In a landmark clinical trial, world’s first stem cell therapy treats spina bifida before birth
The day Emily learned that her developing child had spina bifida was also the day she first heard about the world-first clinical trial for the treatment.
The one-of-a-kind treatment, known formally as CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele, is a landmark clinical trial at UC Davis Health, wherein a stem cell patch is administered to the fetus’ spine while still in the mother's womb. This could vastly improve outcomes for children with this birth defect.
The clinical trial, which was launched in the spring of 2021, seemed like a lifeline to Emily, one that she couldn't refuse. She and baby Robbie were the first to undergo the groundbreaking treatment. A year down, both mom and baby are home and in great health.
Today, three babies in total have been born after receiving spina bifida treatment combining surgery with stem cells. They will be monitored by the research team until 30 months of age to fully assess the procedure’s safety and effectiveness. Thirty-five patients will be treated in total.
"I’ve been working toward this day for almost 25 years now," Diana Farmer, the world’s first woman fetal surgeon, professor, and chair of surgery at UC Davis Health and principal investigator on the study, said in a press release.
If Robbie had remained untreated, she was expected to be born with leg paralysis.
Combining surgery with stem cells in a landmark treatment
Spina bifida, also known as myelomeningocele, occurs when spinal tissue fails to merge properly during the early stages of pregnancy. Often diagnosed through ultrasound, the birth defect can lead to a range of lifelong cognitive, mobility, urinary, and bowel disabilities. It affects 1,500 to 2,000 children in the U.S. every year. While there’s no cure for spina bifida, surgery after birth can improve the symptoms in some cases.
Farmer and bioengineer Aijun Wang and their research team at UC Davis have been working on their novel approach using stem cells in fetal surgery for more than 10 years. Over that time, with animal modeling, the team demonstrated that the treatment could prevent the paralysis associated with spina bifida.
The stem cells work to repair and restore damaged spinal tissue, much beyond what surgery can accomplish alone.
According to the press release, preliminary work by Farmer and Wang proved that prenatal surgery combined with human placenta-derived mesenchymal stromal cells, held in place with a biomaterial scaffold to form a "patch," helped lambs with spina bifida walk without noticeable disability.
And, when the team refined their surgery and stem cell technique for canines, the treatment also improved the mobility of dogs with naturally occurring spina bifida.
Farmer and Wang’s team manufacture clinical-grade stem cells from placental tissue in the UC Davis Health’s CIRM-funded Institute for Regenerative Cures. The process takes four days. "The time we pull out the cells, the time we seed on the scaffold, and the time we deliver, are all critical," said Priya Kumar, the scientist at the Center for Surgical Bioengineering in the Department of Surgery, who leads the team that creates the stem cell patches and delivers them to the operating room.
Changing lives, making history
A 40-person operating and cell preparation team was in-charge of Emily's "historic" procedure.
After the mother was placed under general anesthetic, a small opening was made in her uterus. Surgeons floated the fetus up to that incision point to expose its spine and the spina bifida defect. They then used a microscope to begin the repair in which the stem cell patch was placed directly over the exposed spinal cord of the fetus. The fetal surgeons then closed the incision to allow the tissue to regenerate.
The first-of-its-kind surgery was a huge success.
Robbie was born via C-section, on September 20, 2021, and just celebrated her first birthday. Both mom and baby are home and in great health.
"I got to see her toes wiggle for the first time. It was so reassuring and a little bit out of this world," Emily said. "This experience has been larger than life and has exceeded every expectation. I hope this trial will enhance the quality of life for so many patients to come."
Farmer had fervently hoped for that day. "It was very clear the minute she was born that she was kicking her legs and I remember very clearly saying, 'Oh my God, I think she’s wiggling her toes!'" said Farmer, who noted that the observation was not an official confirmation, but it was promising. "It was amazing. We kept saying, 'Am I seeing that? Is that real?'"
According to the CuRe team, a lot is still to be learned during the safety phase of this trial. They will continue to monitor Robbie and the other babies in the trial until they are six years old, with a key checkup happening at 30 months to see if they are walking and potty training.