Stem Cell Breakthrough Offers Hope for Babies with Spina Bifida
A groundbreaking clinical trial has demonstrated the safety of applying stem cells to the spinal cords of fetuses in utero, offering a potential new treatment avenue for spina bifida, a serious birth defect. The research, led by Dr. Diana Farmer at the University of California, Davis, marks a significant step forward in fetal surgery and regenerative medicine.
Understanding Spina Bifida and Myelomeningocele
Spina bifida, affecting fewer than 1 in 1,000 births globally, occurs when the spinal cord doesn’t fully close during gestation. The most severe form, myelomeningocele, can lead to a range of lifelong disabilities, including paralysis, bladder and bowel control issues, and excess fluid accumulation in the brain. While folic acid supplementation can help prevent some cases, approximately half a million babies worldwide are still affected each year.
The CuRE Trial: A First-of-Its-Kind Approach
The Cellular Therapy for In Utero Repair of Myelomeningocele (CuRE) trial involved six pregnant women who underwent fetal surgery between 24 and 25 weeks of gestation. During the procedure, surgeons applied stem cells derived from donated placentas directly to the exposed spinal cords of the fetuses. The study aimed to determine if this approach was feasible and, crucially, safe for both mother, and child.
Positive Early Results: Safety and Hindbrain Herniation Reversal
The initial findings, published in The Lancet, are encouraging. There were no complications during the surgeries, and the infants were delivered at around 34 weeks without signs of infection, cerebrospinal fluid leakage, or tumor growth. Notably, all newborns showed reversal of hindbrain herniation, a complication associated with spina bifida where the back of the brain descends into the neck.
How Stem Cells Could Improve Outcomes
Current fetal surgery can close the spinal cord defect, but nearly 60% of children still experience difficulty walking or moving independently. This is given that the surgery doesn’t reverse the damage to neurons caused by exposure to amniotic fluid. Researchers believe that placenta-derived stem cells can protect the developing spinal cord from further damage before birth.
From Nobel Prize to Placental Stem Cells
Dr. Farmer’s team began exploring stem cell therapies after Shinya Yamanaka’s Nobel Prize-winning work on induced pluripotent stem cells in 2012. While initial attempts with induced pluripotent stem cells were unsuccessful, they found success using stem cells derived from the placenta, demonstrating positive results in both cell-based studies and sheep models. Sheep treated with stem cells alongside surgery were able to stand and walk independently, unlike the control group.
Cautious Optimism and Future Directions
Experts emphasize the need for caution, noting that positive results from small trials don’t always translate to larger populations. Fetal medicine specialist Clare Whitehead of the Royal Women’s Hospital in Melbourne, Australia, highlights the importance of continued research. However, the CuRE trial represents a significant advancement, paving the way for potential new treatments for spina bifida and other birth defects.
What’s Next for In Utero Stem Cell Therapy?
The success of the CuRE trial opens doors for further research and development in the field of in utero stem cell therapy. Future studies will focus on:
- Expanding the trial to include a larger cohort of patients.
- Long-term follow-up to assess the lasting effects of the treatment on motor function, cognitive development, and quality of life.
- Investigating the potential of stem cell therapy for other congenital conditions.
Did you understand?
Placenta-derived stem cells are considered particularly promising because they are readily available, pose a low risk of immune rejection, and have shown regenerative properties in pre-clinical studies.
FAQ
- What is spina bifida? A birth defect that occurs when the spinal cord doesn’t close completely during pregnancy.
- Is this treatment currently available? No, this is still an experimental therapy undergoing clinical trials.
- Where did the stem cells come from? Donated placentas.
- What are the potential benefits of this therapy? Improved mobility and quality of life for children with spina bifida.
Explore further: Learn more about spina bifida and ongoing research at Science.org.
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