Study: First-ever in-utero stem cell therapy for fetal spina bifida repair is safe

A UC Davis Health team has safely performed the world’s first spina bifida treatment combining fetal surgery with stem cells, according to results from Phase 1 of an ongoing clinical trial. These results were published in The Lancet in February. “Feasibility and Safety of Cellular Therapy for In-Utero Repair of Myelomeningocele (CuRe Trial): a First-In-Human, Phase 1, Single-Arm Study,” tested whether adding a layer of human placenta-derived stem cells to standard fetal surgery could be done safely. The cells, taken from donated placentas, are designed to protect the developing spinal cord from further damage before birth.
The first six babies in the trial were monitored closely from surgery through birth.
“It paves the way for new treatment options for children with birth defects. The future is exciting for cell and gene therapy before birth.”—Diana Farmer, CuRe trial principal investigator and chair of the UC Davis Department of Surgery
Because early safety results were strong, the Food and Drug Administration and an independent monitoring board approved moving forward with the next phase. The CuRe trial is now enrolling up to 35 patients in its Phase 1/2a study; children will be followed through age 6 to evaluate long-term safety and early signs of improved movement, bladder and bowel function.
The first phase was funded by a $9 million grant from the state’s stem cell agency, the California Institute for Regenerative Medicine (CIRM). Phase 1/2a is funded by CIRM and Shriners Children’s. Co-principal investigators are Diana Farmer, chair of the UC Davis Department of Surgery, and Aijun Wang, co-director of the UC Davis Center for Surgical Bioengineering.











