A groundbreaking clinical trial has demonstrated that administering stem cells to unborn babies undergoing surgery for spina bifida is both safe and significantly improves neurological outcomes. The study, conducted in the United States and published in The Lancet, suggests a potential shift in how severe birth defects are treated before birth.
Trial Findings and Methodology
Researchers led by Dr. Diana Farmer at the University of California, Davis, applied stem cells derived from the mothers’ placentas directly to the exposed spinal cords of six fetuses diagnosed with myelomeningocele – the most severe form of spina bifida – between 24 and 25 weeks gestation. Magnetic resonance imaging (MRI) scans conducted after birth revealed complete reversal of hindbrain herniation —a dangerous neurological complication often associated with this condition—in all six participants.
The trial was designed to assess safety, and crucially, no tumors developed, wound healing was not compromised, and hindbrain herniation was consistently corrected. This addresses major concerns around fetal interventions, where unintended side effects can be devastating.
Why This Matters: A Turning Point in Fetal Surgery
Spina bifida affects an estimated 8,100-11,900 people in England and Wales alone, with roughly 536 pregnancies per year resulting in the condition. Current surgical repair of myelomeningocele is effective, but stem cell augmentation could dramatically improve long-term functional outcomes.
“If it makes more children able to walk who wouldn’t have then it would become their standard of care,” says Dr. Farmer, highlighting the potential for widespread adoption if further studies confirm these findings.
The key benefit lies in the regenerative properties of mesenchymal stem cells, which may facilitate faster and more complete spinal cord repair. Improved bladder and bowel function are additional potential advantages, given the often debilitating complications of spina bifida.
Future Implications and Next Steps
The success of this trial is drawing cautious optimism from the medical community. Dr. Magdalena Sanz Cortes, a fetal medicine expert not involved in the study, suggests that if replicated in larger trials, this approach could herald “a new era in fetal surgery.”
While preliminary, these results offer a compelling case for expanding stem cell therapies into prenatal interventions for other birth defects. Further research is needed to refine the technique and confirm long-term safety and efficacy, but this study marks a significant leap toward more effective and less invasive treatments for severe congenital conditions.
The possibility of reversing neurological damage in utero is a transformative prospect, offering hope for improved quality of life for children born with spina bifida and potentially opening doors to similar interventions for other devastating birth defects.
