By Riley Szenasi
SACRAMENTO, CA-– UC Davis Health recently conducted the first successful spina bifida treatment in a trial known as the “Cellular Therapy for In Utero Repair of Myelomeningocele” or the “CuRe Trial.”
The Mayo Clinic defines spina bifida, or myelomeningocele (the most serious type), as a “birth defect that occurs when the spine and spinal cord don’t form properly” and “can range from mild to severe, depending on the type of defect, size, location and complications.”
Spina bifida has long been a health concern in the U.S. The Fetal Health Foundation states the defect affects “1,500 to 2,000 children in the U.S. every year.” It is typically diagnosed through blood tests and an ultrasound, as early as the second trimester of pregnancy.
UC Davis Health’s article on the success of the trial mentions that Diana Farmer, one of the lead researchers in this treatment project, has worked in multiple clinical trials targeting the effects of spina bifida. Her decision to reach out to Aijun Wang, a successful biomedical engineer, proved important in her research.
Wang’s career has focused on “developing technologies that combine stem cell engineering and biomaterial engineering to promote tissue regeneration.” Wang helped Farmer establish the UC Davis Health Biomedical Engineering Laboratory to further their stem cell exploration.
Tricia Tomiyoshi, the Senior Public Information Officer at UC Davis Health, notes that it was a 10-year process that allowed researchers to reach a point where they could begin the treatment of human fetuses. With further progress made, they began using animal patients in their clinical trials.
She states that the researchers delivered healthy lambs, bulldogs, and other animals that were able to walk “without noticeable disability.” After monitoring the animals for the months following their birth, Farmer’s research team proved that the procedure was successful as a permanent cure. According to UC Davis Health, the team began working with human patients with the “CuRe Trial” in the spring of 2021.
In the past, spina bifida has been treated through surgery performed in utero or shortly after birth, though it has been noted that these surgical techniques have not proven entirely effective. It’s for this reason that research on alternative treatments of spina bifida continues to be necessary.
As an alternative method, the “CuRe Trial” focuses on the reversal of “paralysis and other abnormal functions of spina bifida before birth.” For this trial, it was important to treat patients as fetuses, as spinal damage typically worsens as the pregnancy progresses. To prevent this damage, the research team used stem cells derived from human “mesenchymal stromal cells” to treat the fetuses while in the womb.
According to UC Davis Health, the “stem cell patches” are made through a four-day procedure in which the medical lab team “manufactures clinical grade stem cells – mesenchymal stem cells – from placental tissue in the UC Davis Health’s CIRM (California Institute for Regenerative Medicine) -funded Institute for Regenerative Cures.” As explained by Tomiyoshi, the stem cells work to “repair and restore damaged spinal tissue, beyond what surgery can accomplish alone.”
The participants included a total of 35 patient volunteers. To be a part of the trial, they must move to Sacramento for the surgery and agree to return for follow-up appointments to ensure that the treatment worked as a permanent cure and that no new issues arise following the birth.
The first participant in the trial, Emily, had her surgery in July 2021, during her second trimester.
The UC Davis Health website describes her procedure as having gone seamlessly. The medical team applied “the stem cell patch…directly over the exposed spinal cord of the fetus…then closed the incision to allow the tissue to regenerate.”
Emily’s baby, Robbie, was born on Sept. 20, 2021, the first to be born with this treatment. Farmer was present during the delivery and claimed that it was clear upon birth that the surgery was successful as “the minute she was born that she was kicking her legs.”
Currently, three of the 35 patients have been born, showing no signs of defects related to spina bifida. The UC Davis Health team awaits the arrival of the remaining patients in the coming months.
To confirm the success of the treatment, the babies involved in the trial will be given a 30-month check-up and will continue to be observed closely for the first six years of their lives.