Cord Blood Stem Cell Infusion for Children with Acquired Hearing Loss

During the last 25 years, stem cells derived from umbilical cord blood have been used in more than 30,000 transplants performed worldwide for the treatment of over 80 diseases and disorders in both adults and children. The evolution of stem cell therapies has paved the way for further research being conducted through FDA-regulated clinical trials to uncover their potential in regenerative medicine applications.

With this potential in mind, researchers at Florida Hospital for Children are conducting an FDA-regulated clinical trial to determine the safety and feasibility of using a child's stem cells derived from their own umbilical cord blood as a treatment for sensorineural hearing loss. The study is supported by Cord Blood Registry®.

In the United States, approximately 15% of children suffer from low or high-frequency hearing loss (1). The most common type of hearing loss, especially at high frequencies, is sensorineural. Acquired sensorineural hearing loss results from damage to hair cells in the inner ear (cochlea) and can be caused by illness, medication, noise exposure, birth injury, or head trauma. A child's ability to hear affects the development of language skills, and hearing impairments can lead to poor academic and social development (2).

Hearing aids and cochlear implants have become welcome standards in assisting children with acquired hearing loss, but these devices don't actually repair associated damage to the cochlea, cochlear nerve or brain stem. Aiming to restore normal hearing without the use of a prosthesis, the study will investigate whether using cord blood stem cells to help trigger the body's own repair mechanisms could provide a non-invasive therapeutic option for acquired hearing loss that does not exist today. The trial follows promising evidence from preclinical studies suggesting that the infusion of human umbilical cord stem cells may help repair damaged cells in the inner ear in ways that could lead to hearing improvement.

The study has a primary objective of determining the safety of using cord blood stem cells in a select pediatric patient population while also assessing whether this approach to treatment improves inner ear function, speech, and language development. Researchers will follow 10 children, ranging in age from 6 weeks to 6 years, who have been diagnosed with acquired hearing loss for less than 18 months and who have their own cord blood unit processed and stored under Cord Blood Registry's strict quality controls. Children with a known genetic cause of deafness are ineligible for study participation. Patients will receive one intravenous infusion of their own umbilical cord blood stem cells. All patients will return for follow-up at 1 month, 6 months, and 1 year post-treatment. To date, three children have been treated and six more are in the enrollment process.

A growing body of research provides evidence that stem cells derived from cord blood can be used to repair damaged or diseased tissue or organs. Cord Blood Registry is the only family cord blood bank to partner with leading research institutions to establish FDA-regulated clinical trials exploring the potential regenerative ability of newborn stem cells to help treat conditions that have no cure today. The results of the acquired hearing loss study and others may spur a new era in treating common neurological conditions impacting children and adults.

References

1. Niskar, AS, et al. Prevalence of hearing loss among children 6 to 19 years of age: the Third National Health and Nutrition Examination Survey. JAMA.1998; 279: 1071-1075.
2. Tierney, CD, et al. Development of children who have hearing impairment. Pediatr Rev 29, e72-73; discussion e73 (2008)

James & Linda Baumgartner are a husband and wife team who are co-principal investigators of the FDA-regulated clinical trial entitled "Safety of Autologous Stem Cell Treatment for Acquired Hearing Loss in Children". James Baumgartner, MD, serves as surgical director of the Comprehensive Pediatric Epilepsy Center at Florida Hospital for Children. Dr. Baumgartner is a board-certified pediatric neurosurgeon, specializing in traditional and minimally invasive surgical procedures for children and adolescents who are suffering from epilepsy or seizure-related disorders. Linda Baumgartner, MS, CCC-SLP, LSLS Cert. AVT, is a speech-language pathologist and a certified auditory-verbal therapist practicing in Orlando, Florida. Ms. Baumgartner's specific areas of expertise include language acquisition, pediatric hearing loss, as well as language and speech assessment.
Florida Hospital for Children is a full-service facility served by more than 130 pediatric specialists and a highly trained pediatric team of more than 1,000 caregivers and staff. This unique children's hospital provides patients with private, family-centered pediatric rooms, a dedicated pediatric emergency department and an Advanced Center for Pediatric Surgery. The Walt Disney Pavilion at Florida Hospital for Children, in Orlando, officially opened in March 2011, but Florida Hospital has been caring for children for more than 100 years.