It has been proposed to initiate a novel clinical trial to assess the clinical benefits and safety of stem cell based therapy for treatment of traumatic brain injury in children at the University of Texas. The U.S. Food and Drug Administration (FDA) and the university's Committee for the Protection of Human Subjects (CPHS) have already approved the proposal.
The fact that stem cells derived from bone marrow can drift to an injured area in the brain followed by differentiation into neurons and induce repair in animal models has offered a new hope for the present study. The study would be based on the utilization of bone marrow derived stem cells derived from the patient, eliminating the ethical concerns regarding the use of embryonic stem cells.
"There is no reparative treatment for traumatic brain injury. All we can do now is try to prevent secondary damage by relieving pressure on the brain caused by the initial injury, " said principal investigator Charles Cox.
The bone, muscle and other organs have an inherent capacity to heal following an injury. The brain however has very poor regenerative capacity, accounting for the high mortality and functional disability following traumatic brain injury. More that 15 to 25% of children suffering severe traumatic brain injury die. Those who survive face a life long disability even in case of a moderate injury.
The I phase of the clinical trial would be directed towards establishing the safety of the treatment modality while the secondary objective would be observation of the potential therapeutic effects. Ten head injury patients with between 5 and 14 years of age would be recruited for the study based upon the inclusion and exclusion criteria. Followed by initial treatment and evaluation, the parents of the injured child would be approached regarding participation in the clinical trial.
After obtaining a written informed consent, progenitor stem cells from the bone marrow in the hip region would be extracted. This would be processed to obtain two different types of cells; mesenchymal stem cells capable of differentiating into bone, cartilage, fat cells, neurons and hematopoietic stem cells capable of differentiating into blood cells.
This would be followed by processing of the mesenchymal stem cells at Baylor College of Medicine and introduction into the injured child by an intra-venous injection. The entire process would be accomplished within 48 hours of the injury.
A critical monitoring of the injured children would be taken up. In addition, tests to assess the function of the brain would be carried out at end of 1 and 6 months following the procedure. The results obtained would be compared to the existing data on other similar children with brain injury.
Even marginal improvement could mean a great deal to someone who suffers a brain injury. "It could be the difference between being able to recognize your loved ones and not being able to, or between doing things for yourself or having to rely on others. That would be a huge impact on families and on society," Cox said.
Depending on the results obtained, further studies would be carried out on a larger scale. One of the main advantages of the present study is that the possibility of an immune rejection is unlikely, as the study participants would be receiving their own cells.