New Clinicals Trials Use Umbilical Cord Blood Stem Cells to Treat Neurological Conditions and Hearing Loss


Umbilical cord and umbilical cord blood contains a wealth of stem cells for multiple uses. Cord blood contains a blood cell-making stem cell that can be used to constitute bone marrow (see Gluckman E., Blood Rev. 2011;25(6):255-9). It also contains two mesenchymal stem cell populations: Wharton’s Jelly Mesenchymal Stem Cells (WJ-MSCs) and Human Umbilical Cord Perivascular Cells (HUCPVCs). Both of these cell populations have remarkable potential or regenerative medicine (Carvalho MM., et al. Curr Stem Cell Res Ther. 2011;6(3):221-8). Other umbilical cord stem cells include unrestricted somatic stem cells (USSCs; Arien-Zakay H, Lazarovici P, Nagler A., Best Pract Res Clin Haematol. 2010;23(2):291-303), embryonic-like stem cells, blood vessel-based endothelial stem cells, and a stem cell that comes from those cells that cover tissues (epithelial stem cells; see Harris DT., Stem Cell Rev. 2008;4(4):269-74, & Harris DT., Br J Haematol. 2009;147(2):177-84).

The usefulness of cord blood has been recognized by the medical community for some time, and there are now umbilical cord blood registries that bank cord blood for medical use and for research. One of these registries, the Cord Blood Registry (CBR) works with various research laboratories to help discover ways to use a child’s own cord blood stem cells to treat conditions like pediatric brain injury or even acquired hearing loss. Because different laboratories use different protocols or equipment to process umbilical cord blood, the experimental results derived from experiments or clinical trials that use cord blood might vary widely. Therefore, to ensure consistency in the storage and processing of cord blood stems, three separate clinical trials have used cord blood that provided by the CBR in their FDA-authorized protocols. These research institutions include the University of Texas Health Science Center at Houston (UTHealth) in partnership with Children’s Memorial Hermann Hospital, and Georgia Health Sciences University, which is the home of the Medical College of Georgia (MCG). CBR is the only family stem cell bank that pairs researchers conducting clinical trials with prospective patients for their studies.

Heather Brown, MS, CGC, Vice President of Scientific & Medical Affairs at Cord Blood Registry, put it this way: “Partnering with a series of specialists who want to research the use of a child’s own newborn blood stem cells on a variety of disease states allows CBR to help advance medical research for regenerative therapies by connecting the child whose family banked with CBR to appropriate researchers. The pediatric specialists from UTHealth, Children’s Memorial Hermann Hospital, and Georgia Health Sciences University are at the forefront of stem cell research as they evaluate cord blood stem cells’ ability to help facilitate the healing process after damage to nerves and tissue.”

One of the clinical trials examined the ability of cord blood stem cells to treat hearing loss. Hearing loss can result from problems with the middle ear, which conducts sound to the cochlea (conductive hearing loss) or from problems with the inner ear, in which the cochlea itself is damaged or defective (sensorineural hearing). Sensorineural hearing loss affects approximately 6 per 1,000 children by 18 years of age, with 9% of the cases resulting from various external causes (e.g., viral infection and head injury). Samer Fakhri, M.D., surgeon at Memorial Hermann-Texas Medical Center and associate professor and program director in the Department of Otorhinolaryngology – Head & Neck Surgery at UTHealth, heads the research team investigating the use of cord blood to treat sensorineural hearing loss. His collaborator is James Baumgartner, M.D.

The Fakhri-Baumgartner study is a Phase I safety study that uses cord blood-based stem cells to treat children who suffer from acquired hearing loss. The inspiration for this trial comes from animal studies that used cord blood to repair damaged organs in the inner ear. The paper (Revoltella RP., et al., Cell Transplant. 2008;17(6):665-78), used mice that had been made deaf from treatment with aminoglycoside antibiotics, which cause irreversible deafness at particular dosages, and intensely loud noises, which also cause deafness. Intravenous administration of hematopoietic stem cells from umbilical cord blood stimulated some structural recovery in the inner ear that was due to umbilical cord stem cells that had survived and become part of the inner ear tissues.

Parents of children 6 weeks to 2 years old that had experiences hearing soon after birth are eligible for this year-long study. Baumgartner explains, “The window of opportunity to foster normal language development is limited. This is the first study of its kind with the potential to actually restore hearing in children and allow for more normal speech and language development.”

Another clinical trial is examining the ability of cord blood to treat brain trauma. Children who experience brain injury heal better than adults who experienced the same injury. Having said that brain trauma is one of the leading causes of childhood death. Charles S. Cox, M.D., distinguished professor of pediatric surgery and pediatrics at UTHealth, initiated a clinical study that will enroll 10 children ages 18 months to 17 years old, all of whom have umbilical cord blood banked with CBR, and have suffered some type of traumatic brain injury. These children will enroll in the study within 6-18 months of suffering brain injury. This trial grows from a growing corpus of studies that have demonstrated the efficacy of umbilical blood stem cells to treat neurological conditions. Read more about the trial here.

According the Charles Cox, “The reason we have become interested in cord blood cells is because of the possibility of autologous therapy, meaning using your own cells. And the preclinical models have demonstrated some really fascinating neurological preservation effects to really support these Phase 1 trials. There’s anecdotal experience in other types of neurological injuries that reassures us in terms of the safety of the approach and there are some anecdotal hints at it being beneficial in certain types of brain injury.”

James Carroll professor and chief of pediatric neurology at the GHSU in Augusta, Georgia, launched the first FDA-regulated clinical trial to test the ability of cord blood stem cell infusions to improve the condition of children with cerebral palsy. This clinical trial will include 40 children whose parents have banked their umbilical cord blood at CBR and meet all the criteria for inclusion in the trial.

Dr. Carroll explains: “Using a child’s own stem cells as a possible treatment is the safest form of stem cell transplantation because it carries virtually no threat of immune system rejection. Our focus on cerebral palsy breaks new ground in advancing therapies to change the course of these kinds of brain injury–a condition for which there is currently no cure.”

Brain injuries or lack of oxygen either before birth, during birth, or during the first years of life can damage specific motor pathways in the brain and lead to an inability properly move, learn, hear, see, or think normally. According to the Centers for Disease Control, 2-3 / 1,000 children are affected by cerebral palsy.

These clinical trials are part of an innovative push that partners clinical researchers with patients. They also represent a move from preclinical studies with cord blood stem cells in animals, to human clinical trials with genuine human patients. Heather Brown put it this way: “The benefits of cord blood stem cells being very young, easy to obtain, unspecialized cells which have had limited exposure to environmental toxins or infectious diseases and easy to store for long terms without any loss of function, make them an attractive source for cellular therapy researchers today. We are encouraged to see interest from such diverse researchers from neurosurgeons to endocrinologists and cardiac specialists.”