Researchers at UC Davis Health have safely performed the world's first in-utero stem cell therapy for spina bifida, combining fetal surgery with placental-derived stem cells. The Phase 1 trial results, published in The Lancet, demonstrated that adding a stem cell patch to standard fetal surgery could be done without safety concerns. The treatment aims to improve outcomes for children born with this neural tube defect.

During the procedure, surgeons make a small uterine incision and place a patch containing living stem cells directly over the exposed spinal cord. The stem cells, harvested from donated placentas, are designed to protect the developing spinal cord before birth. The trial received $9 million in funding from California's stem cell agency, CIRM, and is now moving to Phase 1/2a with up to 35 patients.

Early results showed no safety concerns related to the stem cells, with no infections, spinal fluid leaks, or abnormal tissue growth. All six infants showed reversal of hindbrain herniation on MRI scans, and none required shunts for hydrocephalus before discharge. The FDA and an independent monitoring board have approved advancing to the next phase. Researchers will follow children through age 6 to evaluate long-term safety and early signs of improved mobility, bladder, and bowel function.