Dr. Charles Cox, director of the Pediatric Surgical Translational Laboratories and Pediatric Program in Regenerative Medicine at the University of Texas Medical School at Houston, is an expert on cellular therapies for traumatic brain injury. He has written over 120 scientific publications about the nature of his work, in addition to serving on study sections and research teams, such as the NIH, American Heart Association, Veterans Affairs MERIT Awards, Department of Defense, and Congressionally Directed Medical Research Programs. Last week, he sat down with the Huffington Post to discuss his clinical trial that assesses potential, adult stem cells that may be able to treat children who have suffered traumatic brain injuries. Below is a transcription of the interview:
Traditionally, what course of action is taken for children with severe head injuries?
“In broad terms, the standard of care in the treatment of severe traumatic brain injury hasn’t fundamentally changed over the past 30-40 years. For injuries of this type, most neurosurgeons focus on blood clots and trying to control the inflammation that causes swelling. Ostensibly, they’re trying to mitigate against “secondary brain injuries,” the cascade of events that occurs after the primary injury. This is critical to control as the brain is encased in a rigid, bony compartment and thus blood flow to the brain can become compromised causing irreversible brain damage beyond the initial event.”
What course do you personally take when confronted by this issue?
“We have an ongoing Phase 2b clinical trial sponsored by the National Institutes of Health (the National Institute of Neurological Diseases and Stroke) that uses the child’s own bone marrow derived mononuclear cells, which are infused intravenously within 48 hours after a severe traumatic brain injury. Normally, after a severe TBI, a child will lose about 10 percent of the white matter in their brain. So, utilizing specialized MRI scans, we’re measuring the preservation of specific structures in the brain, early after the injury and again at six months. This is a double blinded, randomized study that will ultimately include 50 patients, some of who will get a placebo instead of their own cells. After 20 patients have been enrolled, the DSMB will evaluate the progress and safety of the study — we are on patient number 13.”
What mechanism is at work during your treatment and what is the ultimate goal for your experimentations?
“Many people assume we’re drilling a hole in the brain and administering the cells directly. But the cells are actually given intravenously to the children. In pre-clinical and animal studies, we showed that the cells could modify the body’s immune response to the injury itself and stimulate an anti-inflammatory reaction, even in the brain. It’s truly a fascinating treatment and one that reflects how connected our brains are to the rest of the human body — how we’re a complete, integrated system. Once these cells are taken from the child’s body and administered to them, they sequester in the spleen, where they produce a host of molecules/cells/growth factors in distant organs, which then go on to influence the injured brain, especially in regard to inflammation. Overall, the decreased inflammation translates to preservation of brain tissue — and that’s what we are measuring with MRI in the trials.
We hope to see similar results in the Phase 2 study to those in Phase 1, wherein a safety trial indicated structural brain preservation. In that study (published in the journal, Neurosurgery in 2011), we noted that there was no decrease in the brain volumes of children with severe brain injuries, treated with their own bone marrow derived cells. Normally, there is a loss of about 10 percent of this tissue over 6-12 months post injury. That stabilization post treatment led us to design the second trial around the idea of preserving brain tissue rather than a global functional outcome.”
Source: Huffington Post
