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Young lives get a fighting chance
By Rita Rubin, USA TODAY
Cord blood costs $1,500 to $2,000 to collect, process and store a single unit, according to Pablo Rubinstein of the New York Blood Center.
7/13/2005 - As her newborn sister was having groundbreaking treatment for a rare genetic disorder that also had ravaged their brother's brain, 5-year-old Katie Kristoff had one question for her parents:
"Will this baby smile?"
Pamela and Bill Kristoff had struggled over whether to conceive a third child. Although Katie was healthy, younger brother Ryan was diagnosed with Krabbe's disease when he was about 8 months old. Doctors said he wouldn't live to see age 2.
Ryan's devastated parents, who live in Colchester, Conn., scoured the Internet for information about Krabbe's. They discovered the Hunter's Hope Foundation, established in 1997 by NFL Hall of Famer Jim Kelly and his wife, Jill, after their infant son, Hunter, was diagnosed with Krabbe's.
Through Hunter's Hope, the Kristoffs learned that Duke University doctors had halted the progression of Krabbe's with umbilical cord blood transplants. The catch: The earlier babies received a transplant, the better their chances for a normal life.
Cord blood refers to the blood in the placenta and the part of the umbilical cord still attached to it after delivery. Hospitals usually discard this tissue. But it's a rich source of blood-forming stem cells similar to those found in bone marrow. In Krabbe's, those stem cells supply a critical missing enzyme, without which communication between the brain and the rest of the nervous system breaks down.
Worldwide, 5,000 to 6,000 cord blood transplants have been performed since 1988, says Pablo Rubinstein, director of the New York Blood Center's National Cord Blood Program, the world's oldest and largest cord blood bank.
Such transplants have been used to treat more than 70 diseases in adults and children, including those of the immune system and the blood as well as inherited metabolic disorders such as Krabbe's, according to Rubinstein's program. The vast majority of recipients have had leukemia.
Though Krabbe's affects only about 1 in 100,000 live births, about 1 in 5,000 babies born in the USA have some type of metabolic disorder that could be treated with a cord blood transplant, says Joanne Kurtzberg, head of the Duke Pediatric Blood and Marrow Transplant Program. Others include Tay-Sachs and adrenoleukodystrophy, the condition made famous by the 1992 film Lorenzo's Oil.
Cord blood is easier to collect and store than bone marrow, and in the case of a tissue mismatch, it is more forgiving. Fewer than a third of candidates for bone marrow transplants have a sibling who is an identical match. So thousands of Americans die every year waiting for bone marrow from an unrelated donor, according to a report in April from the Institute of Medicine (IOM). Congress had asked the institute to study options for a national cord blood system.
In the past year, nearly 2,000 bone marrow transplants and about 500 cord blood transplants were done in the USA, Rubinstein says. In May, the House overwhelmingly passed a bill that would provide $79 million to boost the U.S. cord blood inventory to 150,000 units, thought to be enough for 90% of Americans needing transplants. In late June, a Senate panel approved a similar bill.
Cord blood banking is expensive. It costs $1,500 to $2,000 to collect, process and store a single unit, Rubinstein says. The blood must be tested to make sure it's not from a child with a virus or genetic disease. It must be frozen properly so the stem cells aren't destroyed.
That's why public banks collect cord blood at only a limited number of hospitals. For example, Rubinstein's bank, which has collected about 29,000 units since its inception in 1992, is adding its sixth hospital this month.
Though seemingly benign, cord blood donation raises a number of ethical and legal questions, the IOM report says: "It is crucial to disclose several kinds of information to the potential donor, including who has access to the cord blood once it is donated, where it is stored, how it is stored, and how the donor's privacy is protected."
Ready to fight
Ryan Kristoff seemed perfectly healthy after his birth in March 2001. At 4 months, though, he was crying inconsolably round the clock. He couldn't hold his head up.
A few months later, tests revealed he had Krabbe's, passed on by both of his parents. The Kristoffs had had no reason to suspect they were among the estimated 2 million Americans who carry the Krabbe's-causing genetic mutation.
Children with Krabbe's lack an enzyme involved in making myelin, the protective covering around nerves. Without an immediate transplant of stem cells that carry the gene for the missing enzyme, babies with Krabbe's rapidly lose mental and motor abilities. Ryan's neurologist told his parents to take him home and make him comfortable for his remaining few months.
Pamela Kristoff, 33, a former schoolteacher, chokes up at the memory. "I thought maybe he was a little slow," she recalls, "but I didn't think he was dying."
The Kristoffs decided they wouldn't give in without a fight. Ryan's mother called Duke's transplant program. "You needed to be here yesterday," she was told. "How soon can you get here?"
Tough road ahead
When it comes to transplanting cord blood into children with Krabbe's disease, "even a few weeks can make a difference," Kurtzberg says.
In May, she co-wrote a report in The New England Journal of Medicine on cord blood transplants in 25 babies with Krabbe's.
The study included 11 newborns who, because they had older siblings with the disease, were diagnosed before symptoms appeared. They were compared with 14 babies — in some cases, their own brother or sister — who received transplants after Krabbe's had begun its assault.
First, the babies had nine days of high-dose chemotherapy to wipe out their bone marrow and prevent their own immune systems from attacking the donor stem cells. On the 10th day, they received the cells through an intravenous line.
"It's kind of like planting seeds in the garden," Kurtzberg says.
The "seeds" sprouted in a month or so. Blood counts began rising, as did levels of the missing enzyme. But while the transplants provided "indisputable benefits" in the newborns, the older babies remained severely impaired, the researchers found. Only time will tell whether transplantation in newborns actually prevents mental decline or simply delays it, they write.
At Duke, tests showed that Ryan "was right on the borderline, and it was up to us" to decide whether to proceed with a transplant, Kristoff says. Ryan had severe brain damage, but, his mother says, Kurtz-berg thought a transplant might improve his quality of life. The Kristoffs felt they had to try.
They ended up spending 10 months at Duke. "Because Ryan's brain was compromised, he had a tough road," Kristoff says. He lost his vision and developed water on the brain and pneumonia. "We got very close to having to let him go."
Every day, Ryan's parents wondered whether they had done the right thing. For the first few years after the transplant, Kristoff says, Ryan appeared to be no better off. Today, at 4, he still needs a feeding tube and can't move, see or speak.
"He's severely handicapped," Kristoff says. "He requires lots of care." But, she says of the transplant, "I think it did make him more comfortable. He loves to be moved around. Before, we couldn't even hold him." Best of all, she says, "six months ago, he started smiling again."
Despite Ryan's battle, the Kristoffs couldn't abandon their pre-Krabbe's dream of a houseful of kids. Kurtzberg reminded them they had a 75% chance of conceiving a healthy baby. And now that they knew to look out for Krabbe's, they could test their third child before birth and if necessary, arrange for a cord blood transplant immediately.
"I promise you your child will smile through it," Kristoff says Kurtzberg told her. "The road won't be as hard as it was with Ryan."
The Kristoffs got the call in April 2004. Against the odds, amniocentesis revealed that their third child, a girl, had Krabbe's. "I felt like I got hit in the stomach," Kristoff says.
The family arrived at Duke on July 5, and Alyssa Ryan Kristoff was born two weeks later. If not for Ryan, Kristoff says, Alyssa, who got a transplant at 3½ weeks old, wouldn't have had a chance.
Although she's extremely small for her age and her motor skills are delayed, Alyssa has far surpassed Ryan's accomplishments.
She still needs a feeding tube, and she may never walk. But, her mom says, she can hold her head up, grab toys and smile, smile, smile.
"The first time we heard one of her toys crinkle, we were crying," Kristoff says. "She's reaching milestones he never met."
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Doubt cast on private 'banks'
At least two dozen companies are trying to sell expectant parents on the idea of banking their newborn's umbilical cord blood in case the baby or a sibling might need it in the future.
Private banking isn't cheap. Cord Blood Family Trust charges $1,745 ·$100 less if you sign up at least six weeks before the baby's due date ·plus a $95 annual storage fee, says chief operating officer David Dickinson.
Some of the top doctors involved in cord blood transplantation say that's probably not money well spent. "Much of the hype (about private banking) is based on research that may or may not pan out," says John Wagner of the University of Minnesota.
On its Web site and in magazine ads, Cryo-Cell International suggests that parents can protect children from heart attacks, strokes, diabetes and Alzheimer's by banking their cord blood. The site links to press releases and medical journal abstracts about roden — not human — studies using stem cells from cord blood.
"We're not representing that banking your baby's cord blood is the cure for a heart attack," says Gerald Maass, Cryo-Cell executive vice president. "The message that we're trying to convey is that research is proving to be very positive in animal models. Many of the cures that we look at today started off that way."
But today, even when a transplant is called for, doctors prefer to treat inherited conditions or leukemias with cord blood from healthy donors. Privately banked cord blood has been used, but in nearly all cases it went to an older sibling known to need a transplant before the baby was born.
Wagner says he actually has twice used children's own cord blood to treat aplastic anemia, "a very rare circumstance." In a third case, though, he says, the cord blood "was so badly stored and tested, I couldn't use it."
Lawrence Petz, chief medical officer of StemCyte, which runs the Cord Blood Family Trust as well as U.S. and Taiwanese donor banks, says private banks' standards for collecting and storing have improved over time.
Petz acknowledges "it would be quite unusual" for children who are healthy at birth to eventually need a transplant of their own cord blood. "The odds are that the unit would not be used," he says. "But there is a finite possibility of that happening."
By Rita Rubin
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