Spina bifida: Surgery plus stem cells before birth could improve outcomes (video)
One-of-a-kind treatment approved for human testing
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A UC Davis Health fetal surgeon and a stem cell scientist have received approval from the U.S. Food and Drug Administration (FDA) to test a groundbreaking spina bifida treatment that combines surgery with stem cells. The one-of-a-kind treatment, delivered while the baby is still in the mother’s womb, could improve outcomes for children with the birth defect.
Spina bifida occurs when spinal tissue improperly fuses during the early stages of pregnancy, leading to a range of lifelong cognitive, mobility, urinary and bowel disabilities. It affects about 1,500 to 2,000 children each year in the U.S.
The new treatment was developed by Diana Farmer, professor and chair of the UC Davis Department of Surgery, surgeon-in-chief of UC Davis Children’s Hospital and an expert in prenatal treatment of pediatric conditions. Her chief collaborator on this work is Aijun Wang, an associate professor of surgery and biomedical engineering and leader in developing cellular therapies that promote tissue regeneration.
Answers fueled by collaboration
Farmer and Wang have been working for years to prepare for human clinical trials. Their preliminary work proved that prenatal surgery combined with a specific type of stem cells (human placenta-derived mesenchymal stromal cells, or PMSCs), if held in place with a biomaterial scaffold, helped lambs with spina bifida walk without noticeable disability. It is believed that the stem cells work to repair and restore spinal tissue, beyond what surgery can accomplish alone.
When the team refined their prenatal surgery and stem cells technique for canines, the treatment also improved the mobility of dogs with spina bifida that were treated by UC Davis veterinary medicine experts.
Now, once clinical trials funding is obtained, Farmer and Wang will recruit pregnant women whose babies have been diagnosed with spina bifida to test this combined surgery and stem cell procedure. The clinical trial is expected to begin in January or February of 2021.
A surgeon driven to find a cure
Farmer’s career has been defined by her passion for finding a cure for spina bifida. As a leader of the MOMS trial, she proved that in utero surgery to repair the spinal cord injury reduced the neurological deficits of spina bifida. Many children in the study, however, still required wheelchairs or leg braces.
She recruited Wang specifically to help take that work to the next level. Together, they launched the UC Davis Health Surgical Bioengineering Laboratory to find ways to use stem cells to advance the effectiveness of surgery and improve functional outcomes for patients.
She also launched the UC Davis Children’s Hospital Fetal Care and Treatment Center (with pediatric surgeon Shinjiro Hirose) and UC Davis Children’s Surgery Center to provide comprehensive diagnostic, medical and surgical solutions for fetal, newborn and pediatric conditions. These centers are where clinical trial participants will be treated. Follow-up care will take place at UC Davis Children’s Hospital and Shriners Hospitals for Children — Northern California.
Farmer and Wang’s work has been funded by the National Institutes of Health, California Institute for Regenerative Medicine and Shriners Hospitals.
More information about UC Davis Health — including its children’s hospital, fetal care center, surgical services and stem cell research program — is at health.ucdavis.edu.
Related stories and resources
Spina bifida MOMS trial
Prenatal stem cell treatment improves mobility issues caused by spina bifida
Spina bifida patient healthy, happy after fetal surgery
Stem cell treatment for children helps dogs first
UC Davis Health and Shriners Hospital partner to provide expertise to spina bifida patients
Researchers seeking a cure for spina bifida get a step closer to their goal
Learn about spina bifida (information from the U.S. Centers for Disease Control and Prevention)