UTHealth Houston fetoscopic spina bifida trial receives NIH funding to evaluate long-term efficacy of the treatment

Ramesha Papanna, MD, MPH, a professor at McGovern Medical School at UTHealth Houston and a maternal-fetal surgeon at UTHealth Houston Fetal Center and Children’s Memorial Hermann Hospital. (Photo by UTHealth Houston)

A five-year, $2.8 million grant was awarded to researchers at UTHealth Houston by the National Institutes of Health to evaluate the long-term effects on patients enrolled in the “Cryopreserved Human Umbilical Cord as a Meningeal Patch in Fetoscopic Spina Bifida Repair” trial.

The study is led by Ramesha Papanna, MD, MPH, a professor at McGovern Medical School at UTHealth Houston and a maternal-fetal surgeon at UTHealth Houston Fetal Center and Children’s Memorial Hermann Hospital.

Funding will allow patients enrolled in the one-of-a-kind fetoscopic spina bifida repair study to be monitored until they are 30 months old.

Researchers will analyze neurological, motor, and developmental outcomes and their correlation with radiologic markers of spinal cord tethering in patients who were part of the study.  

“It’s remarkable that an idea we had 13 years ago has become a therapy improving outcomes for children with spina bifida, helping them walk sooner,” said Papanna, who is internationally recognized for his research on improving outcomes following fetal intervention and investigating methods for the prevention of preterm delivery. “Advancing from bench concept to FDA phase 3 trial — backed by NIH grants — has been extraordinary. This new funding for the efficacy trial, already showing mobility gains, highlights the impact and promise for patients.”  

Spina bifida impacts roughly 1 in every 2,875 births in the U.S. each year. The neural tube defect occurs when the spine and spinal cord fail to close properly during early pregnancy, leading to varying degrees of disability such as leg weakness, paralysis, and bladder or bowel issues. The condition can be categorized into three levels of severity: spina bifida occulta (mild), meningocele (moderate), and myelomeningocele (severe).

While there is no cure, treatment for the condition includes surgery, ongoing medical care, and physical therapy.

The UTHealth Houston team is currently in late-stage testing of the trial after successfully completing an early feasibility trial to establish the safety of the procedure.

Patients enrolled in the trial undergo a fetoscopic procedure that differs from open in utero repair, which requires a large incision on the uterus and delivery by cesarean section. Instead, the fetal intervention team repairs the spina bifida defect in two layers through three small incisions in the uterus using a fetoscope, a high-resolution camera, and tiny surgical tools. The first layer is closed using a cryopreserved human umbilical cord patch placed over the spinal cord, followed by a second layer of primary closure of the skin. Mothers undergo vaginal delivery, unless there is an obstetrical indication for C-section.

Once delivered, a team of experts, who are co-investigators in the trial, schedule appointments for follow-up evaluations at 12 and 30 months old.

The team includes Stephen Fletcher, DO,  the Dr. Marnie Rose Professor in Pediatric Neurosurgery at McGovern Medical School and a pediatric neurosurgeon at UTHealth Houston Fetal Center; Gabriel Anzueto, MD, the Raghuthaman Family Professor in Pediatric Neurology and Developmental and Behavioral Pediatrics at McGovern Medical School and a developmental pediatrician at UT Physicians; and Rajan Patel, MD,  a pediatric neuroradiologist at Texas Children’s Hospital.  

During these appointments, the children undergo comprehensive neurological and developmental assessments, as  well as MRIs of the brain and spinal cord.

“Follow?up is essential, not only for each child’s care but also for the success of the study,” Papanna said. “These evaluations show us how children are progressing after this innovative therapy and help identify both positive outcomes and any areas that may need attention.” 

Additional UTHealth Houston clinical collaborators for the study include McGovern Medical School and the Fetal Center’s KuoJen Tsao, MD, The Children’s Fund, Inc. Distinguished Professor in Pediatric Surgery and a pediatric general and thoracic surgeon; Jimmy Espinoza, MD, professor of obstetrics, gynecology, and reproductive sciences and an OB-GYN; Sami Backley, MD, assistant professor of obstetrics, gynecology, and reproductive sciences and a maternal-fetal medicine specialist; Peter Yang, MD, assistant professor of pediatric neurosurgery and a pediatric neurosurgeon; Jason Au, MD, assistant professor of diagnostic and interventional imaging and a pediatric urologist; and Suzanne Lopez, MD, professor of neonatal-perinatal medicine and a neonatologist. Research collaborators part of the trial includes Lovepreet K. Mann, MBBS, associate professor of obstetrics, gynecology, and reproductive sciences, and Dejian Lai, PhD, professor of biostatics at UTHealth Houston School of Public Health.

Families and physicians looking for more information on how to enroll in the trial can visit the UTHealth Houston Fetal Center’s trial page.