A clinical trial at UC Davis Health is pioneering a novel approach to treating spina bifida, combining fetal surgery with stem cell therapy. The "CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele" is the world's first spina bifida treatment to integrate fetal surgery with stem cells, offering hope for improved outcomes for children diagnosed with this condition.
The trial involves performing fetal surgery before the 25th week of gestation. During the procedure, surgeons apply a stem cell patch directly to the spinal cord of the developing baby. This innovative technique aims to repair and protect the spinal cord, potentially mitigating the lifelong disabilities associated with spina bifida.
Nixon's Journey: A Glimmer of Hope
Lindsey and Jared Metcalf learned their unborn son, Nixon, had myelomeningocele, the most severe form of spina bifida, at 18 weeks into the pregnancy. Myelomeningocele occurs when spinal tissue fails to fuse properly during early pregnancy, potentially leading to cognitive, mobility, urinary, and bowel disabilities. Approximately 1,500 to 2,000 children in the U.S. are affected by spina bifida each year.
Desperate for answers, the Metcalfs discovered the CuRe Trial at UC Davis Children's Hospital. Lindsey became the sixth patient in the trial, undergoing fetal surgery at 21 weeks.
"Walking Lindsey into surgery, I had complete confidence in the care. I researched the CuRe Trial, but I researched the doctors as well," Jared said. "I came to realize that I was among the brightest minds on the planet... The team is phenomenal."
Promising Early Results
Nixon, now a year old, is showing promising progress. He is taking steps with assistance and actively exploring his environment. His parents are optimistic about his future.
"He is cruising around and gets where he wants to go," Lindsey said. "Nixon is a little fighter. He's determined and I know there are great things in store for him."
Dr. Diana Farmer, the surgeon-scientist leading the trial, is encouraged by Nixon's results. "I can say it looks promising," she said. "You see patients like Nixon and it's hard not to be pretty excited."
The CuRe Trial: Aims and Future Directions
The CuRe Trial aims to enroll a total of 35 patients over the next two years. This will allow researchers to gather sufficient data to determine whether stem cell therapy provides additional benefits compared to fetal surgery alone. The trial's primary focus is to assess the safety and efficacy of this combined approach.
Dr. Farmer hopes that the trial will pave the way for a new standard of care for spina bifida, potentially preventing a lifetime of therapy for affected children. The team will continue to monitor Nixon's progress through follow-up appointments as part of the clinical trial.
