Stem cells in utero for spina bifida improve quality of life in small trial

A small US phase one trial applied placenta-derived mesenchymal stem cells to the exposed spinal cord during fetal surgery in six fetuses with myelomeningocele and reported safety and reversal of hindbrain herniation on postnatal MRI.

Researchers in the US reported early results from a phase one trial testing placenta-derived mesenchymal stem cells applied during fetal surgery for spina bifida. The study involved six fetuses with myelomeningocele who underwent repair at 24–25 weeks' gestation between 2021 and 2022. Postnatal MRI scans showed reversal of hindbrain herniation in all six infants, and the trial team reported no tumours, abnormal tissue growth, infections, or interference with wound healing. The lead investigator said it was conceivable that, if further studies confirm benefits, adding stem cells to fetal repair could become part of standard care for some cases. Key facts: - The trial took place in California and enrolled six patients between July 2021 and December 2022. - Surgeons applied placenta-derived mesenchymal stem cells directly to the exposed fetal spinal cord during in utero repair for myelomeningocele. - MRI after birth indicated reversal of hindbrain herniation in all six infants. - Reported safety outcomes included no tumours, no abnormal tissue growth, no infections, and no observed interference with wound healing. - The children will be followed up at intervals until age six, and enrolment has begun for a larger study of 35 patients. Summary: The phase one trial reported anatomical improvement and a reassuring safety profile in a small group, and a linked expert comment described the findings as potentially important for the future of fetal surgery. Further research is under way, with planned long-term follow-up of the trial children and a larger study now enrolling.