University of California, Davis Health
Tobi, with parents Michelle Johnson and Jeff Maginnis, was one of 6 children who received experimental stem cell therapy for spina bifida before birth
After years of trial and error, a team of researchers managed to successfully treat a child with spina bifida in the womb using stem cells derived from placentas.
A team of researchers resorted, for the first time, to stem cells and surgery to treat spina bifida in the womb. The study focused on safety, but the results raise hope that this approach could give children a opportunity to walk.
Michelle Johnson was 20 weeks pregnant when she found out that the child she was expecting had spina bifida. As the column had not closed completely, the spinal cord was exposedexiting through a deep opening. Without surgery, I would face a lifetime of disability.
Therefore, he took the opportunity to join a small experimental trial on this condition at the University of California. The treatment combines fetal surgery, an existing approach, with a dose of stem cells to stimulate healing.
Now four years old, Johnson’s son Tobi can walk and does not present symptoms such as loss of bladder and bowel control. “Tobi’s physical and mental abilities are nothing short of a miracle,” he said in a statement from the University of California/Davies Health.
Tobi is one of six children included in the CuRe assaythe first study to test whether using stem cells to repair tissue in fetuses with spina bifida is safe, the results of which were recently presented in a published in The Lancet.
Administered through a small graft sewn onto the injured areathe stem cells protected the spinal cord from inflammation and helped the wound to heal. None of the babies or mothers suffered side effects short-term, such as unwanted tissue growth or cancer.
With so few participants, it is still too early to understand how this approach will evolve as children grow. But thanks to the promising safety profile, the FDA approved the recruitment of more pregnant women with the same diagnosis.
“This is an important step toward a new type of fetal therapy that not only repairs, but can also help heal and protect the developing spinal cord,” he said in the statement. Aijun Wangauthor of the study.
CuRe joins other attempts to combat diseases with stem cells in the womb. Although this is a very new area, this approach could slow down, crash or cure various diseases before birth.
Tobi, who is 4 years old, can walk without difficulty
A starting advantage
Spina bifida is a rare clinical condition in which the spine or spinal cord do not close properly during developmentexplains .
Every year, 1 in every 2875 newborns is affected in the USA. In its most serious form, the cerebrospinal fluida fluid that surrounds the brain and eliminates toxins, accumulates, causing progressive damage to the spinal cord of the fetus, permanent mobility problems and even paralysis.
The condition began to be treated after birth, when surgeons closed the defect. But by then the damage had already set in..
Surgery before birth could alleviate symptoms, an idea validated in a 2011 trial. more than half of babies treated continued ter difficulty walking without assistanceprobably because injured neurons in the brain and spinal cord of fetuses did not have the opportunity to recover.
Stem cells promote regeneration by releasing protective nutrients, and the fetal environment is particularly favorable to these cells. The team wanted to see if its addition could improve prenatal surgery.
Researchers began testing the idea around 2012, using at the time induced pluripotent stem cells. This is a type of stem cell obtained from the skin or other mature cells through a chemical cocktail. This approach could guarantee a virtually unlimited supply of stem cells. But it didn’t work.
After years of trial and error, the team found success with stem cells derived from placentas. In the laboratory, these cells protected neurons from injury and stimulated their growth. They also helped cure defects in an ovine model of spina bifida. Newborns who received the cells, along with prenatal surgery, were able to stand and walk; Those who only had surgery were unable to do so.
Stem cell therapy looked promising. But in the case of unborn babies, could entail risks. As the cells come from donors, they could trigger immunological reactions. They could also cause abnormal tissue growth, or even cancer.
Because stem cell treatments are rarely used in the womb, little is known about its effects on pregnancy or the general health of mother and baby. The first phase of the CuRe trial focused precisely on these safety concerns.
The team of researchers placed stem cells derived from donated placental tissue in a small graft. To help integrate the cells, the researchers designed the graft to replicate the conditions that normally surround the cells.
Surgeons made a small opening in the uterus between the 24th and 25th weeks of pregnancy and administered a small dose of painkillers and muscle relaxants. They then placed the stem cell graft over the exposed spinal cord and sutured the opening.
The trial closely followed six babies, including Tobito detect possible side effects. After the cesarean section, none had complications, such as cerebrospinal fluid leakage, infection or signs of cancer.
In all cases, the treatment stopped parts of the brain from sliding to the neck, and none of the babies needed a shunt, a small tube used to drain excess fluid from the brain — an encouraging sign of success.
The team turned to stem cells, the authors wrote in their paper, because they can reduce brain inflammation and brain cell death. At the same time, they produce growth proteins that “support the preservation of neural tissue and the integrity of the spinal cord.”
The study joins the increasing use of stem cells before birth in conditions such as thalassemiaa blood disorder, and osteogenesis imperfecta, also known as brittle bone disease.
“Introducing stem cells into a developing fetus was a big unknown. We are excited to be able to report a high level of safety,” he said. Diana Farmerlead author of the study and principal investigator of the CuRe trial.
“These results open the way to new treatment options for children with congenital malformations. The future of cell and gene therapy before birth is exciting”, concludes the researcher.
