Autologous Stem Cells for Spinal Cord Injury (SCI) in Children

Of the estimated 11,000 cases of acute spinal cord injury (SCI) which occur each year in
North America, 5% involve children. The injury is divided into the primary mechanical event
which causes the injury, and the secondary events which follow. Outcome for SCI depends of
the severity of the primary injury (complete vs. incomplete) and the spinal cord level of
the injury. Current therapy is designed only to minimize the secondary events of SCI and
other trauma-associated injuries. Because the current therapy does nothing to reverse the
primary insult, significant advances in reducing the disability associated with SCI are
unlikely. Recent basic science and animal studies suggest that stem cell treatment can
foster functional improvement after SCI by helping repair the primary injury and reducing
the secondary injury.

Stem cells are "unspecialized" cells in the body that do not have a specific function yet
(for example, they have not become "heart cells" or "brain cells" yet.) Stem cells are able
to divide and develop into more mature, function-specific cells and take the place of those
cells that die, are injured or can no longer function the way they are supposed to. Stem
cells are being studied a lot because of this ability and there is the possibility that they
may be used to take the place of cells that are no longer working in different parts of the
body because of disease (as in cancer, diabetes, and heart disease). Stem cells can be found
throughout the body, but they are most common in the bone marrow, the thick, spongy material
inside the bones.

The primary objective of this study is to determine the safety of transplantation of the
patient's own (autologous) Bone Marrow Progenitor Cells (BMPC) in children with SCI. The
secondary objective is to determine if functional, physiological and anatomic outcome
measures are improved after BMPC autologous transplantation in children with SCI.

Inclusion Criteria:

1. Between 1 year and 15 years of age on the day of study BMPC infusion.

2. Survived at least six months with PSCI, but are less than 4 years post injury (± 30
days), and have fixed neurologic deficits related to their injury at the time of
enrollment.

3. Ability of child to understand and speak English.

4. Ability of child and caregiver to travel to Houston, Texas, and stay for at least 4
days, and to return for all Follow-up visits (patient is responsible for cost of
travel and lodging while in Houston).

5. Pediatric patients with any type of spinal cord injury as long as their spinal cords
are continuous on MRI evaluation. This includes paraplegic and quadriplegic patients
with complete or incomplete spinal cord injuries. This includes patients with ASIA
impairment scales from A to D. The clinical classification will be described by the
level below which motor/sensory function is impaired, and the degree of that
impairment. (i.e. C-5 if the deltoid muscle is intact but the biceps and other muscle
groups below the C-5 level are weak [incomplete motor injury] or paralyzed [complete
motor injury]. A similar evaluation of sensory function will be established
clinically.

Exclusion Criteria:

1. Lack of informed consent.

2. Uncorrected coagulopathy during the baseline period defined as: INR > 1.4; PTT > 35
sec; PLT < 100,000.

3. Pre-injury history of seizure disorder and/or neurological impairment where the
patient would not be able to participate in age appropriate pain rating scales.

4. A history of prior SCI or severe traumatic brain injury.

5. Known history of:

- Recently diagnosed infection (within past 2 weeks) requiring treatment and/or
medical intervention.

- Renal disease or altered renal function as defined by serum creatinine > 1.5
mg/dL.

- 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 (10x3) at screening and/or baseline
evaluation lab.

- HIV.

- Hepatitis B or C.

8. Unhealed fractures or wounds including osteomyelitis.

9. Pneumonia, or chronic lung disease requiring oxygen.

10. An anatomically discontinuous spinal cord diagnosed by CT or MRI imaging.

11. Positive urine pregnancy test (urine pregnancy test will be routinely performed on
females of childbearing potential, age 11 or older.

12. Participation in a concurrent intervention study.

13. Desire for organ-donation in the event of death.

14. Unwillingness or inability to stay for at least four days following BMPC infusion
(should any problems arise following the infusion) and to return for a 30 day, and 6
month follow-up visit, and be available for 1 year, and 2 year follow-up phone calls.