UC Davis Health in Sacramento, Calif., on March 1 announced the launch of the first FDA-approved human clinical trial using stem cells before birth to treat spina bifida, a defect that occurs when the spine and spinal cord do not form properly.
The clinical trial will administer stem cells derived from placental tissue to babies that are still in the womb. Researchers hope to repair the defect that occurs when the protective tissue around a baby's developing spinal cord fails to fully close before birth.
The California Institute for Regenerative Medicine provides a $9 million grant to Diana Farmer, MD, professor and chair of surgery at UC Davis Health, and Aijun Wang, PhD, associate professor of surgery and biomedical engineering at the health system, to fund the clinical trial.
"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," Dr. Farmer said. "Our team has spent more than a decade working up to this point of being able to test such a promising therapy."
The clinical trial will treat six patients who will be monitored by researchers for 30 months after birth to assess the safety and efficacy of the stem cell procedure.
During the procedure, the fetal surgeon will place a biological scaffold of placental mesenchymal stem cells directly over the exposed spinal cord. The surgeon then closes the opening in the baby's back to allow the tissue to regenerate and protect the infant's spinal cord.
"Our cellular therapy approach, in combination with surgery, should encourage tissue regeneration and help patients avoid devastating impairments throughout their lives," Dr. Wang added.