A groundbreaking trial has shown that a stem cell-based patch, applied during fetal surgery, effectively reverses brain damage associated with severe spina bifida. The new approach demonstrates at least comparable efficacy to standard treatment while promising long-term improvements in mobility.
The Challenge of Spina Bifida
Spina bifida affects roughly 1 in every 2800 births in the US, where the fetal spine doesn’t fully develop, causing a severe form called myelomeningocele. This condition can result in spinal tissue protruding through an opening in the vertebrae, impairing movement and bladder/bowel control. While the exact cause remains unknown, folic acid deficiency during pregnancy is a known risk factor.
The standard treatment involves in-womb surgery to reposition the spinal cord and seal the opening. However, many children still require wheelchairs, and bowel/bladder issues remain common. This prompted researchers to explore whether stem cells could enhance spinal tissue repair and growth.
The Stem Cell Patch Approach
A team led by Diana Farmer at UC Davis conducted a trial with six pregnant women carrying fetuses diagnosed with myelomeningocele. All fetuses developed hindbrain herniation, a complication where excessive fluid buildup pushes part of the brain through the base of the skull. Conventional surgery often alleviates this, but not always completely.
The trial involved standard surgery plus a patch containing stem cells derived from donated placentas embedded in a protein matrix. The patch was applied to the spine before closing the incision. According to Farmer, “The cells secrete their magic stem cell juice,” encouraging tissue regeneration.
Promising Results
The surgery sites healed normally in all babies, with no signs of uncontrolled cell growth. Crucially, MRI scans revealed complete reversal of hindbrain herniation in every case. One mother reported her 4-year-old son, initially expected to need a wheelchair, now walks, runs, and has full bladder control—an uncommon outcome with standard treatment.
Next Steps and Expert Commentary
Researchers are now launching a larger trial with 35 fetuses to compare the stem cell patch against conventional surgery. Panicos Shangaris from King’s College London believes the patch will yield superior long-term results based on existing animal studies. A head-to-head trial, randomly assigning fetuses to each intervention, would provide the strongest evidence for regulatory approval.
This innovation represents a significant step forward in treating spina bifida, potentially improving the lives of thousands of children born with this condition annually. The ability to reverse brain damage in utero opens new possibilities for long-term functional outcomes.
