Extraordinary operations in California on pregnant women to correct a serious abnormality of the spine in babies in the womb. The damage was repaired by stem cells taken from the placenta, treated and then reintroduced.
Spina bifida is an anomaly of the spine during foetal development, which can lead to very serious damage like paralysis of the lower limbs, genitourinary issues and even cognitive impairment. However, in the future it could be treated more effectively than today, even before birth, with an operation directly inside the womb with the aid of stem cells.
The first three children who underwent this new experimental technique at the University of California in Davis, are all doing well. They survived the operation, were born by caesarean section, and so far are in a much better condition than would be expected if they had been operated on after birth (as is usually the case).
The news was announced by the same team of surgeons and paediatricians, led by Professor Diana Farmer, who launched the final phase of a trial called CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele in 2021. Myelomeningocele is one of the forms in which spina bifida can occur and a total of 35 foetuses will be operated on during the trial.
The project, funded with $9 million from the California Institute for Regenerative Medicine, started by studying the potential of mesenchymal stem cells, taken directly from the placenta, in helping complete spinal fusion.
Once grown and processed in the laboratory on a biocompatible support that resembles a patch, the stem cells are ready to be placed on the foetus’ spine while it is still exposed and not covered with skin. They then help to repair it.
Spina bifida, the road to a cure through a long series of preliminary tests
Over the past few years, Farmer's team has conducted several experiments on animals, in particular sheep and English bulldogs, which are extremely prone to spina bifida. In view of their excellent results (puppies developed normally, without any disabilities), it was decided to progress with the first tests on humans, and the first three children are now one year old.
From a technical point of view, the procedure involves around forty specialists and the final phase lasts four days. This is the time needed to extract the mother's stem cells, grow them on the support and then surgically implant them into the foetus. The procedure involves making a small incision in the uterus, manipulating the foetus until the spine is accessible, inserting the stem 'patch', and then closing up. Each of these highly delicate phases can fail, or unforeseen events like contamination can occur, so they must be carried out by highly specialised professionals who work in different phases in teams over many hours.
However, if everything goes well, as it did when the Californian team operated on a woman called Emily under general anaesthetic when she was 25 weeks pregnant, the result was Robbie, a baby girl who started kicking as soon as she was born (by caesarean section at 35 weeks). Children with spina bifida often cannot perform this simple, spontaneous movement because their lower limbs are paralysed. Those kicks were a great source of joy and hope to both the parents and the team, although a long series of checks will still be needed to see how Robbie is really doing. For the moment, however, everything is going according to plan.
The decisive role of stem cells
In the early 2000s Diana Farmer made an extremely significant breakthrough for children with spina bifida by proving that it was possible to operate on them in utero (as the world's first female foetal surgeon). The babies born were equally compromised, and dependent on a wheelchair, but they were in a much better condition than babies who were not operated on, or who were operated on in the first few months of their life.
But the addition of the stem 'patch' is a huge leap forward, according to the researchers, and could almost completely defeat the disease, resulting in children who are destined to function normally.
The children operated on in Davis will be followed until they are six years old, with a check-up every six months, to see what kind of mobility they are acquiring. Early results look promising.