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The Lancet: First-ever in-utero stem cell therapy for fetal spina bifida repair is safe, study finds
A phase 1 clinical trial published in The Lancet has shown that combining stem cell therapy with standard fetal surgery before birth is a safe and promising approach to treat myelomeningocele, a severe form of spina bifida. This is the first time live stem cells have been used on a fetus’ damaged spine, which could potentially lead to better health outcomes for babies compared to traditional fetal surgery.
Spina bifida is a congenital condition in which the spinal cord does not develop properly, leaving part of it exposed. This can cause lifelong health challenges, including paralysis, difficulty walking, and issues with bladder and bowel control. Current treatments involve surgery during pregnancy to close the spinal opening, which can reduce some complications but often does not prevent all neurological problems.
In the trial, six pregnant women with fetuses diagnosed with spina bifida underwent standard fetal surgery plus an additional step where surgeons applied cells from the placenta—called placenta-derived mesenchymal stem cells (PMSCs)—directly onto the exposed spinal cord during the surgery. These cells are known for their ability to reduce inflammation, promote healing, and protect nerve tissue.All six babies, who were born between July 2021 and December 2022, had their spinal repairs intact and showed no signs of infection, abnormal tissue growth, or tumour formation. Post-birth MRI scans confirmed that brain abnormalities associated with spina bifida, called hindbrain herniation, were reversed in every case.
In addition, the infants experienced no serious adverse effects attributable to the stem cell treatment during the trial and through follow-up care. The children in this trial will be carefully monitored, with regular check-ups and assessments, until they turn six. This long-term follow-up will help researchers confirm that the stem cell treatment remains safe and improves children’s mobility, health, and quality of life as they grow.
Additional large-scale, long-term clinical trials are underway to further refine surgical techniques and treatment protocols. These studies aim to confirm that children who receive this therapy experience benefits at birth, improved mobility, and a better quality of life. Throughout this process, regulatory agencies will work closely with the research team to carefully monitor safety and effectiveness. The researchers aim to establish this stem cell therapy as a safe and standard option for fetal repair of spina bifida, providing new hope for families affected by this condition worldwide. They say these results represent a major milestone in the field of in-utero stem cell treatment for birth defects, paving the way for future advances using stem cells during fetal surgery to address other congenital conditions.
Expert Reaction
These comments have been collated by the Science Media Centre to provide a variety of expert perspectives on this issue. Feel free to use these quotes in your stories. Views expressed are the personal opinions of the experts named. They do not represent the views of the SMC or any other organisation unless specifically stated.
A/Professor Kuldip Sidhu is co-founder and director, CK Cell Technologies and Conjoint with University of New South Wales Medicine.
"Spinal bifida is a rare congenital malfunction of the nervous system due to incomplete closure of the neural tube and if not attended early may lead to various neurological, urological, orthopedics and cognitive complications later in life. In-utero surgical inventions are successfully employed with good outcomes. However, to decrease morbidity associated with surgery, further studies and attentions are required. From those perspectives, this study is very timely. Regenerative strategies using stem cells, biomaterials, and scaffolds have been tried in pre-clinical animal models with good success. In the current study, minimal data are provided to make any meaningful interpretation, such as lack of information about the level of spinal bifida defect, lack of quality, and number of mesenchymal stem cells used. Further controlled studies are required to differentiate the effects of surgery vs stem cells alone or their derived products as exosomes, and that may shift the focus from pure fetal surgery as protective to a complete regenerative paradigm"
Dr Lana McClements is an Associate Professor in the School of Life Sciences, Faculty of Science at University of Technology Sydney
"Researchers from the University of California Davis Health have made an exciting breakthrough in treating the most severe form of spina bifida, known as myelomeningocele. In their study, they combined a special gel containing placenta-derived stem cells (donated from other people) with established fetal surgery, known to reduce complications of this type of spina bifida.
After years of carefully testing their approach in the lab and using animal models, the team moved on to treating pregnant individuals whose babies had been diagnosed with myelomeningocele. During the surgery performed while the baby was still in the womb, they applied the gel directly to the exposed spinal cord. The results were remarkable: all babies (six) delivered after this procedure were healthy, with no signs of wound problems, infection, leakage of spinal fluid, or tumour growth.
Earlier research (MOMS study) showed that fetal surgery alone could improve outcomes for these babies, but many still struggled to walk independently after birth. This new treatment, which uses the regenerative power of stem cells alongside fetal surgery, holds great promise for giving affected babies a better chance at improved mobility and quality of life. While the study has shown the treatment is safe, more research involving a larger group of participants is needed to determine its true effectiveness."
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