Hospital Begins 1st Stem Cells Approved Study to Treat Hearing Loss

First FDA-approved study of Stem Cells to treat hearing loss begins at Children's Memorial Hermann Hospital in Houston, TX.

What does it mean to be born with a hearing loss and grow up in a world of muffled sounds or silence? Unfortunately, children born with a hearing loss may never have speech and language skills equal to their hearing peers.

According to the National Institute on Deafness and Communication Disorders (NIDCD) 1 in every 350 infants is born with a significant hearing loss. Hearing loss occurs more often than any other medical condition for which newborn screening is available.

To date, no treatment is available to reverse or repair an acquired sensorineural hearing loss. Recently, the FDA approved the first Phase I trial that will evaluate the safety of using a child’s own cord blood stem cells to regenerate cells in the inner ear and potentially restore a child’s hearing. To ensure consistency in cord blood stem cell processing, storage, and release for infusion, CBR is the only stem cell bank providing clients for the study. The year-long study will follow 10 children, ages 6 weeks to 18 months, who have a moderate to profound hearing loss. Children who are deaf as a result of a genetic anomaly or syndrome are not eligible. Those who meet the eligibility criteria will be infused with their own stem cells and subsequently receive regular medical and audiology follow ups to evaluate the safety and efficacy of the treatment. The study participants will continue to benefit from their prescribed hearing aids, attend special education programs and have regular speech and language therapy.

Child development researchers have determined that there is a critical window for children to develop speech and language. Early identification of a hearing loss is imperative so that educational therapy can capitalize on the child’s formative years for developing language. Currently, families rely on specialized educational programs and either a cochlear implant or hearing aids to help their hearing impaired child learn language.

Research on the use of autologous human cord blood stem cells has emerged as a potential treatment for treating acquired sensorineural hearing loss. Roberto Revoltella and his colleagues at the Biomedical Institute reported that a variety of stem cell populations can induce repair or replace hair cells in the cochlea, the area of the inner ear that is typically damaged in a sensorineural loss. Experiments utilizing an animal model demonstrated inner ear hair cell regrowth followed an umbilical cord blood stem cell transplant in animals with an acquired sensorineural loss induced by ototoxic drugs and noise damage. Results of the study appear in Cell Transplantation, Vol 17.

Physicians and researchers at Children’s Memorial Hermann Hospital are hopeful that this Phase I trial will demonstrate that there is a viable medical treatment for a sensorineural hearing loss in young children. The Principal Investigator of the clinical trial is 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. “Currently, the only treatment options for sensorineural hearing loss are hearing aids or cochlear implants,” Dr. Fakhri said. “We hope that this study will open avenues to additional treatment options for hearing loss in children.”

Written by Linda Baumgartner, MS, CCCSLT, LSLS, Cert. AVT, auditory-verbal therapist and co-investigator on the trial.