A groundbreaking new treatment offers hope for infants diagnosed with spina bifida, a serious birth defect affecting the spinal cord. Researchers have safely applied stem cells to the exposed spinal cords of six fetuses in utero, marking a significant milestone in the quest to improve outcomes for those living with this condition. The early results, published this week, suggest the therapy is feasible and does not cause immediate harm to mother or child, though long-term effects remain to be seen.
Spina bifida, affecting roughly 1 in 1,000 births globally, occurs when the spinal cord doesn’t fully close during fetal development. The most severe form, myelomeningocele, can lead to a range of disabilities, including paralysis, bladder and bowel control issues, and fluid buildup in the brain. Whereas folic acid supplementation during pregnancy can significantly reduce the risk, spina bifida still impacts approximately half a million babies worldwide each year, according to the National Institute of Neurological Disorders, and Stroke.
A Novel Approach to Fetal Repair
The clinical trial, led by fetal and neonatal surgeon Diana Farmer at the University of California, Davis, utilized placenta-derived stem cells. These cells were applied directly to the exposed spinal cords of the fetuses during surgery performed between 24 and 25 weeks of gestation. The Cellular Therapy for In Utero Repair of Myelomeningocele (CuRE) trial aimed to determine if the treatment was safe and well-tolerated. According to a UC Davis Health news release, the surgeries were completed without complications, and the newborns were delivered around 34 weeks gestation without signs of infection, cerebrospinal fluid leakage, or tumor growth.
An in utero treatment using placenta-derived stem cells could treat infants with a neural-tube condition called spina bifida. Credit: Simon Fraser/Science Photo Library
Importantly, all of the newborns showed signs of reversal of hindbrain herniation, a complication of spina bifida where the back of the brain descends into the neck, potentially blocking cerebrospinal fluid circulation. This suggests the stem cell therapy may have a protective effect on brain development, though further investigation is crucial.
Building on Years of Research
This breakthrough builds upon decades of research into fetal surgery and stem cell therapies. Farmer previously led a trial demonstrating the benefits of in utero surgery to close the spinal cord defect, but noted that nearly 60% of children still experience limitations in walking or independent movement following the procedure. This represents often due to nerve damage caused by exposure to amniotic fluid before the defect is closed. The hope is that the addition of stem cells can mitigate this damage and improve functional outcomes.
The team’s work with stem cells began after Shinya Yamanaka was awarded the Nobel Prize in 2012 for his work on induced pluripotent stem cells. While those cells proved unsuitable for this application, Farmer’s team found success using stem cells derived from the placenta, first in laboratory studies and then in animal models. Sheep that received both surgery and stem cell treatment were able to stand and walk independently, unlike those who received surgery alone.
Diana Farmer (right), a fetal and neonatal surgeon at the University of California, Davis, led the Cellular Therapy for In Utero Repair of Myelomeningocele (CuRE) trial. Credit: UC Davis
Cautious Optimism and Next Steps
While the initial safety data is encouraging, experts emphasize the need for larger, controlled trials to confirm the efficacy of this stem-cell therapy for spina bifida. Clare Whitehead, a fetal-medicine specialist at the Royal Women’s Hospital in Melbourne, Australia, cautioned that “it is still too early to know whether it will be beneficial.” She added, “With fetal therapy, we have to be very cautious,” noting that positive results in small studies don’t always translate to broader populations.
The researchers are now planning expanded trials to assess the long-term impact of the treatment on motor function, cognitive development, and overall quality of life for children born with spina bifida. The team will continue to monitor the initial six participants, and recruitment for the next phase of the CuRE trial is expected to commence in the coming months. Updates on the trial’s progress will be available through UC Davis Health.
Disclaimer: This article provides information for general knowledge and informational purposes only, and does not constitute medical advice. It is essential to consult with a qualified healthcare professional for any health concerns or before making any decisions related to your health or treatment.
This innovative approach to treating spina bifida represents a significant step forward in fetal medicine. As research continues, it offers a glimmer of hope for improved outcomes and a better future for families affected by this challenging condition. Share this article to spread awareness about this promising new treatment.
