Stem Cell News

Stem cells offer hope in CP battle

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Dr. Joanne Kurtzberg

Dr. Joanne Kurtzberg received a $10.2 million grant for her work.

Triangle Business Journal - by James Gallagher — Friday, June 25, 2010

Durham – A Duke University physician believes the key to curing, or at least lessening the severity of, cerebral palsy lies within cord blood stem cells, and she has begun a clinical trial to find out if that is true.

Dr. Joanne Kurtzberg, director of Duke’s Pediatric Bone Marrow and Transplant program and director of the Carolinas Cord Blood Bank, has begun a U.S. Food and Drug Administration-authorized random clinical trial to see if cord blood stem cells have the ability to cure or lessen spastic cerebral palsy in children aged 1 to 6. It is among a handful of FDA-authorized clinical trials regarding stem cells in the U.S.

The study, which is funded through a $10.2 million grant from the Robertson Foundation, has the potential to provide hope to people with cerebral palsy and their families as well as to open new doors or establish protocols for the use and gathering of stem cells.

Cerebral palsy, or CP, is a disorder that affects a person’s ability to move and maintain balance and posture. It is most often caused by a lack of oxygen to the brain, usually before or during birth, that results in damage to the portion of the brain that controls muscle tone.

The condition limits movement of arms and legs, can force people to use walkers or wheelchairs and can make it difficult for people to swallow or speak. Many patients need full-time care, and the federal Centers for Disease Control and Prevention reported in 2003 that the cost of treating CP totaled $921,000 over a person’s lifetime.

“We don’t have a cure right now, and if stem cell treatment could be a cure or improve symptoms early on and keep them from getting that bad would be huge,” says Dr. Anne Schmidt, medical director for United Cerebral Palsy of Greater Birmingham in Alabama.

The goal of the clinical trial, says Kurtzberg, is as much about finding a treatment for cerebral palsy as it is about finding out what cord blood can do. She is looking to see if the cells will reduce inflammation in the brain, produce new hormones to repair damaged brain cells or evolve into new brain cells to replace the damaged ones.

And, she says, the work could encourage more parents to bank their child’s cord blood in private cord blood banks or could lead to public cord blood banks changing their policies to assure a child’s blood is held long enough to treat that child, if needed, before the blood is donated for other uses.

“This is a very important question to answer,” she says of the trial. “If cord blood does help with cerebral palsy, more families may want to bank their cord blood.”

Cord blood comes from the umbilical cord when a child is born. In the past 10 years or so, there has been a concerted effort to collect that blood for its stem cells. Some of that blood is donated to public cord blood banks for use in other patients and research, while some of it is held in private cord blood banks for the use of that person later on. Storing cord blood in a private bank can cost hundreds to thousands of dollars.

The bulk of the banking so far has been done at public sites, says Kurtzberg.

Already, parents of children with CP have sought out these treatments, often traveling to countries such as China, Mexico and some countries in Europe in hopes that these treatments work, says Dr. James Carroll, chief of pediatric neurology at the Medical College of Georgia School of Medicine. Carroll, who has studied stem cell therapies in animals, also is conducing an FDA-authorized clinical trial using cord blood to treat cerebral palsy.

Even Kurtzberg has used the cord blood cell therapy to treat patients with CP and has found that they improved. However, she says, cerebral palsy patients often improve on their own, and this blind clinical trial will better determine whether the cord blood or something else caused that improvement.

For this trial, Kurtzberg is looking to study the effects on children with spastic cerebral palsy between the ages of 1 and 6 and who have their own cord blood.

The age limitations are set for two reasons. First, it is impossible to definitively diagnose cerebral palsy before the age of 1. And second, there are a limited number of cord blood cells that can be collected, and beyond the age of 6 a child grows too large for the number of cord blood cells available.