Stem Cell Breakthrough Helped Babies With Spina Bifida Defy Expectations
SACRAMENTO, Calif. — Diagnosed before birth with a spinal defect, three babies have kicked their legs, wiggled their toes and blown away their parents and a team of researchers at UC Davis Health who developed a novel approach to treat them.
While still in the womb, each child was diagnosed with spina bifida, a condition that often leaves a section of a newborn’s spinal cord exposed and unprotected by the backbone. Babies born with the defect can have intellectual and physical disabilities that range from mild to severe, depending on how big of a hole they have in their spines, where it is and the impact on the spinal cord or nerves.
For 25 years now, Dr. Diana Farmer has been working on ways to treat this prenatal defect. She launched a clinical trial in the spring of last year on a new treatment that pairs surgery with stem cell implantation. Early results have left her enthused, though she said there’s still a long way to go.
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“The most exciting thing was a baby came out, kicking her legs and wiggling our toes, which really exceeded expectations,” said Farmer, chair of surgery at UC Davis Health and principal investigator on the study. “It was very clear the minute she was born that she was kicking her legs, and I remember very clearly saying, ‘Oh my God, I think she’s wiggling her toes.'”
Right now, Farmer said, this initial stage of the clinical research is about showing that the surgery and stem cell treatment are safe. UC Davis plans to enroll 35 moms-to-be in this trial.
“We could not do any of this work any of this discovery, without the partnership of the women who have been willing to, frankly, in a way risked their lives for the advancement of their child and science,” she said.
The first baby and mother to go through the procedure have been identified only as Robbie, now a little over 12 months old, and Emily from Austin, Texas. Robbie was born Sept. 20, 2021, via C-section at 35 weeks and five days gestation. She weighed 5 pounds, 10 ounces and measured 19 inches long.
“She shouldn’t be able to move anything below the hips,” Emily said in a video that UC Davis Health released, “so to have hips and knees and ankles and toes, and to have good strength in all of them, too, is pretty, pretty wild. pretty unreal.”
Farmer was one of the pioneers in using fetal surgery to reduce the neurological deficits caused by spina bifida. In the early 2000s, she participated in a clinical trial that showed that fetal surgery could reduce the neurological deficits caused by spina bifida. While many children from that study showed improvement, they still required wheelchairs or leg braces.
Farmer continued to explore ways to improve outcomes and recruited bioengineer Aijun Wang to determine whether the use of stem cells or other methods of bioengineering could achieve new breakthroughs. Farmer also teamed with fetal surgeon Shinjiro Hirose to further advance the field of fetal surgery in utero and during the birth process.
How team tested stem cell treatment
Wang and Farmer led a team who spent more than 10 years studying the use of human stem cells in sheep and dogs, searching for the right stem cells to use, the best way to implant them and the best surgical techniques.
“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 said.
They later tried their techniques on a pair of English bulldogs: Darla and Spanky. Spina bifida is a common birth defect in this breed, often leaving the dogs with little function in their hindquarters. Darla and Spanky were able to walk, run and play by the time of their post-surgery re-check at four months.
Wang and Farmer were able to move into safety studies in humans after getting this and other research under their belt and after securing a $9 million grant from the California Institute for Regenerative Medicine, the stem cell agency voters created in 2004 through Proposition 71.
UC Davis trial helped Texas mom
New mother Emily said she and her husband Harry did not expect any pregnancy complications when they learned they were expecting their first child. Then came the news about Robbie’s condition. Fortunately, that same day, Emily also learned about the UC Davis Health trial.
It’s formally known as the “CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele.” Pronounced my-low-ma-nin-jo-seal, it is the most serious type of spina bifida and it’s what Robbie had.
With this condition, a part of the baby’s spinal cord and nerves sit in a sac of fluid that pokes out of an opening in the back. Even when repaired with fetal surgery, the condition still can affect how people who had it go to the bathroom, whether they have feeling in their legs or feet, and whether they can move their legs.
Faced with this prognosis for her daughter, Emily said the decision to take the risk of surgery was easy to make. She ended up having to make a temporary move to Sacramento for the fetal surgery and then for weekly follow-up visits during her pregnancy.
The researchers are collecting data on Robbie and the other babies they treat and will release the information on how they are doing when the study reaches certain milestone dates. They plan to follow the children for six years.
“This experience has been larger than life and has exceeded every expectation. I hope this trial will enhance the quality of life for so many patients to come,” Emily said. “We are honored to be part of history in the making.”
© 2022 The Sacramento Bee
Distributed by Tribune Content Agency, LLC
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