Safety Study of Umbilical Cord Blood To Treat Pediatric Traumatic Brain Injury

Traumatic brain injury is the primary cause of pediatric trauma related morbidity and
mortality. Currently there is no reparative therapeutic option available, and all
interventions are designed to prevent injury progression or secondary brain injury.
Pre-clinical data suggest that progenitor cellular infusions may reduce the severity of
injury by a number of proposed mechanisms. The current study proposes a Phase 1 Safety Trial
using stored autologous UCB to treat patients that sustain a severe or moderate TBI, and
have not fully recovered as measured by the Glasgow Outcome Score-Expanded/Child at 6 to 18
months post-injury. We have chosen to use one bank that uses standardized processing and
storage protocol to reduce cell product variability.

Families who have banked hUCB at Cord Blood Registry, Inc. (CBR), will be prospectively
notified of the possibility of using their child's stored UCB if they sustain a moderate or
severe TBI and have a persistent deficit at 6-18 months. Prior to enrolling in the study,
patients will have their medical records, imaging studies reviewed, and a telephone
interview will determine potential eligibility and exclusion criteria. If eligible, the
patients will travel to Houston to undergo a medical history and physical exam,
neuropsychiatric evaluation, DT-MRI imaging of the brain, and baseline laboratory
evaluation. The UCB will be shipped to the Center for Cell and Gene Therapy for reanimation
and characterization/determination of release criteria of the cell product
(contamination-free). The UCB will be infused intravenously and the patient will be
monitored as an in-patient in the Pediatric Intensive Care Unit (PICU) located within
Children's Memorial Hermann Hospital for 24 hours, after which the patient will be
discharged but will return the next day for a final examination. Follow-up visits will occur
back at UT-Houston at 180 days, 1 year and 2 years post-infusion - these visits will include
medical history and physical exam, neurological and neuropsych evaluations, and DT-MRI
imaging of the brain.

Inclusion Criteria:

- Hospital admission Glasgow Coma Score between 3 and 12 at the time of injury

- Injury occurring 6 to 18 months prior to study cord blood infusion (+/- 30 days)

- Ability of child and caregiver to travel to Houston, and stay for at least 4 days,
and to return for all Follow-up visits

- Ability of child to understand (and speak) English

- Child's own cord blood banked at Cord Blood Registry

Exclusion Criteria:

- Inability to obtain all pertinent medical records, including pertinent physician
notes, laboratory findings, and radiographic images, related to the original injury,
hospitalization and rehabilitation

- Recent radiographic evidence of extensive stroke as evidenced by >100ml lesion

- Pre-injury history of seizure disorder and/or neurological impairment

- Obliteration of perimesencephalic cistern on initial head CT/MRI

- Initial hospital Intracranial Pressure (ICP) > 40

- Unhealed fractures or wounds including osteomyelitis

- Pneumonia, or chronic lung disease requiring oxygen

- Spinal cord injury as diagnosed by CT or MR imaging or by clinical findings

- Cord blood sample contamination

- Participation in a concurrent intervention study