Stem cells aid treatment of some patients

Success stories lead doctors to study cord blood

Chloe Levine's mother says the child has made a remarkable recovery since an infusion of her own cord blood in a clinical trial at Duke University Medical Center. It is these types of stories that have fueled what is probably the first controlled clinical trial of cord blood to treat cerebral palsy at Medical College of Georgia.


But a prominent researcher at Duke, who conducted the trial in which Chloe participated, warned against commercial cord blood banks using such stories as a marketing tool, particularly because neither the results of the trial nor any other controlled trial in humans with cerebral palsy has been published.

Dr. James Carroll, the chief of child neurology at MCG, has been working for the past five or six years with adult stem cells in animal models of brain injuries such as those that can lead to cerebral palsy. In animals with stem cells infused after the injury, "they do have significant improvement in their motor abilities," he said.

Why is still unclear. A very small amount turn into neurons, but the bigger impact appears to be from a neuroprotective effect from the cells and perhaps factors they trigger or release, Carroll said.

Dr. Joanne Kurtzberg, the chief of pediatric blood and marrow transplantation and director of the Carolinas Cord Blood Bank at Duke, has been working with cord blood for various diseases for the past two decades. In cerebral palsy, her theory is that the mechanism could depend on when and how the cells are administered, the dosage and the extent of the injury. The effect within hours to weeks might be because of inflammation, whereas years later that might not be as much of a factor, Kurtzberg said.

Carroll said he agrees with the inflammation theory in acute injuries and that it might be different for the kids in his clinical trial.

"That's a concern as to how much difference that will make in any favorable outcome," he said. But there was enough evidence for the Food and Drug Administration to approve the MCG trial in children ages 2 to 13, which is sponsored by Cord Blood Registry, a commercial cord blood bank. And there are also the stories such as Chloe's.

"We felt there was enough scientific reason to pursue it in conjunction with these individual reports of success," Carroll said. Giving the blood, collected from the child's umbilical cord at birth, is relatively safe, he said.

"I thought it was an opportunity to do a safe study and to find out if it works, to try to either make this a widely used treatment or to say, 'Well, folks it really doesn't work,' " Carroll said.

"It's very exciting for us that Dr. Carroll is really going to begin to get us to a place where we're going to have much better answers about what role these cells may play in helping these children," said Dave Zitlow, the senior vice president for public affairs for Cord Blood Registry.

That hasn't stopped the company from featuring patient stories such as Chloe's on its Web site. The company also reimburses patient families for travel to do interviews and gives them "a very cursory amount of token compensation for their time away from work," Zitlow said.

Kurtzberg, who conducted the trial Chloe took part in, said she is wary of having the commercial companies fund her research because it could lead to inappropriate promotion.

"They are using this in a way as a marketing tool," she said. And those companies are "very comfortable with the idea of premature publication and lack of scientific proof because it brings more business to them," Kurtzberg said.

Carroll said he was confident that would not happen and that the study has already met rigorous standards to get FDA and MCG approval.

In fact, if Chloe's family hadn't seen another child's story on the news, they would not have considered the treatment, Zitlow said.

The Levines, who live near Denver, were concerned when Chloe wasn't meeting milestones at nine months, such as holding her own bottle, said her mother, Jenny Levine. Her right hand was often clenched, and she seemed unable to use much of her right side.

A CT revealed damage from a stroke Chloe suffered in the womb, leaving her with cerebral palsy.

"Basically what we were told was that she would never be an Olympian," Levine said. "That you would face the next 17-18 years of therapy, all kinds of therapy. Their main words of comfort to us was that 'we don't know; we don't know what she is going to be able to do.' "

After seeing the story of a child who had a remarkable recovery after the treatment, however, the family pushed to have her treated in the Duke study. The results of that study, which was a phase I study on the safety of the treatment, will likely be published later this year, Kurtzberg said. There are no other published controlled trials in humans showing a benefit as far as Carroll knows. But the Levines say Chloe was showing an impact within days.

"We were on her way back from Duke, and the first thing she said was her nickname, which is CoCo," Levine said. "And that's something that we had worked with therapists for months to try and get her to say."

Now, a little more than a year and a half later, the right-side paralysis is gone, though she still needs some therapy for fine motor skills in the right hand.

"She's great. She's completely normal," Levine said.

She said she is glad to hear about the MCG study.

"(That) is absolutely what needs to be done to get the scientific evidence out there," Levine said. "I think it's wonderful."

Cord blood study

Cord blood is taken from the umbilical cord, which some parents store with public banks or pay companies such as Cord Blood Registry to store in case the child needs it. It has been studied as a replacement for a bone marrow transplant.

Medical College of Georgia is beginning what might be the first controlled clinical trials in cord-blood treatment for children with cerebral palsy. It is open to children ages 2 to 12 who have cord blood banked with Cord Blood Registry, which is sponsoring the trial at MCG.

The children will be examined by doctors. Half will be given their cord blood and the other half a sham infusion, with those conducting the trial blinded as to what the children are getting. The children will be examined again at three months for differences, and those who have not yet received their cord blood will get it then, with the others getting the fake infusion. The children will be examined again at six months and 12 months. Children must meet certain other medical criteria. For more information on the study, call (706) 721-3371.