The clinical trial at The University of Texas Medical School at Houston and Memorial Hermann Children's Hospital begins late this month or early next month and will first test the safety of using stem cell therapy to treat brain injuries in children. Dr. Charles Cox is a professor of Pediatric Surgery at the medical school and the trial's principal investigator. He says the therapy has the potential to change the way severe brain injuries are looked at.
"What I mean by that is right now we focus on stopping bleeding and minimizing swelling in the brain and trying to minimize the secondary effects of the primary brain injury. There's no therapy to restore the neurons that make up the functional units of the brain."
The trial, which obtained FDA approval last month, will use bone marrow cells from the patient's hip, cells that doctors think will be able to find their way to the injury. Cox says the stem cells seem to respond to cues or signals emitted from the brain.
"The really fascinating component of this is that these cells have the ability to home to the site of injury without us having to directly place them at the site of injury. We can give them intravenously as opposed to directly placing them, through injections, into the site of the brain injury, but we can give them intravenously and then they home to that site based on the signals that the brain is releasing at the site of the injured tissue."
Referring to the first clinical trial as a "baby step", Cox says doctors will first ensure the safety of the therapy before they move on to the second phase of the trial to determine if the treatment benefit to patients.
"Our goal is to develop therapies that can substantially change the paradigm by which we approach these injuries. It's an exciting thing for us but it's one that we're very realistic and cautious about in terms of potential benefits."
The trial will initially use 10 brain injury patients between 5 and 14 years old from Memorial Hermann Children's Hospital. Cox says the most effective treatment will likely take place in the first 48 after the injury. The trial does not involve the use of embryonic stem cells, which come from human embryos.