Bone marrow stem cell transplantation feasible, safe treatment for children with severe traumatic brain injury

For children with severe traumatic brain injury (TBI), transplantation of stem cells derived from the patients' own bone marrow is a "logistically feasible and safe" treatment procedure, reports the March issue of Neurosurgery, official journal of the Congress of Neurological Surgeons. The journal is published by Lippincott Williams & Wilkins, a part of Wolters Kluwer Health, a leading provider of information and business intelligence for students, professionals, and institutions in medicine, nursing, allied health, and pharmacy.

The study paves the way for further research to evaluate whether treatment with autologous bone marrow-derived mononuclear cells (BMMNCs) can enhance brain recovery after TBI in children. The lead author was Dr. Charles S. Cox, Jr., professor of pediatric neurosurgery at The University of Texas Health Science Center at Houston (UT Health) Medical School.

Study Shows No Complications—and Preliminary Evidence of Effectiveness
The study included ten children, aged 5 to 14 years, with severe TBI. (The study excluded patients with very severe brain trauma associated with a high risk of death.) Within 48 hours after the injury, all children were treated with stem cells obtained from their own (autologous) bone marrow, called bone marrow-derived mononuclear cells (BMMNCs).

The BMMNCs were developed from bone marrow collected from the hip bone by a simple surgical procedure, then infused back into the patient intravenously.

All of the children, who were treated at Children's Memorial Hermann Hospital, survived. The bone marrow collection procedure was safe, with no harmful effects on blood circulation overall or in the brain specifically. There were also no apparent harmful effects of stem cell infusion, based on several assessments of organ function.

As a "phase I" trial, the study was designed solely to assess the feasibility and safety of the stem cell harvest and transplantation procedures. However, after six months of follow-up, all of the children had significant improvement. Final outcomes were rated good—with no or only mild disability—in seven of the ten children.

Children who survive severe TBI are often left with serious complications and disability. Currently, there are no effective treatments to protect or promote repair of the brain in these brain-injured children.

Previous studies have suggested that various types of stem cells, including BMMNCs, have the potential to promote or improve recovery of the brain after trauma. As a source of stem cells, BMMNCs have several important advantages, including relatively easy access to large numbers of the patient's own stem cells.

"Our data demonstrate that the acute harvest of bone marrow and infusion of bone marrow mononuclear cells to acutely treat severe TBI in children is safe," Dr. Cox and coauthors conclude. The study permits no conclusions as to whether the stem cell procedure improved the children's final outcomes. However, the results are promising so far and significant benefits may become apparent with longer follow-up

Many basic questions remain to be answered by future research. If BMMNCs (or any type of stem cell) do improve recovery after TBI, they may do so either by helping to develop new brain cells or by modifying the body's response to the injury. In addition to these scientific and clinical issues, many "logistical and practical requirements" will need to be addressed before stem cell treatment for brain injury can provide a treatment option for children with brain injuries.