A UC Davis Health research 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 have been published today in The Lancet.
The study, called “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. Spina bifida, also known as myelomeningocele, occurs when spinal tissue fails to fuse properly during the early stages of pregnancy. The birth defect can lead to a range of lifelong cognitive, mobility, urinary and bowel disabilities.
“Putting stem cells into a growing fetus was a total unknown. We are excited to report great safety,” said Diana Farmer, the CuRe Trial’s principal investigator and chair of the UC Davis Department of Surgery. “It paves the way for new treatment options for children with birth defects. The future is exciting for cell and gene therapy before birth.”
The first phase of the trial was funded by a $9 million state grant from the state’s stem cell agency, the California Institute for Regenerative Medicine (CIRM).
About the treatment
During fetal surgery, a small opening is made in the uterus. The surgeons then float the fetus up to that incision point so they can expose the back of the fetus and the spina bifida defect. The care team places a small patch containing living stem cells directly over the fetus’s exposed spinal cord, before closing the layers of the back to allow the tissue to regenerate. The stem cells, taken from donated placentas, are designed to protect the developing spinal cord from further damage before birth.
It is the world’s first in-utero stem cell therapy for spina bifida, and the only trial aimed at improving the outcomes for the children over fetal surgery alone.
Key Phase 1 findings
The first six babies in the trial were monitored closely from surgery through birth. Researchers reported:
No safety concerns related to the stem cells.
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