Researchers believe that stem cell therapy may open new doors for treating specific birth defects. Initial findings released recently indicate that donor stem cells, administered in the womb, could benefit children with spina bifida.
The University of California, Davis, carried out the first-ever in-utero trial of stem cell therapy for this condition. They discovered that this treatment, when combined with prenatal surgery, can be applied safely, with no negative effects observed in the babies after birth. Interestingly, the stem cells might have enhanced the efficacy of the surgery, ultimately improving the children’s quality of life.
“Introducing stem cells to a developing fetus was entirely uncharted territory. We’re thrilled to report great safety,” remarked Diane Farmer, who leads the study and is the chair of the UC Davis Department of Surgery.
New hope for a challenging condition
Spina bifida is classified as a neural tube defect, where a fetus does not properly form and close its spine and spinal cord. Its severity can differ, depending on how much of the spine is exposed. Unfortunately, it’s frequently a serious condition, resulting in lifelong challenges related to cognition, bladder control, and mobility.
Typically, treating spina bifida involved surgery to close exposed spinal tissue shortly after birth. More recently, prenatal fetal surgery has emerged as the standard procedure, leading to better outcomes. However, many children continue to face significant health issues, such as an inability to walk unaided, even after this early surgical intervention.
Stem cells have sparked considerable scientific interest for their potential to enhance the body’s natural healing processes. This curiosity prompted researchers at UC Davis and elsewhere to explore if they could further improve prenatal surgery results for spina bifida. Prior animal studies seemed to support their hypothesis, serving as a foundation for this ongoing phase I CuRe trial.
This trial involved six pregnant women whose fetuses were diagnosed with spina bifida from 2021 to 2022. Each fetus underwent the usual spinal repair surgery. However, before completing the surgery, the team applied a stem cell patch—taken from donor placentas—onto the fetuses’ exposed spinal tissue.
Phase I trials primarily focus on assessing the safety of new treatments, and this one achieved that goal. No complications typically associated with stem cell therapy, like tumor growth, were reported. All surgeries proceeded smoothly, with no infections and complete healing observed in the fetuses.
While it’s still preliminary, it appears the stem cells might have contributed to better surgical results. MRI scans indicated that all newborns experienced a reversal of hindbrain herniation, an encouraging sign. Additionally, none of the children required a shunt for hydrocephalus (a build-up of fluid in the brain) before being discharged, which is often a common issue for spina bifida patients.
The findings from this study were published recently in the Lancet.
What’s next?
These findings are still in the early stages. Further research is needed to determine if this stem cell therapy has truly enhanced the long-term health of these children, or whether it can reliably benefit others diagnosed with spina bifida.
Despite the uncertainty, there’s significant optimism about this approach—not only for spina bifida but for other conditions that develop in fetuses. “It opens up new therapeutic avenues for children with birth defects. The future looks bright for cell and gene therapy initiated before birth,” Farmer noted.
The research team plans to monitor the long-term health of the original six children involved in the study. They are also preparing to initiate a larger Phase I/IIa trial involving 35 children.
