The world's first successful operation on a "fetus" was successful

After receiving the world's first spina bifida surgery combined with stem cells, three babies were
born.
That's thanks to a landmark clinical trial
at the University of California, Davis.
This unique treatment, administered while the fetus is still developing in the mother's womb, could improve the prognosis of
children with this birth defect.
The clinical trial was launched in the spring of 2021 and is officially titled "Cure Trial: Cell Therapy for Intrauterine Repair of Myelomeningocele.
"
A total of 35 patients were treated
.
The three trial babies who have been born so far will be monitored by the research team up to 30 months of age to fully evaluate the safety and efficacy
of the procedure.

The first phase of the trial was funded
by a $9 million state grant from the California Institute for Regenerative Medicine (CIRM), the California stem cell agency.

"This clinical trial could improve the quality of life for a lot of patients," said Emily, who was the first clinical trial participant
to travel from Austin, Texas.
Her daughter, Robbie, was born
last October.
"We didn't know spina bifida
until it was diagnosed.
We are grateful to be a part of it
.
We are giving our daughter the best chance to have a bright future
.
”

Spina bifida, also known as myelomeningocele, occurs early in pregnancy and spinal tissue does not fuse
properly.
This birth defect can lead to a lifelong range
of cognitive, mobility, urinary and bowel dysfunction.
Between 1,500 and 2,000 children in the United States suffer from the disease
each year.
Diagnosis is usually by
ultrasound.
While surgery performed after birth can help reduce some of the effects, prenatal surgery can prevent or reduce the severity of spinal damage to the fetus, which worsens during pregnancy
.

"I've been fighting for this day for nearly 25 years," said Diana Farmer, the world's first fetal surgeon, professor and chief of surgery at UC Davis and principal investigator
of the study.

The path to future healing

As a clinical trial leader in the early 21st century for myelomeningocele management research, Farmer previously helped demonstrate that fetal surgery can reduce neurological deficits
in spina bifida.
Many of the children in this study had improvements but still needed wheelchairs or leg braces
.
Farmer hired bioengineer Aijun Wang to help the work move to the next stage
.
Together, they founded the UC Davis Health Surgery Bioengineering Laboratory to find ways to
use stem cells and bioengineering to enhance surgical outcomes and outcomes.
A few years ago, Farmer also founded the UC Davis Fetal Care and Treatment Center with fetal surgeon Shinjiro Hirose and the UC Davis Center
for Pediatric Surgery.
Farmer, Wang and their research team have been working on a new method of using stem cells in embryonic surgery for more than 10 years
.
During this time, animal models have shown it to prevent paralysis caused by spina bifida
.
It is believed that the role of stem cells is to repair and restore damaged spinal tissue, which cannot be done with surgery alone
.
Farmer and Wang's preliminary work demonstrated that prenatal surgery combined human placenta-derived mesenchymal stromal cells, fixed in place with biomaterial scaffolds to form "patches," helped lambs with spina bifida walk without obvious disability
.

"When baby sheep receiving stem cells are born, they can stand up as soon as they are born and can run
around almost normally.
It's
incredible.
While the team improved their surgical and canine stem cell techniques, the treatment also improved mobility in dogs with natural spina bifida
.
An English pit bull named Dalla and Spanky was one of the first dogs
in the world to successfully undergo surgery and stem cell therapy.
Spina bifida is a common birth defect that often makes their hindlimbs less functional
.
At post-operative follow-up at 4 months of age, Dalla and Spanky were able to walk, run and play
.

The world's first human trial

When Emily and her husband Harry learned that they would be parents for the first time, they never expected pregnancy complications
.
But the day Emily learned that her developing child had spina bifida was also the day
she first heard about the CuRe trial.
For Emily, it was a lifesaver
they couldn't turn down.
Participating in the trial meant she needed to temporarily move to Sacramento for fetal surgery and then have weekly follow-up
visits during her pregnancy.

After screening, MRI scans, and interviews, Emily received life-changing news: she was accepted for the trial
.
Her fetal surgery was scheduled for July 12, 2021, when she was 25 weeks and 5 days
pregnant.

The laboratory is a GMP laboratory
for safe use by humans.
It was here that they made a stem cell "patch"
for Emily's fetal surgery.

"It takes four days to make the stem cell patch," said
Pria Kumar, a scientist at the Center for Surgical Bioengineering.
He leads a team responsible for making stem cell patches and transporting them to the operating room
.
"The timing of our removal of the cells, the time to sow on the scaffold, and the timing of delivery are critical
.
"

For the first time in the history of medicine

After Emily received general anesthesia, a small opening was made in her uterus, and they floated the fetus to the incision so they could expose the fetus's spine and spina bifida defects
.
The surgeon uses a microscope to carefully begin the repair
.
The moment of truth arrived: the stem cell patch was applied directly to
the exposed spinal cord of the fetus.
The fetal surgeon then closes the incision and allows the tissue to regenerate
.

The team announced that this was the first successful operation
.

Production day

On September 20, 2021, after 35 weeks and 5 days of gestation, Robbie was born by caesarean section, weighing 5 pounds, 10 ounces, and 19 inches long
.

"One of my initial fears was that I might not see her, but they brought her to me
.
For the first time, I saw her toes move
.
It's so reassuring and a bit out of this world
.
”

For Farmer, this day is what she has been waiting for for a long time, and it brings surprises
.
If Robbie had not been treated, she could have been born paralyzed
in her legs.

"It was clear that she was kicking the moment she was born, and I remember saying very clearly, 'Oh my God, I feel like she's wiggling her toes!'" Farmer said
.
He noted that this observation has not been officially confirmed, but it is promising
.
"It's amazing
.
We kept saying, 'Did I see that?' Is this true?'"

Mom and baby are at home and in good health
.
Robbie had just celebrated her first birthday
.

The CuRe team is cautious about drawing conclusions and says there's still a lot to learn
at this safe stage of the trial.
The team will continue to monitor Robbie and the other babies enrolled in the trial until they reach age 6, undergoing critical tests at 30 months of age to see if they can walk and potty train.

"This experience transcends life and exceeds all expectations
.
I hope this trial will improve the quality of life
of more patients.
" We are honored to be a part of
the history that is being made.
”