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Providing stem cells before birth to babies with spina bifida improves their quality of life, trial reveals.

Providing stem cells before birth to babies with spina bifida improves their quality of life, trial reveals.

Stem Cell Treatment Shows Promise for Unborn Babies with Spina Bifida

Administering stem cells to unborn babies diagnosed with spina bifida during in utero surgery might represent “a major milestone” in addressing birth defects, according to medical professionals.

A trial conducted in the US demonstrated that using stem cells harvested from the mother’s placenta to treat the baby’s spine during repair was both safe and effective, enhancing the child’s mobility and overall quality of life.

Dr. Diana Farmer, who spearheaded the study, suggested that this experimental therapy could eventually become the standard care for treating spina bifida prior to birth. “If it enables more children to walk who otherwise wouldn’t, then it really just makes sense as a primary approach,” she explained. “The results are incredibly exciting and open up a range of possibilities for new treatments addressing spina bifida and other congenital conditions.”

The study, published in the Lancet on Friday, was conducted between 2021 and 2022 and involved six unborn babies diagnosed with myelomeningocele, the more severe form of spina bifida.

Surgeons from the Davis School of Medicine at the University of California applied placenta-derived mesenchymal stem cells onto the exposed spinal cords of these babies, who were between 24 and 25 weeks gestation at the time of the procedure.

Follow-up MRI scans revealed that all six babies showed a reversal of hindbrain herniation—a common brain abnormality linked to spina bifida where the lower brain protrudes into the neck area.

Importantly, the stem cells did not lead to tumors, interfere with healing, or exacerbate the condition—all potential side effects that researchers had been cautious about.

Kate Steele, CEO of the charity Shine, characterized the results as “very exciting.”

“The strides in stem cell research that made this initial human trial possible are nothing short of remarkable, and we’re thrilled to see that the procedure’s safety has been confirmed,” she noted. “Observing the difference that fetal surgical repair is already making for children with myelomeningocele, and knowing that introducing stem cell therapy could enhance outcomes for babies born with spina bifida, is incredibly encouraging.”

Steele also mentioned that enhancements in bladder and bowel function would be especially beneficial.

A commentary related to these findings, authored by Dr. Magdalena Sanz Cortes, a fetal medicine specialist from Texas who didn’t participate in the trial, highlighted the implications of the results. She suggested that if further studies reaffirm the benefits, we could be on the brink of a new era in fetal surgery that may significantly help in reversing neurological damage in utero linked to spina bifida.

It’s estimated that between 8,100 and 11,900 individuals in England and Wales live with open spina bifida, and around 536 pregnancies each year are affected by this condition, according to Shine.

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