HOUSTON – (Nov. 27, 2018) - A new clinical trial at The University of Texas Health Science Center at Houston (UTHealth) is the first in the world to investigate whether infusing an infant’s own umbilical cord blood cells, or autologous cells, can protect the brain, mitigating the risk of neurodevelopmental challenges in babies with severe congenital diaphragmatic hernia (CDH).
CDH occurs when the diaphragm, which separates the chest from the abdomen and controls the lungs’ ability to inhale and exhale, fails to form normally, allowing abdominal organs into the chest cavity. This crowding can result in abnormal lung growth. CDH affects 1 in 2,500 children born in the U.S., with about half of all cases considered severe. Severe CDH carries a 50 percent chance of death, along with an increased risk for brain abnormalities and delays that may be caused by low oxygen levels within the first few weeks of life.
“Currently, there is no treatment that protects infants with CDH, who otherwise may have normal brain function, from neurological injury,” said Matthew Harting, MD, assistant professor in the Department of Pediatric Surgery at McGovern Medical School at UTHealth. “I am fortunate and honored to be able to offer these patients and their families a novel therapy, an investigational new drug being evaluated in a clinical trial specifically to study the benefits of cord blood cell therapy in infants born with CDH. Simply surviving is not good enough anymore – we are striving to help CDH kids reach their full potential and highest quality of life with new therapies.”
The phase one clinical trial, being conducted under the auspices of the regulatory framework of the U.S. Food and Drug Administration (FDA), will assess the safety and efficacy of the therapy, measuring neurological and developmental outcomes. The research will also assess the potential impact cellular therapy has on the progression of pulmonary hypertension, or high blood pressure in the lungs, which is a common challenge faced by CDH patients in the newborn period.
The stem cells extracted from the child’s own umbilical cord blood will be reinfused in up to four doses over a six-day period after birth. The cells are isolated, processed, and quality-controlled within the Evelyn Griffin Stem Cell Laboratory, a UTHealth FDA-registered and FACT-accredited CGMP facility. The study is supported by a Texas Medical Center-sponsored clinical trial grant. The research team will follow the child’s progress until hospital discharge and at routine clinic follow-up visits for the first two years of life.
The interdisciplinary CDH team at McGovern Medical School is affiliated with Children’s Memorial Hermann Hospital, and the experts involved in the care of the complex disease include obstetricians, maternal-fetal medicine specialists, neonatologists, pediatric surgeons, pediatricians, nutritionists, nurses, and more. The team works in collaboration with The Fetal Center at UTHealth and Children’s Memorial Hermann Hospital.
“This trial is just one of the ways we push the envelope to provide the most cutting-edge care for our CDH patients, who wouldn’t be able to receive this specialized treatment anywhere else,” Harting said.
For more information about this trial, visit: https://clinicaltrials.gov/ct2/show/NCT03526588?term=Harting&cond=CDH&rank=1.
Established in 1972 by The University of Texas System Board of Regents, The University of Texas Health Science Center at Houston (UTHealth) is Houston’s Health University and Texas’ resource for health care education, innovation, scientific discovery and excellence in patient care. The most comprehensive academic health center in the UT System and the U.S. Gulf Coast region, UTHealth is home to Jane and Robert Cizik School of Nursing, John P. and Kathrine G. McGovern Medical School, and schools of biomedical informatics, biomedical sciences, dentistry, and public health. UTHealth includes The University of Texas Harris County Psychiatric Center, as well as the growing clinical practices UT Physicians, UT Dentists, and UT Health Services. The university’s primary teaching hospitals are Memorial Hermann-Texas Medical Center, Children’s Memorial Hermann Hospital, and Harris Health Lyndon B. Johnson Hospital. For more information, visit www.uth.edu.