A groundbreaking trial in the United States has demonstrated that administering stem cells to unborn babies diagnosed with spina bifida during in utero surgery could represent a significant advancement in the management of birth defects. The study, which involved six fetuses with myelomeningocele, the more severe form of spina bifida, found that applying stem cells derived from the mother's placenta to the baby's spine while it was being repaired was not only safe but also led to improved mobility and enhanced quality of life for the children.
Safety and Efficacy of the Experimental Therapy
Conducted in 2021 and 2022 and reported in the Lancet, the trial focused on babies at 24 to 25 weeks of gestation. Surgeons from the Davis School of Medicine at the University of California applied placenta-derived mesenchymal stem cells directly onto the exposed spinal cord during the surgical repair process. Post-birth MRI scans confirmed that hindbrain herniation, a common brain abnormality associated with spina bifida where the lower part of the brain shifts into the neck area, was reversed in all six cases. Importantly, the stem cells did not cause tumors, interfere with wound healing, or prevent the reversal of hindbrain herniation, addressing potential side-effects that researchers had anticipated.
Expert Reactions and Future Implications
Dr. Diana Farmer, who led the study, expressed enthusiasm about the results, stating that it is "conceivable" this experimental therapy could become the standard of care for treating spina bifida before birth. She emphasized, "If it makes more children able to walk who wouldn't have then it would become their standard of care. The results are super exciting and pave the way for lots of potential new treatments for spina bifida and other birth defects." Kate Steele, CEO of the charity Shine, echoed this sentiment, calling the findings "very exciting" and highlighting the potential for improvements in bladder and bowel function, which are critical for quality of life.
In a linked comment piece in the Lancet, Dr. Magdalena Sanz Cortes, a fetal medicine expert in Texas, noted that if further studies confirm these benefits, it could herald a new era in fetal surgery, potentially reversing neurological damage seen in utero for spina bifida. With an estimated 8,100 to 11,900 people in England and Wales living with open spina bifida and about 536 pregnancies affected annually, this therapy offers hope for better outcomes and enhanced mobility for future generations.
