Stem cell therapy offers a new hope to repair brain damage in newborns

Stem cell therapy offers new hope for repairing brain damage in newborns

Utrecht (Netherlands), May 16

A few hours after the birth of Tom (a pseudonym), he became restless and did not want to breastfeed. His mother noticed that his left arm and leg were shaking harmoniously – nothing was right.

Tom was immediately transferred to the neonatal intensive care unit. An MRI showed that he had had a severe stroke. Doctors told Tom’s parents that there was no treatment they could give the child. Likely to 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 major arteries to the brain becomes blocked, reducing the supply of blood – and therefore oxygen – to certain areas of the brain. One in 5,000 births suffers a stroke. This usually happens in the first few days after they are born.

Most children will have problems later in life, as the severity of the problems depends on which areas of the brain are affected. These problems can include muscle tightness in the arms and legs (cerebral palsy), behavior problems, learning difficulties, and epilepsy.

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

Stem cells have the ability to transform into many different cells in the body, and they are small factories of many growth factors (proteins that stimulate the growth of certain tissues). The theory is that if we could introduce stem cells into the damaged part of a child’s brain, the stem cell growth factors would stimulate the brain to repair itself.

Active in animals

Previous animal studies showed that injecting stem cells into the brains of newborn mice with stroke significantly reduced the amount of brain damage and disability they experienced. Experiments showed that the treatment was safe and had no side effects in mice. These animal studies gave us hope that the treatment would work on newborns as well, preventing lifelong disability.

But how do you deliver stem cells to a child’s brain without the need for needles or surgery? We decided to try the intranasal (through the nose) route, which was tested on mice. After we deliver the stem cells intranasally, the cells travel quickly and specifically to the affected brain regions. The affected area of ​​the brain sends “alarm signals” that direct stem cells to the right place in the brain.

Once the stem cells reached the damaged area, they released growth factors that boosted the mice’s brains’ repair systems. Within a few days, the stem cells had broken down and could no longer be traced back to the brain. After several experiments with this method, we concluded that instilling stem cells into the nose is the safest and most effective way to deliver them to the brain.

ten children

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

Baby Tom, mentioned earlier, was the first child to take part in the study and received stem cells within a week of his birth. Requiring parents to enroll in a trial treatment in the first week of their newborn’s life is a very delicate process.

After a long conversation with his parents, they decided to allow their son to take part in the study. He obtained the stem cells via nasal drops, a process that only took several minutes. After that, Tom was closely monitored for a few days before he went home.

We treated ten newborns who were transferred from hospitals across the Netherlands to the University Medical Center in Utrecht after suffering a stroke. In all 10 newborns, stem cell drops were administered without any complications. There was a child with a mild fever after the treatment, which quickly disappeared on its own.

An MRI scan of the brain performed three months after the stroke showed less injury than expected, possibly due to stem cells. At four months of age, the treated babies, including Tom, performed well when the quality of their movements was tested. When the children are two years old, we will check their growth again.

We are now looking for opportunities to initiate a randomized controlled trial (the gold standard for medical studies) to demonstrate that stem cell therapy can effectively repair brain injury after perinatal stroke.

The discovery of a new and safe stem cell therapy also opens opportunities for other babies with traumatic brain injury, such as babies born prematurely or babies who suffer from a lack of oxygen during birth (perinatal asphyxia). Stem cell therapy gives hope to the most vulnerable group of patients, with potentially lifelong benefits. (Conversation)

2022-05-16 08:26:00

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