Grant to aid work of stem cell groundbreaker

Innovative ways of treating cerebral palsy are under study at Duke

Staff WriterMarch 19, 2010 

  • What is umbilical cord blood? It's the blood that remains in the umbilical cord and placenta after the baby is born. It's usually discarded.

    Why is it valuable? It's a rich source of stem cells, which are unspecialized cells that produce all blood cells and can be used to treat genetic diseases and certain cancers. To date, more than 70 disorders have been treated with cord blood stem cells

    How can cord blood be saved? Private cord-blood banks now market to parents in case their baby or a family member needs the cells in the future. Additionally, public cord blood banks, including the one at Duke, rely on donated umbilical cord tissue for transplant therapies.

    How are stem cells used to treat diseases? In most cases, patients undergo chemotherapy and/or radiation to destroy their own stem cells. Following that, they receive an intravenous infusion of stem cells. The transplanted cells migrate to the bone marrow, where they make new copies of themselves and produce blood cells that rebuild a healthy immune system.

    What are the advantages of using cord blood? Compared to bone marrow cells, cord blood stem cells are safer and easier to collect, there are more matches, and they result in fewer complications.

    Are there disadvantages? There are often not enough blood cells that can be harvested from the cords.

An internationally known pioneer in using umbilical cord stem cells will research novel cerebral palsy treatments thanks to a $10.2 million gift to Duke University.

The money from the Robertson Foundation will establish a Translational Cell Therapy Center at Duke for cell-based treatments, notably the work of Dr. Joanne Kurtzberg. It is the latest large donation from private sources to advance medical research at local universities.

Kurtzberg has used umbilical cord cells to treat cancer and genetic disorders in children. In many cases, infusions of cord blood have reversed and even cured otherwise fatal disorders. Kurtzberg has recently begun using the once-discarded material in hopes it can also mend brain damage in children diagnosed with cerebral palsy.

"I don't think we'd be able to do this research without this grant," Kurtzberg said. "When you are doing work at that cutting edge, you don't have enough preliminary data to get funding in traditional ways."

Dr. Victor Dzau, chancellor for Health Affairs at Duke, said the Robertson Foundation gift will speed new cell therapies to patients. In addition to funding Kurtzberg's cerebral palsy study, the money will establish a specialized laboratory where therapeutic cells can be made and stored, benefiting cell scientists throughout the medical center.

"It's a significant gift," Dzau said, "creating a place where we are doing state-of-the-art work."

The Robertson money comes on the heels of more than $150 million in scientific research grants to Triangle institutions from federal tax stimulus dollars. Last week, Duke announced a $12 million gift to its eye center, and UNC-Chapel Hill officials last year received $22 million from the Bill & Melinda Gates Foundation for health projects in Africa.

Julian Robertson Jr., a North Carolina native who established the foundation to advance work in medicine, education, environmental stewardship and religion, was out of the country and unavailable for comment. He is the founder of Tiger Management.

In a prepared statement, Robertson said Kurtzberg's work "has the potential to change the lives of thousands of children throughout the country and around the world."

Umbilical cord blood

Kurtzberg has long been at the forefront of regenerative medicine, and was among the first to prove that umbilical cord blood was a viable alternative to bone marrow for use in transplants.

The blood is rich in stem cells, which are prized because they haven't yet specialized and can therefore build healthy cells and tissue where damage or deficiencies have caused problems. They appear to work in three ways: repairing damage, particularly if they're infused soon after an injury has occurred; recruiting other healing cells to help with repairs; and replacing dead or damaged cells.

In 1988, Kurtzberg successfully used cord blood in a transplant to treat a child with Fanconi anemia, a rare genetic disease in which the bone marrow fails to produce adequate immune cells and blood clotting agents.

She has since blazed trails using umbilical cord blood to treat leukemia, metabolic disorders and sickle cell disease, and her clinic at Duke attracts parents from around the world seeking cord blood transplants for their children. Many have banked their children's umbilical cords, and others have used donated cells from a public bank Kurtzberg has helped build at Duke.

Now she's targeting cerebral palsy. The condition, marked by problems with muscle coordination, afflicts an estimated 2 in 1,000 children worldwide. Often caused by oxygen deprivation or trauma during birth, cerebral palsy has no cure. Current treatments include physical therapy and drugs that reduce seizures.

Proving the treatment

Marla Dunlap, a mother of three from Arlington, Texas, arrived at Duke this week seeking an infusion of cord blood for her 7-year-old son, Cayden. The youngster, who wears braces but can walk and run, has cerebral palsy after suffering a stroke before he was born.

A registered nurse, Dunlap said she and her husband decided to bank Cayden's cord blood when he was born, hoping stem cell science would one day provide a treatment to help their son.

Then one morning last summer, she saw a network television program featuring a family of a child with cerebral palsy who had been treated by Kurtzberg at Duke.

"For me, it was the theory that the cells will go to where the damaged parts are in the body, hoping to regenerate parts of the brain that were injured," Dunlap said.

She said Cayden qualified for a small trial Kurtzberg has been running to test whether cord blood infusions are safe for children with cerebral palsy. He underwent the 30-minute infusion Tuesday as an outpatient. Dunlap said she will monitor his progress at home in Texas and report to Kurtzberg periodically.

Kurtzberg said that trial, which is continuing, has had tantalizing results in the first 188 patients, showing little danger and possible improvements. But it's ultimately inconclusive.

"We couldn't say one way or another if it was beneficial," she said, "because children with cerebral palsy naturally will improve to some degree. What we have to show is that they improve more than they would have anyway."

And that's where the Robertson Foundation funding will help, Kurtzberg said. With some of the money, she will begin a new cerebral palsy study in which children who are infused with cord blood are compared to children who have received a placebo infusion. Only that kind of comparison can prove the intervention is a success.

Cord blood banks

Kurtzberg said if proof of the procedure's effectiveness is established, the study could change the way cord blood is managed.

Currently, many parents, such as the Dunlaps, pay to store their children's cord blood in private, for-profit banks. Kurtzberg has been a proponent of a national, public bank.

The one at Duke draws donations from eight hospitals in North Carolina, plus several others across the nation where the donated cells are publicly available for children and even adults who need transplants.

Kurtzberg would like cord blood to be automatically collected and stored at every hospital, as a matter of national policy.

"But that's something for another day," she said. or 919-829-4882

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