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There is no need to repeat
the dangers of birth defects.
The National Health Commission pointed out that three levels of preventive measures should be taken to reduce the occurrence
of birth defects by pre-marital pre-pregnancy examination, prenatal screening and diagnosis, and newborn disease screening.
However, under this system, primary and secondary prevention is mainly focused on screening and diagnosis, and intervention and treatment of pre-existing diseases are often limited to the neonatal stage
.
In fact, surgery in the womb before the birth of the fetus is also a secondary prevention of birth defects, but it is not well known to the public
.
Especially for those diseases that need to be taken as soon as possible to curb their development, if the intervention treatment can be advanced to the fetal stage, it can not only save many young lives that have been judged "unfit for birth" in utero, but also minimize the impact of the disease after birth and improve the quality of life
of the "sick child".
Stem cell therapy, known as regenerative medicine, uses stem cells to isolate, culture, and induce differentiation in vitro to cultivate new, normal cells, tissues, and organs and then transplant them into the body to repair or rebuild normal cells and tissues, thereby restoring body function
。 What kind of miracle will be created by using a unique stem cell "patch" to repair the fetus in the mother's womb? On March 2, 2021, with FDA approval, the research team at the University of California, Davis, announced the launch of the world's first clinical trial Cure (Cellular Therapy for In Utero Repair of Myelomeningocele
), which caused quite a stir.
Today, three babies have been born
after the CuRe trial.
Spina bifida, also known as spinal canal insufficiency, is one of the most common neural tube defects, most severe as myelomeningocele (MMC).
Most spina bifida can be avoided
by the mother supplementing with adequate folic acid during pregnancy.
In the United States, about 4 babies are born
every day with spina bifida.
If left untreated, exposed spinal cord can cause severe nerve damage, leading to lifelong impairments
in cognition, mobility, urination, and bowel function.
Previous studies have shown that the impact of spina bifida on the health of patients increases with the extension of spinal cord exposure during pregnancy, and surgery in the second trimester of pregnancy can effectively prevent nerve damage caused by deteriorating spinal conditions, and the prognosis is better than surgery after birth
.
As early as the summer of 2018, a joint team from Great Ormond Street Hospital for Children, University College London Hospital and University College London successfully completed two intrauterine operations on spina bifida fetuses - opening the uterus, exposing the spina bifida, closing the defect, and repairing the uterus
.
Although spina bifida is not completely cured, children are less likely to have disabilities after birth and have a better
quality of life.
But the UC Davis team doesn't want to stop there
.
They used biomaterials to scaffold human placenta-derived mesenchymal stromal cells to make "patch" patches, which cover the fetus-exposed spinal cord and grow with the fetus, hoping to reverse nerve damage and improve fetal motor function
.
Until then, although intrauterine surgery had become the standard of care, "more than half of children with spina bifida could not walk
independently.
" Diana Farmer, the world's first female fetal surgeon, professor and chief of surgery at UC Davis Health Center, and principal investigator of the CuRe trial, said, "There is an urgent need for a treatment that can prevent or reduce the severity of this disease
.
" Figure
1 Stem cell patch repairs spina bifida (Source: UC Davis Health Center official website)
For more than a decade, Farmer has been working on this human clinical trial
of spina bifida.
Farmer invited Wang Aijun, a professor of biomedical engineering and a leader in developing cell therapies to promote tissue regeneration, to set up the Surgical Bioengineering Laboratory to find ways
to use stem cells and bioengineering to improve surgical outcomes and outcomes.
A few years ago, she also founded the UC Davis Children's Hospital Fetal Care and Treatment Center with pediatric surgeon Shinjiro Hirose in preparation for
higher standards of care.
The first phase of the Cure trial received a $9 million grant
from the California Institute for Regenerative Medicine (CIRM), a stem cell institution.
Figure 2 Diana Farmer and Wang Aijun (Source: UC Davis Health Center official website).
The collaboration with the UC Davis School of Veterinary Medicine also confirmed that stem cell therapy is safe and effective in animals: Intrauterine surgery was performed on twin sheep with MMC defects, and the one that received the stem cell patch was born to walk like a normal lamb, while the other that did not receive the stem cell patch could not even stand on its hind legs
.
Researchers believe that it is placental stem cells and many of the important growth factors they secrete — what Farmer calls "miracle stem cell juice" — that protect neurons from death
.
But the team also needs to perform the operation on a more human-like animal model where spina bifida occurs naturally
.
Bulldogs became their choice
.
Myrtle, a tricolored English bulldog, was part of the trial and was part of the trial and left her incontinent and unable to use her hindquarters, and now she is able to walk and climb stairs
.
Figure 3 Myrtle, a pit bull (Source: UC Davis official website).
Animal experiments gave beautiful results, and the FDA subsequently approved the initiation of human clinical trials
of intrauterine stem cells for MMC.
Emily from Austin, Texas, became the first participant
in the trial.
On the same day Emily learned that her developing child had spina bifida, she also heard about the CuRe trial, a "life-saving rope"
she couldn't turn down.
The intrauterine surgery is scheduled for July 12, 2021, when Emily is 25 weeks and 5 days
pregnant.
The surgical stem cell patch was born in a GMP lab and took four days to make
.
Priya Kumar, a scientist at the Center for Surgical Bioengineering, said: "The time we spend pulling out the cells, the time we plant on the stent, the time we hand over to the operating room team, is critical
.
"
Emily, along with a team of 40 people for surgery and cell preparation, accomplished the first time in the
history of medicine.
After general anesthesia, the surgeon makes a small incision in the Emily uterus, allowing the fetus to float near the incision, exposing spina bifida
defects.
Then comes the crucial moment: placing the stem cell patch on
the fetus's bare spinal cord.
The team declared the operation successful
.
"The stem cell patch placement went smoothly
.
Both mother and fetus did a great job! Farmer said
.
On September 20, 2021, 35 weeks and 5 days of gestation, Robbie was born
.
Farmer has been looking forward to this day for a long time, and she expects that if Robbie does not receive treatment, she will be born with leg paralysis
.
Surprisingly, the child began to kick his legs as soon as he was born
.
Farmer recalls still being excited: "I remember me saying, 'Oh my God, I think she's twisting her toes!'" Although the observations do not represent official confirmations, they still show great promise
.
Figure 4 Emily and Robbie 15 days old (Source: UC Davis Health Center website).
reaching conclusions.
They say there is still a lot to learn
during this safe phase of the trial.
The CuRe trial is planned to treat a total of 35 patients
.
They will conduct ongoing testing of the three babies born in the trial to date until age 6 to fully assess the safety and effectiveness of the procedure, especially when the babies are 30 months old, with a focus on their walking and potty training
.
"This experience was more important than life and exceeded all expectations
.
I hope this trial will improve the quality of life for many patients," Emily said, "and we are honored to be a part of
history.
" Written
by| Wind is standing up all night
Typesetting| Qiao Weijun
End
Resources:
[1]https://health.
ucdavis.
edu/news/headlines/worlds-first-stem-cell-treatment-for-spina-bifida-delivered-during-fetal-surgery--/2022/10
