stem cell therapies, world-class facilities, and state-of-the-art equipment.  

• Expert Care Team:
  • Kaushal Research Laboratory – based at the Stanley Manne Research facility on the Northwestern Campus in downtown Chicago, the research laboratory is staffed by stem cell researchers with multiple years of experience in the field, including four MD PhD-level researchers, six post-doctoral (PhD) researchers, and two technologists. 
  • Pediatric cardiologists – board certified pediatricians with subspecialty certification in Pediatric Cardiology by the American Board of Pediatrics
  • Pediatric cardiac intensivists – board certified pediatricians with subspecialty certification in Pediatric Cardiology by the American Board of Pediatrics, plus additional certification or training in pediatric critical care or pediatric cardiac intensive care
  • Pediatric cardiac anesthesia team – board certified anesthesiologists with additional certification in pediatric anesthesia by the American Board of Anesthesia and additional training in pediatric cardiac anesthesia
  • Pediatric radiologists – board certified radiologists with certificates of added qualification in pediatric radiology by the American Board of Radiology
  • Congenital cardiac surgeons – board certified in Thoracic Surgery with additional certification in Congenital Cardiac Surgery by the American Board of Thoracic Surgery
• Facilities:
  • Echocardiography laboratory certified for fetal, transthoracic and transesophageal echocardiographic studies (by Intersocietal Commission for the Accreditation of Echocardiography Laboratories - ICAEL)
  • Fully equipped cardiac diagnostic interventional catheterization laboratories
  • On-site cardiac MRI and cardiac CT angiography imaging
  • Two dedicated pediatric cardiac surgical operating rooms
  • 44-bed Regenstein Cardiac Care Unit with remote monitoring capability
  • Neurodevelopmental Clinic that provides close monitoring of our hypoplastic left heart syndrome patients

Eligible Patients

The only approved study populations are patients with hypoplastic left heart syndrome who:

• Are awaiting the Norwood procedure – CHILD study with autologous (infant’s own) stem cell administered
• Are awaiting stage II bidirectional Glenn surgery – ELPIS study with allogeneic (not patient’s own) stem cells administered

What to Expect

Screening for participation in the clinical trial is rigorous, following all NIH and Lurie Children’s Stanley Manne Children’s Research Institute Institutional Review Board guidelines for human subject research.

Informed consent to participate in the clinical trial must be given by the patient’s legal guardian(s).

If your child’s first surgery was at Lurie Children’s, your child will be closely monitored by your cardiologist. In the weekly multidisciplinary conference, where cardiologists, cardiac intensivists, cardiac surgeons, medical imagers, and cardiac anesthesiologists meet to discuss treatment options for patients, the potential for participation in the study will be discussed, and recommendations will be communicated to you at visits after the stage I procedure hospitalization.

Make an Appointment

If your infant is a patient hospitalized at or followed at Lurie Children’s, there is no need to contact the Cardiovascular-Thoracic Surgery offices for placing your child under consideration for inclusion in either the CHILD or ELPIS studies. Our review procedure at multidisciplinary conferences would have identified your child as a potential patient to include in either study, and you would be contacted directly about this. If you have questions about either study, please call the Cardiovascular-Thoracic Surgery offices 312.227.4240 to arrange for a consultation with review of records.

For those whose infants are hospitalized at or followed at a hospital other than Lurie Children’s, the following procedure(s) would apply:

• For the CHILD study: If you would like to have your infant considered for the CHILD study, when their own stem cells are administered to them at the time of a stage I Norwood procedure, call the Cardiovascular-Thoracic Surgery offices 312.227.4240 to arrange for a second opinion consultation with review of records
• For the ELPIS study: If you are calling for a second opinion after your child has had stage I surgery elsewhere for hypoplastic left heart syndrome, call the Cardiovascular-Thoracic Surgery offices 312.227.4240 to arrange for a second opinion consultation with review of records.