Safety of Autologous Stem Cell Treatment for Spinal Cord Injury 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.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
American Spinal Injury Association (ASIA) - Standard Neurological Classification of Spinal Cord Injury.
ASIA classification assessment will take place pre-procedure, 1 day, 30 days, and 180 days post-procedure to define changes in functional outcome.
James E. Baumgartner, MD
Memorial Hemann Health System
United States: Food and Drug Administration
|Children's Memorial Hemann Hospital; University of Texas Health Science Center - Houston||Houston, Texas 77030|