American Journal of Case Reports and Clinical Images
The new technique attempts to heal nerve damage
caused by spina bifida, a disabling birth defect. In this condition, the bony
tissue of a fetus’s spine doesn’t knit together properly around the spinal
cord. That can cause a kaleidoscope of medical issues, including lifelong
paralysis and bladder and bowel problems.
Traditional fetal surgery to patch up the spine can
limit the scope of these problems — but it does not repair nerve damage that
has already occurred. Adding living stem cells to the procedure might.
At least, that’s the goal of fetal surgeon Diana
Farmer’s team. So far, the approach appears to be safe, the researchers
reported earlier this year in theLancet. In six fetal patients with severe
spina bifida, applying a stem cell–loaded patch to their exposed spinal cords
did not cause infection, tumor growth or interfere with healing. That’s
important because “no one knew what stem cells would do inside a fetus,” says
Farmer, of the University of California, Davis.
For now, the vital question — whether the technique
mends fetal spinal cords — remains unanswered. That’s because researchers are
still performing follow-up assessments of the patients, who are now toddlers.
At this stage, it’s too early to say how well the surgery worked, and Farmer is
careful not to speculate. “If we could get every kid to not be in a
wheelchair,” she says, “that would be fantastic.” But the team won’t know for a
few years. Until then, Farmer says, she doesn’t want to give people false hope.
In some ways, this study represents “a seismic
shift” in the field, says Ramen Chmait, director of Los Angeles Fetal Surgery
at the University of Southern California, who was not involved with the work.
If the technique pans out, he says, it “could be a huge, important step in
modern-day medicine.”
Maternal fetal medicine specialist Magdalena Sanz
Cortes echoes the sentiment in a commentary that accompanied Farmer’s study.
“We eagerly await follow-up results and definitive studies that might show
benefit,” writes Sanz Cortes, of Baylor College of Medicine in Houston. “Such
results would herald a new era in fetal surgery.”
First, doctors will need to better understand the
procedure’s risks and benefits. At this point, Chmait says, Farmer’s team has
taken the “first step of a marathon.”
Spina bifida can be repaired in the womb, but
there’s room for improvement.
In the United States, roughly 1 in every 2,800
babies are born with spina bifida. This abnormality leaves the delicate spinal
cord exposed in the womb. Without the bony protection of the vertebrae, the
spinal cord can bulge through the back, making it especially vulnerable to
injury. Like a chemical that burns, amniotic fluid washing over the open spinal
cord can degrade it. And as the baby grows, it rubs against the walls of the
uterus, damaging unprotected nerve cells.
Beyond paralysis and other serious problems, this
damage can cause fluid to build up in the brain. Some babies require a shunt
surgically implanted in the days or weeks after birth for drainage. That can be
lifesaving, but it’s also lifelong ¬— a permanent implant that can malfunction
or spur infection.
One way to avoid the shunt, and potentially some of the nerve damage accrued during pregnancy, is to surgically close the hole in the fetus’s spine in the womb. That was the conclusion of a landmark clinical trial 15 years ago that compared surgery done before and after birth. Prenatal surgery cut the need for shunts in half and doubled the chance of being able to walk without leg braces or other devices, Farmer’s team reported in the New England Journal of Medicine.