Safety of Autologous Stem Cell Infusion for Children With Acquired Hearing Loss

There is currently no treatment available to repair/reverse acquired sensorineural hearing
loss. Recent experiments using human umbilical cord blood treatment of a mouse and guinea pig
models have demonstrated hair cell re-growth following acquired sensorineural loss as well as
partial restoration of ABR. Autologous human umbilical cord blood therapy, which has been
used for over twenty years, has an excellent safety record. This study will determine if
autologous human umbilical cord blood infusion in children with hearing loss is safe and
feasible, improves inner ear function, audition, and language development. The patients
umbilical cord stem cells collected at birth and stored at Cord Blood Registry will be used
for infusion.

Inclusion Criteria:

1. Evidence of a sensorineural hearing loss

- Unilateral or bilateral in configuration

- Symmetrical or asymmetrical configuration

- Sudden or progressive in presentation

- Moderate to profound in degree (40-90 Decibels (dB) in at least one ear

2. Normally shaped cochlea, as determined by MRI

3. The loss must be considered:

- Acquired

- Unknown with a negative genetic test.

4. Fitted for hearing aids no later than six months post detection of loss.

5. Enrollment in a parent/child intervention program

6. Age 6 weeks - 6 years old at time of infusion with less than 18 months of hearing loss
at the time of cord blood infusion.

7. Ability of the child and caregiver to travel to Orlando, and stay for at least 4 days,
and to return for all follow-up visits.

Exclusion Criteria

1. Inability to obtain all pertinent medical records:

- (pertinent physician notes, speech language pathology notes, laboratory findings,
test results and imaging studies-must be sent to the research team at least prior
to the subject arriving at the study location for preliminary screening and
eligibility assessment, preferably14 days before the scheduled hUBC treatment.)

2. Known history of:

- Recently treated infection less than 2 weeks before infusion.

- Renal disease of altered renal function as defined by serum creatinine > 1.5
mg/dl at admission.

- Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and or T.
Bilirubin > 1.3 mg/dL

- Malignancy

- Immunosuppression as defined by WBC < 3,000 at admission

- Human Immunodeficiency Virus (HIV)

- Hepatitis B

- Hepatitis C

- Evidence of an extensive stroke (> 100ml lesion)

- Pneumonia, or chronic lung disease requiring oxygen

- Genetic syndromic sensorineural hearing loss

3. hUBC sample contamination

4. Banked cord cells totaling less than 6x106 mononuclear cells/kilogram body weight.

5. Evidence of the following maternal infections during the pregnancy (Hepatitis A,
Hepatitis B, Hepatitis C, HIV 1, HIV 2, Human T-lymphotropic Virus (HTLV) 1, HTLV 2
(CMV and Syphilis can be included in the study)

6. participation in a concurrent intervention study

7. Unwillingness or inability to stay for 4 days following hUBC infusion (should problems
arise following the infusion) and to return for the one month, six month and one year
follow-up visits.

8. Presence of a cochlear implantation device

9. Evidence of a genetic syndrome

10. Evidence of conductive hearing loss

11. Documented recurrent middle ear infections which are frequent (>5 per year)

12. Otitis media at the time of examination

13. Sensorineural loss is mild

14. Over 18 months from identification of hearing loss at time of infusion