Tobi Maginnis was the second baby to have spina bifida repaired before birth using rejuvenating stem cells taken from the placenta, according to researchers. An ultrasound scan at 20 weeks into pregnancy showed Tobi had spina bifida, a condition where the spinal cord fails to develop properly, leaving part growing outside the body. Spina bifida occurs when a baby's spine fails to develop fully in the womb, typically diagnosed during pregnancy or shortly after birth. Open spina bifida, known as myelomeningocele and meningocele, is less common but more serious, causing the spinal cord to push out of a gap in the spine. Symptoms of open spina bifida can include problems with walking or paralysis, incontinence, loss of feeling in the legs, a curved spine, and fluid buildup in the brain causing learning difficulties. Spina bifida affects about one in 1,000 babies. Around 500 babies a year in the UK are born with myelomeningocele, the most severe form of spina bifida, according to experts.
Tobi was one of six babies in a world-first clinical trial led by the University of California Davis Children's Hospital. The trial in California included six women between 24 and 25 weeks pregnant, each with babies diagnosed with myelomeningocele and hindbrain herniation. Around 25 weeks into pregnancy, surgeons made a small opening in the mothers' abdomen and womb, placed a patch of stem cells over the babies' exposed spinal cord, and closed the incision to allow tissue regeneration. Surgeons applied stem cells derived from donated placentas to the exposed spinal cord during fetal surgery. All six babies were delivered between July 2021 and December 2022, born with spinal repairs intact and no signs of infection or abnormal tissue growth. An MRI scan after birth confirmed that hindbrain herniation had been reversed in the babies.
All six babies in the trial were born healthy with no sign of side effects from treatment, according to results published in The Lancet medical journal. The study published in The Lancet establishes a scalable and clinically feasible platform for targeted delivery of biological therapeutics to the fetus, according to researchers. Researchers said the successful integration of cell-based therapy into fetal surgery shows the feasibility and safety of delivering rationally designed biologics during gestation, with potential to alter lifelong health trajectories. Researchers are optimistic the procedure could alter the lifelong health of those with spina bifida.
Tobi's mother, Michelle Johnson, said he was full of energy and runs, walks, jumps, and is all over the place. She added, 'We expected Tobi to be wheelchair-bound. So to see where he is now, it's nothing short of a miracle.' Tobi's father, Jeff Maginnis, said, 'He's the second human to go through this surgery with stem cells. So there was a lot of uncertainty, a lot of unknowns. We're just blessed every day when we see how Tobi's reacted and been affected by this very successful surgery.'
Dr. Diana Farmer, who led the study, said, 'The most exciting thing was the baby came out kicking her legs and wiggling her toes, which really exceeded expectations. We expected her, had she not had treatment, to not be able to have that kind of leg movement.' She added, 'We're just making sure that there are no unexpected things from having stem cells in the spinal cord of a foetus. This is brand new.'
Surgery in the womb to correct spina bifida is increasingly common, but one study showed about half of babies are unable to walk independently, according to researchers. Fetal surgery to close open spina bifida does not fully address mobility problems when lower motor neurons are damaged, according to researchers. US surgeons hope using stem cells will improve success rates for spina bifida surgery, helping with walking and potty training. Stem cell therapy performed on babies in the womb could reverse brain abnormalities linked to spina bifida, according to researchers.
The long-term effects of stem cell therapy on the babies' development and health are not yet known. A larger clinical trial has started with 35 pregnant women, and their babies will be monitored over several years. Findings from the phase one trial have allowed experts to proceed with enrolling 35 patients on another study. The six children in the trial will be followed up until age six to assess long-term safety of the treatment.