A few hours after Tom (pseudonym) was born, he became restless and did not want to breastfeed. His mother noticed that his left arm and left leg were shaking rhythmically—something was wrong.

Tom was immediately transferred to the neonatal intensive care unit. An MRI scan revealed he had suffered a severe stroke. Doctors told Tom’s parents that they were unable to treat the child. He may be disabled.

Most people think that stroke mainly affects the elderly, but it can also occur in newborns. These “perinatal strokes” occur when one of the brain’s main arteries is blocked, resulting in a lack of blood supply — and therefore oxygen — to certain brain areas. About 1 in 5,000 newborns have a stroke. It usually occurs in the first few days after they are born.

Most babies experience problems later in life, and the severity of the problem depends on the area of ​​the brain injured. These problems may include muscle tension in the arms and legs (cerebral palsy), behavioral problems, learning difficulties, and epilepsy.

There is no treatment for newborns with stroke. Researchers, including our own team at the Utrecht University Medical Center, have been working on new treatments, one of which involves stem cells.

Stem cells have the ability to become many different cells in the body, and they are small factories for several growth factors (proteins that the growth of specific tissues). The theory is that if we can introduce stem cells into damaged parts of a baby’s brain, the stem cells’ growth factors will stimulate the brain to repair itself.

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It works in animals Early animal studies have shown that injecting stem cells into the brains of newborn mice with a stroke can significantly reduce the brain damage and disability they suffer. Experiments showed that the treatment was safe and had no side effects on the mice. These animal studies give us hope that the treatment could also work in newborn babies, preventing lifelong disability.

But how can stem cells be delivered to a baby’s brain without using needles or surgery? We decided to try an intranasal route (through the nose) that was tested in mice. After we deliver the stem cells through the nose, the cells move quickly and exclusively to the damaged brain area. Injured brain regions send out “alarm signals” that direct stem cells to the right in the brain.

Once the stem cells reached the damaged area, they secreted growth factors that boosted the mouse brain’s repair system. Within a few days, the stem cells were broken down and no longer tracked in the brain. After several experiments with this method, we concluded that dripping stem cells into the nose is the safest and most efficient way to deliver them to the brain.

Ten Babies After years of laboratory research, we finally tested the treatment on babies. The results were published in The Lancet Neurology.

The aforementioned baby Tom was the first to take part in the study and receive stem cells within a week of birth. Asking parents to participate in experimental treatments during the first week of a newborn’s life is a very delicate process.

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After we had a lengthy conversation with his parents, they decided to enroll their son in the study. He receives stem cells via nasal drops, a process that takes only a few minutes. Afterwards, Tom was closely monitored for several days before returning home.

We treated ten newborns who were transferred from hospitals across the Netherlands to the University Medical Center Utrecht after a stroke. In all ten neonates, the administration of the stem cell droplets did not have any complications. One infant developed a mild fever after treatment, which soon resolved on its own.

Follow-up MRI scans of the brain three months after the stroke showed less damage than expected, possibly because of stem cells. At four months old, the treated infants, including Tom, performed well when tested for the quality of their movements. When the children are two years old, we will check their development again.

We are now looking for an opportunity to conduct a randomized controlled trial (the gold standard in medical research) to demonstrate that stem cell therapy can effectively repair brain damage after perinatal stroke.

The discovery of a new, safe stem cell therapy also opens up opportunities for other babies with brain damage, such as those born prematurely or born with hypoxia (perinatal asphyxia). Stem cell therapy offers hope for the most vulnerable patient populations, with potentially lifelong benefits.

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