Stem Cell Patch Shows Promise in Treating Spina Bifida in Womb
A false-colour X-ray showing a large neural tube defect (red) on both sides of the lower back in someone with spina bifida
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A groundbreaking trial has utilized a patch composed of stem cells derived from donor placentas to treat fetuses diagnosed with a severe form of spina bifida while still in the womb. This innovative approach has shown promising results in reversing brain complications associated with the congenital condition, potentially offering improved long-term outcomes for affected children.
One mother, whose child Toby underwent the treatment and is now 4 years old, expressed her astonishment at the positive impact it had on her son’s life. Initially expecting that Toby would require a wheelchair due to his spina bifida diagnosis, she is now thrilled to witness him achieving significant milestones such as walking, running, and maintaining bladder control – outcomes that are rare for individuals with this condition.
Spina bifida, a condition affecting approximately 1 in every 2800 births in the US annually, occurs when the spinal cord and spine fail to fully develop during fetal growth. In the most severe form known as myelomeningocele, the spinal cord protrudes through an opening in the vertebrae, often leading to mobility issues and challenges with bowel and bladder function. While the exact cause of spina bifida remains unknown, insufficient folic acid intake during pregnancy is linked to an increased risk.
Traditional treatment for severe cases involves in-utero surgery to reposition the spinal cord within the vertebrae and close the opening in the skin. However, this procedure does not always result in improved mobility or bladder control for affected children, prompting researchers to explore additional therapeutic options.
Dr. Diana Farmer and her team at the University of California, Davis, investigated the potential of incorporating stem cells into the treatment to enhance spinal tissue growth and repair. In a trial involving six pregnant women carrying fetuses with myelomeningocele, the researchers observed a common complication called hindbrain herniation, where excess fluid accumulation in the skull forces a part of the brain through an opening at the base of the skull.
During the trial, all fetuses underwent the standard surgery along with the placement of a patch containing stem cells derived from donated placentas. This patch, infused with a matrix of adhesive proteins, was applied to the spine before suturing the skin. The stem cells within the patch released beneficial factors to promote tissue regeneration without causing abnormal cell growth.
Post-birth assessments revealed successful healing at the surgical site in all infants, with no signs of adverse cell proliferation. Additionally, MRI scans indicated complete reversal of hindbrain herniation, indicating a positive response to the stem cell intervention.
Experts like Dr. Panicos Shangaris from King’s College London anticipate that this novel approach could lead to improved long-term outcomes compared to conventional methods, based on findings from animal studies. The research team plans to conduct a larger trial involving 35 fetuses with myelomeningocele to further evaluate the effectiveness of the stem cell patch treatment.
While the initial results are promising, Dr. Shangaris suggests that a direct comparison between the stem cell approach and standard surgery through a randomized controlled trial would provide more robust evidence for regulatory approval. Such comprehensive studies are essential in determining the safety and efficacy of innovative treatments for spina bifida in utero.
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