World-First Stem Cell Therapy for Spina Bifida Shows Safety in Groundbreaking Fetal Surgery Trial

UC Davis Health (搜索)搜索该词条 researchers have achieved a medical milestone by successfully completing the world's first clinical trial combining fetal surgery with stem cell therapy to treat spina bifida in utero. The Phase 1 CuRE (Cellular Therapy for In Utero Repair of Myelomeningocele) trial demonstrated the safety and feasibility of applying placenta-derived stem cells (搜索)搜索该药品 directly to exposed spinal cords of fetuses, with results published in The Lancet.

Breakthrough Treatment Approach

The innovative procedure involves making a small opening in the uterus during fetal surgery at 24-25 weeks of gestation. Surgeons float the fetus to the incision point, exposing the spina bifida defect on the back. A small patch containing living stem cells derived from donated placentas is then placed directly over the fetus's exposed spinal cord (搜索)搜索该靶点 before closing the tissue layers to allow regeneration.

"Putting stem cells into a growing fetus was a total unknown. We are excited to report great safety," said Diana Farmer, the trial's principal investigator and chair of the UC Davis Department of Surgery. "It paves the way for new treatment options for children with birth defects. The future is exciting for cell and gene therapy before birth."

Promising Safety Results

All six pregnant women in the initial phase underwent surgery without complications. The newborns were delivered at approximately 34 weeks of gestation with no signs of infection, cerebrospinal fluid leakage, or tumor growth. Researchers had particular concerns about potential cancer risk from the stem cells, but no abnormal tissue growth or tumors formed at the repair site.

Notably, all six newborns showed reversal of hindbrain herniation, a serious complication where the back of the brain descends into the neck and blocks cerebrospinal fluid circulation. Additionally, no babies required shunts for hydrocephalus before hospital discharge, indicating successful surgical outcomes.

Addressing Critical Medical Need

Spina bifida affects 1,500 to 2,000 children annually in the United States and fewer than 1 in 1,000 births globally, though rates are higher in low-income countries. The most severe form, myelomeningocele, can cause excess fluid accumulation in the brain, potential brain damage, paralysis, and bladder and bowel control issues.

Current fetal surgery to close spinal cord (搜索)搜索该靶点 defects has improved outcomes significantly, but limitations remain substantial. According to Farmer, who previously led the trial demonstrating the benefits of in utero surgery, nearly 60% of children are still unable to walk or move independently following the procedure. This occurs because surgery cannot reverse damage to spinal cord neurons caused by exposure to amniotic fluid during fetal development.

Clinical Impact and Patient Experience

The stem cell treatment aims to protect the developing spinal cord (搜索)搜索该靶点 from further damage before birth, potentially adding regenerative power to standard surgical repair. "This is a major step toward a new kind of fetal therapy, one that doesn't just repair but potentially helps heal and protect the developing spinal cord," said Aijun Wang, co-inventor of the placental-derived stem cell treatment technology and study co-principal investigator.

Trial participant Michelle Johnson, whose son Tobi was born in 2022, described the experience as life-changing. "Our family couldn't feel more blessed and fortunate to participate in the CuRe Trial," Johnson said. "Tobi's physical and mental abilities are nothing short of a miracle. We are forever grateful for the many health professionals who supported Tobi's journey and continue to watch him conquer the world."

Future Development

Based on the strong safety results, the FDA and an independent monitoring board approved advancement to the next phase. The CuRE trial is now enrolling up to 35 patients in its Phase 1/2a study, with children followed through age 6 to evaluate long-term safety and early signs of improved movement, bladder, and bowel function.

Clare Whitehead, a fetal-medicine specialist at the Royal Women's Hospital in Melbourne, Australia, acknowledged the significance while emphasizing caution. "It's exciting and it's important that we're advancing in the field and that we're looking at novel therapies," Whitehead said. "It is also reassuring that the therapy did not cause harm, but it is still too early to know whether it will be beneficial. With fetal therapy, we have to be very cautious."

The first phase of the trial was funded by a $9 million grant from the California Institute for Regenerative Medicine (搜索)搜索该公司 (CIRM), highlighting the state's investment in advancing regenerative medicine approaches for treating birth defects.