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A UC Davis Health team this month announced the launch of the world’s first, FDA-approved human clinical trial using stem cells before birth to treat the most serious form of spina bifida. The condition is a birth defect that occurs when the spine and spinal cord don't form properly. Without treatment, an exposed spinal cord causes severe neurological damage, resulting in problems that can include lifelong cognitive, mobility, urinary and bowel disabilities.
first stem cell clinical trial for spina bifida treatment announced uc davis health combines fetal surgery and cellular therapy special editorial submitted by friends steward partner uc davis health 46 connexions
The one-of-a-kind treatment will be delivered while the baby is still in the mother’s womb (in utero). It will be the standard surgical procedure combined with the use of a unique stem cell “patch” to repair the defect before birth. The team anticipates seeing improvements for those born with the most severe form of spina bifida known as myelomeningocele (MMC). “Currently, the standard of care for our patients is fetal surgery, which, while promising, still leaves more than half of children with spina bifida unable to walk independently,” said Diana Farmer, professor and chair of surgery at UC Davis Health and principal investigator on the study. “There is an extraordinary need for a treatment that prevents or lessens the severity of this devastating condition. Our team has spent more than a decade working up to this point of being able to test such a promising therapy.” In the United States, four babies a day are born with spina bifida (about 1 in every 2,700 live births each year). The condition leaves a portion of the spinal cord and nerves exposed without any bone or skin covering them. It causes a variety of problems because the spinal cord controls a person’s ability to move their legs and walk. It can also lead to extra fluid in and around the brain (hydrocephalus), causing brain injury.