Therapeutic use of stem cells in congenital anomalies: A pilot study

Introduction: Stem cells with potential to transform into healthy cells and repair damaged cells may prove beneficial in various congenital malformations.
Aim: To explore the use of stem cells in liver cirrhosis and meningomyelocele.
Materials and Methods: During July 2005 to July 2006, stem cells were used in 27 patients; 12 with liver cirrhosis and 15 with meningomyelocele. Autologous stem cells were injected during definite surgery into hepatic artery and portal vein or hepatobiliary radicles for liver cirrhosis or spinal cord and caudal space for meningomyelocele. The pre-operative status of the patient served as control for that patient.
Results: The patients with liver cirrhosis were between 1.5 and 9 months (mean 4.12 months). Liver cirrhosis was due to extra hepatic biliary atresia (EHBA); neonatal hepatitis and choledochal cyst in 8; 2 and 2 patients, respectively. About five patients expired due to late presentation and ongoing cirrhosis. Follow up results evaluated at 3-12 months (n=7) showed absence of cholangitis (4/7); yellow stools (5/7); decreased liver firmness (3/7); improved liver functions (6/7) and improved appetite (6/7). Hepatobiliary scan was excretory in 6/7 with improved uptake in 4/7. Histopathology repeated after stem cells demonstrated comparative improvement in fibrosis in three. The meningomyelocele patients were between 0 and 1 month; 1-5 months, and 1-4 years in 5; 8 and 2 cases, respectively. 5 had history of rupture. 3 had undergone meningocele repair in past with neurological deficits. Redo surgery for tethered cord was done in 1. Follow up (3-11 months) in 14 cases showed improved power in 4 (28%), dramatic recovery in 3 (22%), and status quo in 7 (50%). One patient is still under observation.
Conclusion: Initial use of stem cells in EHBA and meningomyelocele has shown beneficial results. However, long-term evaluation with randomized-controlled trials is essential to draw further conclusions.