A groundbreaking trial utilized a patch made from donor placenta stem cells to treat a fetus suffering from severe spina bifida in utero. This innovative technique appears to reverse brain complications associated with congenital disorders, showing potential to improve long-term mobility in affected children.

The mother of a now four-year-old boy named Toby, who was diagnosed with spina bifida during pregnancy, was initially prepared for him to rely on a wheelchair. “But Toby is thriving. He has met all his developmental milestones, including walking, running, and jumping, and remarkably has no issues with bladder control, which is rare among those with this condition,” she commented.

Spina bifida, affecting approximately 1 in 2,800 births annually in the United States, occurs when a baby’s spine and spinal cord do not fully develop in utero. The most severe form, myelomeningocele, involves the spinal cord and surrounding tissues protruding through vertebrae, often leading to mobility challenges and bowel or bladder control issues. The precise cause of spina bifida remains unclear, although a deficiency in folic acid during pregnancy can heighten risks.

Standard treatment often involves in-utero surgery where the spinal cord and surrounding tissues are repositioned before closing the skin. “However, many children still struggle with mobility, and often bowel or bladder control remains unimproved,” notes Diana Farmer of the University of California, Davis.

To explore alternatives, Farmer and her team proposed the addition of stem cells to enhance growth and repair of spinal cord tissue. They enlisted six pregnant women carrying fetuses diagnosed with myelomeningocele.

By approximately 24 weeks of gestation, all fetuses exhibited a common complication known as hindbrain hernia. This condition causes excess fluid to accumulate in the skull, pushing the cerebellum through an opening at the base of the skull. While standard surgical procedures can often help alleviate hindbrain hernias, many children continue to face complications post-surgery.

In this latest trial, all fetuses received standard surgery along with a patch, measuring several centimeters, that included stem cells from the donated placenta, set within a matrix of sticky proteins. The surgeons applied this patch to the spine before suturing the skin around it. “The cells release what we like to call ‘magical stem cell juice’,” Farmer explains.

Upon birth, all babies showed positive surgical site healing with no indications of abnormal cell growth. “Our primary concern was that adding stem cells would lead to excessive cell proliferation, but we did not observe this,” Farmer reported. MRI scans of their brains demonstrated complete resolution of hindbrain herniation.

“In my opinion, this will enhance long-term outcomes compared to standard methods,” added Panicos Shangaris from King’s College London, citing evidence from animal studies.

The research team is optimistic about conducting a trial aimed at administering the stem cell patch to 35 fetuses with myelomeningocele, comparing results with prior studies that utilized traditional surgery, as stated by Farmer.

However, Professor Shangaris suggests that a more suitable approach would involve head-to-head trials to thoroughly assess safety and efficacy between the two techniques, providing clear pathways for treatment approvals.