Children's Hospitals Today - Spring 2023 - 6

ROUNDS / INNOVATION
Finding
a cure
A baby with spina bifida is
born after a one-of-a-kind
treatment in utero.
BY TRICIA TOMIYOSHI
T
hree babies have been born after
receiving the world's first spina
bifida treatment combining surgery
with stem cells. This was made possible
by a clinical trial at UC Davis Children's
Hospital in Sacramento, California. The
one-of-a-kind treatment, delivered while
a fetus is still developing in the mother's
womb, could improve outcomes for
children with this congenital disorder.
Launched in the spring of 2021, the clinical
trial is known formally as the " CuRe
Trial: Cellular Therapy for In Utero
Repair of Myelomeningocele. " Thirty-five
patients will be treated during the trial.
The three babies from the trial who have
been born so far will be monitored by the
research team so they can fully assess
the procedure's safety and effectiveness.
Spina bifida, also known as myelomeningocele,
occurs when spinal tissue
fails to fuse properly during the early
stages of pregnancy. The condition can
lead to a range of lifelong cognitive,
mobility, urinary and bowel disorders.
It affects 1,500 to 2,000 children in the
U.S. every year, and it is often diagnosed
through ultrasound. While surgery
performed after birth can help reduce
some of the effects, surgery before birth
can prevent or lessen the severity of the
fetus's spinal damage, which worsens
over the course of pregnancy. " I've been
working toward this day for almost 25
years now, " says Diana Farmer, M.D.,
the world's first woman fetal surgeon,
surgeon-in-chief at UC Davis Children's
Hospital, professor and chair of surgery
6
CHILDREN'S HOSPITALS TODAY Spring 2023
Robbie was the first baby born in a clinical trial for a new spina bifida treatment.
at UC Davis Health and the principal
investigator on the study.
Long path to success
As a leader of the Management of
Myelomeningocele Study (MOMS) clinical
trial in the early 2000s, Farmer had
previously helped to prove that fetal
surgery reduced neurological deficits
from spina bifida. Many
children in that study
showed improvement but
still required wheelchairs
or leg braces.
Farmer recruited bioengineer
Aijun Wang to
help take that work to
the next level. Together,
they launched the UC
Davis Health Surgical
Bioengineering Laboratory
to find ways to use stem cells and
bioengineering to advance surgical
effectiveness and improve outcomes.
Farmer also launched the UC Davis
Fetal Care and Treatment Center with
fetal surgeon Shinjiro Hirose and the UC
Davis Children's Surgery Center several
years ago.
Farmer, Wang and their research
team have been working on their novel
approach using stem cells in fetal
surgery for more than 10 years. Over that
time, animal modeling has shown it is
capable of preventing the paralysis associated
with spina bifida. It is believed
that the stem cells work to repair and
restore damaged spinal tissue, beyond
what surgery can accomplish alone.
Preliminary work by Farmer and
" I'VE BEEN
WORKING
TOWARD THIS DAY
FOR ALMOST 25
YEARS NOW. "
Wang proved that prenatal surgery
combined with human
placenta-derived mesenchymal
stromal cells,
held in place with a biomaterial
scaffold to form
a " patch, " helped lambs
with spina bifida walk
without noticeable disability.
" When the baby
sheep who received stem
cells were born, they were
able to stand at birth and
they were able to run around almost
normally. It was amazing, " Wang says.
When the team refined their surgery
and stem cell technique for canines, the
treatment also improved the mobility
of dogs with naturally occurring spina
bifida. A pair of English bulldogs named
Darla and Spanky were the world's first
dogs to be successfully treated with
surgery and stem cells. Spina bifida,
a common congenital disorder in this
Children's Hospitals Today - Spring 2023

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