Results from a clinical trial examining bone marrow stem cells as a medical intervention for pediatric traumatic brain injury were published in the March issue of the journal Neurosurgery. This is the first published clinical trial using mononuclear cells (MNCs) to treat traumatic brain injury (TBI) in humans. It demonstrates that infusion of autologous (one’s own) MNCs harvested from the bone marrow is safe for pediatric TBI.

A follow-on clinical trial evaluating cord blood stem cells, also in pediatric TBI patients, began in January 2011 at the University of Texas Health Science Center at Houston.

Although the bone marrow trial was designed to determine the safety of the procedure, the patients also showed preservation of brain tissue structures, as well as significant improvement in functional and neuropsychological outcomes like cognitive functioning, processing speed, working memory (listening skills), fine motor skills and declarative memory (verbal skills). There were no reports of infusion-related adverse events.

As researchers conclude this kind of treatment is safe, their results add to promising preclinical findings showing treatment benefits in animal models which will lead to phase 2 clinical trials to evaluate treatment efficacy.

Future Potential

No current therapies have the ability to repair, replace, or salvage cellular damage to the brain. (1,2) This investigational treatment suggests that stem cell research holds great promise for people in the U.S. who sustain a TBI.

Furthermore, the use of autologous cells in this investigational treatment avoids the potential for cell rejection, graft vs. host disease, and blood‐borne disease transfer further supporting the development of treatment strategies using autologous umbilical cord blood after a TBI to preserve potential cognitive and motor skills.

(1) Narayan RK, Michel ME, Ansell B, et al. Clinical trials in head injury. J Neurotrauma. 2002;19(5):503‐557.

(2) Cox CS Jr, Baumgartner JE, Harting MT, at al. Autologous Bone Marrow Mononuclear Cell Therapy for Severe Traumatic Brain Injury in Children. Neurosurgery. 2011; 68(3):588-600.