Health and Me

Rachel V Thomas

1st-Ever In-Womb Stem Cell Therapy for Spina Bifida Found Safe, Gets US FDA Backing: The Lancet

In a medical milestone, a team of US surgeons has deemed an in-utero surgery that adds <span>stem cells</span> to treat <span>spina bifida</span> -- a birth defect -- to be safe. This new type of fetal therapy does not just repair the defect but may also help heal and protect the baby’s developing spinal cord. 

The Phase 1 <span>clinical trial</span>, published today in <span>The Lancet</span> and based on six babies, showed that adding a layer of human placenta-derived stem cells to standard fetal surgery can be done safely. The early safety results have prompted the US Food and Drug Administration (<span>FDA</span>), along with an independent monitoring board, to approve the next phase of the study. 

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. 

While surgeons have previously performed prenatal surgeries, this is the world’s first in-utero <span>stem cell therapy</span> for spina bifida. It is also the only clinical trial aimed at improving outcomes beyond those achieved with fetal surgery alone. 

“Putting stem cells into a growing fetus was a total unknown. We are excited to report strong safety results,” said Diana Farmer, the clinical trial’s principal investigator and chair of the Department of Surgery at the University of California-Davis Health. 

“It paves the way for new treatment options for children with <span>birth defects</span>. The future is exciting for cell and gene therapy before birth,” she added. 

<h2>How The Breakthrough Fetal Surgery Was Conducted </h2>

Surgeons made a small opening in the mother’s uterus and gently positioned the fetus to expose its back and the spina bifida defect. 

A small patch containing living stem cells was then placed directly over the 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. 

<h2>Treating Spina Bifida Before Birth </h2>

The findings from the first six babies in the trial, who were closely monitored from surgery through birth, revealed no safety concerns related to the stem cells. 

After surgery, the babies experienced no infections or spinal fluid leaks. No abnormal tissue growth or tumors formed at the repair site. 

All six surgeries were successful, and the stem cell patch was placed as planned in every case. All surgical wounds healed completely. 

MRI scans also showed reversal of hindbrain herniation -- a condition commonly associated with spina bifida -- in all infants, an indicator of surgical success. 

Hindbrain herniation in babies, often referred to as Arnold-Chiari II malformation, involves the lower part of the brain descending into the cervical spinal canal. 

This can block the flow of cerebrospinal fluid, causing dangerous hydrocephalus (fluid buildup). None of the babies required a shunt for hydrocephalus before hospital discharge, the researchers said. 

Spina bifida affects about 1 in 2,500 births worldwide, with an estimated 1,500 to 2,000 babies diagnosed each year in the United States alone. 

It is a common neural tube defect, with higher prevalence observed in regions with lower folic acid fortification. Open spina bifida specifically occurs in approximately 1 in every 2,875 births. 

This surgical intervention aims to significantly improve motor function, and increase the likelihood of walking independently. 

The first phase of the trial was funded by a $9 million state grant from the California Institute for Regenerative Medicine (CIRM). 

The trial is now enrolling up to 35 patients in its Phase 1/2a study. 

Children will be followed through age six to evaluate long-term safety and early signs of improved movement, as well as bladder and bowel function.

Health Conditions A-Z

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Spina Bifida

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The Lancet

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birth defects

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cognitive disability

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In a medical milestone, a team of US surgeons has deemed an in-utero surgery that adds stem cells to treat spina bifida -- a birth defect -- to be safe. This new type of fetal therapy does not just repair the defect but may also help heal and protect the baby’s developing spinal cord. The Phase 1 clinical trial, published today in The Lancet and based on six babies, showed that adding a layer of human placenta-derived stem cells to standard fetal surgery can be done safely. The early safety results have prompted the US Food and Drug Administration (FDA), along with an independent monitoring board, to approve the next phase of the study. 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. While surgeons have previously performed prenatal surgeries, this is the world’s first in-utero stem cell therapy for spina bifida. It is also the only clinical trial aimed at improving outcomes beyond those achieved with fetal surgery alone. “Putting stem cells into a growing fetus was a total unknown. We are excited to report strong safety results,” said Diana Farmer, the clinical trial’s principal investigator and chair of the Department of Surgery at the University of California-Davis Health. “It paves the way for new treatment options for children with birth defects. The future is exciting for cell and gene therapy before birth,” she added. How The Breakthrough Fetal Surgery Was Conducted Surgeons made a small opening in the mother’s uterus and gently positioned the fetus to expose its back and the spina bifida defect. A small patch containing living stem cells was then placed directly over the 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. Treating Spina Bifida Before Birth The findings from the first six babies in the trial, who were closely monitored from surgery through birth, revealed no safety concerns related to the stem cells. After surgery, the babies experienced no infections or spinal fluid leaks. No abnormal tissue growth or tumors formed at the repair site. All six surgeries were successful, and the stem cell patch was placed as planned in every case. All surgical wounds healed completely. MRI scans also showed reversal of hindbrain herniation -- a condition commonly associated with spina bifida -- in all infants, an indicator of surgical success. Hindbrain herniation in babies, often referred to as Arnold-Chiari II malformation, involves the lower part of the brain descending into the cervical spinal canal. This can block the flow of cerebrospinal fluid, causing dangerous hydrocephalus (fluid buildup). None of the babies required a shunt for hydrocephalus before hospital discharge, the researchers said. What Is Spina Bifida Spina bifida affects about 1 in 2,500 births worldwide, with an estimated 1,500 to 2,000 babies diagnosed each year in the United States alone. It is a common neural tube defect, with higher prevalence observed in regions with lower folic acid fortification. Open spina bifida specifically occurs in approximately 1 in every 2,875 births. This surgical intervention aims to significantly improve motor function, and increase the likelihood of walking independently. The first phase of the trial was funded by a $9 million state grant from the California Institute for Regenerative Medicine (CIRM). The trial is now enrolling up to 35 patients in its Phase 1/2a study. Children will be followed through age six to evaluate long-term safety and early signs of improved movement, as well as bladder and bowel function.