World-First Stem Cell Therapy Shows Promise for Treating Spina Bifida in the Womb

A UC Davis Health research team has safely performed the world's first spina bifida treatment combining fetal surgery with stem cells, according to Phase 1 results of the CuRe Trial published in The Lancet. The groundbreaking approach involved placing a patch containing living stem cells from donated placentas directly over the fetus's exposed spinal cord during surgical repair in the womb.

Six pregnant patients carrying fetuses diagnosed with myelomeningocele — the most severe form of spina bifida — were enrolled between June 2021 and December 2022. All infants were born with intact repair sites and had no cerebrospinal fluid leak, infection, or abnormal tissue growth.

“The groundbreaking approach involved placing a patch containing living stem cells from donated placentas directly over the fetus's exposed spinal cord during surgical repair in the womb.”

Perhaps most remarkably, post-birth MRI scans confirmed that hindbrain herniation — a brain abnormality associated with spina bifida — was reversed in every case. This finding suggests the stem cells may provide neuroprotective benefits beyond what standard fetal surgery alone can achieve.

The stem cells, derived from donated placentas, are designed to protect the developing spinal cord from further damage before birth. The care team places a small biocompatible patch containing the living cells directly over the exposed neural tissue, then closes the layers of the back to allow regeneration.

Spina bifida affects approximately 1 in 2,500 births worldwide and can cause lifelong mobility challenges, bladder and bowel dysfunction, and neurological complications. While fetal surgery to repair the defect before birth has been available since the early 2000s, adding stem cells represents a significant evolution of the approach.

The FDA has approved enrollment of additional pregnant women for the next phase. Children in this trial will be monitored until age six to confirm long-term safety and assess whether the treatment improves mobility and quality of life as they grow.