June 25, 2007 (Chicago) -- Infusions of cord blood infusions seem to help kids with type 1 diabetes -- apparently by partially reversing the misguided immune system attack that is the keystone of the disease.
The experimental treatment is being tested by University of Florida researchers Desmond A. Schatz, MD, medical director of the University of Florida diabetes center, and Michael J. Haller, MD, assistant professor of pediatric endocrinology.
So far, they've treated 11 kids ranging in age from 2 1/2 to 10 years. They reported the six-month results from the first eight of these kids at the American Diabetes Association's 67th Annual Scientific Sessions, held June 22-26 in Chicago.
"This approach is outside the box," Schatz said at a news conference. "It is the first cellular therapy where we are using the patient's own cells -- cells that are naive to the environmental insult that may trigger the disease."
Schatz and Haller compared the results of cord-blood treatment to results for 18 kids who received current state-of-the-art treatment for their type 1 diabetes. Children who got cord blood showed signs of reduced immune damage.
"Kids who used cord blood had half of the insulin requirement of kids getting standard therapy," Haller said at the news conference. "This implies that kids who got cord blood made some insulin on their own."
Cord Blood Not a Type 1 Diabetes Cure
It's not a cure. Both Schatz and Haller expect that these children eventually will lose the ability to make insulin as their disease progresses. But they stress that the extra insulin-making time the kids gained will have lasting benefit.
Schatz and Haller favor the theory that the stem cells contain what researchers call regulatory T cells -- the quarterbacks of the immune system. The new cells, they suggest, try to halt out-of-control, anti-self immune responses.
Unlike cord-blood treatment for life-threatening autoimmune disease and cancer, where harsh and risky chemicals are used to kill off existing immune cells or cancer cells, the diabetes treatment is a simple infusion of cord blood.
That is likely to change as researchers become more confident in the technique -- and start looking for ways to get the immune system to become more tolerant of insulin-producing cells.
"We ultimately will have to add immune suppression," Schatz says. "The solution to reversing diabetes will require a cocktail approach. It will require a way of achieving tolerance, of restoring regenerative cells, and then giving enough cells back to reverse the process. Until we move to that era the progress will be slow."
Why not try that now? Schatz says that type 1 diabetes may eventually have devastating effects, but with insulin treatment, it is not an immediate threat to the life of a child.
"Diabetes is not cancer. It is not OK to have a mortality rate of 3%, which does occur with many of these transplants," he says. "We are very confident that some of these studies may come to fruition. But for now, from our perspective, the dictum of 'first, do no harm' has to be premium."
Parent Sparked Cord-Blood Treatment
Schatz says it wasn't his idea to start treating child diabetes with banked cord blood. The idea came from a parent.
"I received a call from a gentleman in Orlando," Schatz says. The man said he was the father of a child with diabetes, and began asking sophisticated questions about the use of experimental treatments. Thinking he was a fellow scientist, Schatz asked about the man's background.
"He said he was a financier, and offered to buy my time," Schatz recalls. "Of course I offered to meet him without charge. We spoke for two hours. And then he said that because he could afford it, he'd banked his child's blood. He said, 'I want you to use it to treat my 3-year-old.' That is how we got started."
So far, the treatment is available only to children whose parents could afford private cord blood banking. In the future, it may be possible to use cord blood from other children. But for now, Schatz and Haller consider this too risky. Even when tissue rejection isn't an issue, transplanted immune cells sometimes attack their new host -- a life-threatening situation called graft-versus-host disease.
"Even though we have treated 11 patients with more on the way, we have received 500 queries," Schatz says. "Parents say, 'I have a child with diabetes and I am pregnant -- can I use the cord blood to treat the older child?' But even with the closest matches you see some graft-versus-host disease."
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