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ONLINE DOCUMENT: CORD BLOOD: MEDICAL MIRACLE OR MARKETING OPPORTUNITY?

When chemotherapy failed to cure Alex Thompson's acute leukemia, doctors in Alabama looked elsewhere to try to save the 5-year-old's life.

A bone marrow transplant was not an option. A national search for a donor turned up no marrow match.But an umbilical cord blood transplant, an experimental treatment being pioneered at Duke University, held out hope.

In February, Alex received a transfusion of a half cup of raspberry-colored fluid drained from an anonymous baby's umbilical vein moments after her birth.

So far, so good. Blood-making cells spawned by the cord blood are replacing his faulty bone marrow and making more normal blood. Complications are minimal. And Alex's blood type has changed.

"We were O negative and now are O positive," said Barbara Hyde, holding on tight to her shy grandson in an exam room at Duke Hospital. "That's from a negative to a positive in more way than one."

Such outcomes bolster the belief that umbilical cord blood, once considered medical waste, may be the next medical miracle. But its success is creating some sticky questions about whether newborns' blood should be bought or shared.

Some worry that biotechnology entrepreneurs are taking unfair advantage of the good news cord blood is making. Companies already sell expectant couples the chance to store a sample of their baby's umbilical blood. The founder of the largest company in the field, ViaCord in Boston, charges parents $1,500 and then an annual fee of $95 to preserve a baby's blood.

But some scientists support a Food and Drug Administration plan to step in and forbid anyone from making a profit banking the blood until its benefits are proven. The companies don't always make plain just how unlikely it is the blood will be needed, they say.

"It's not quite like insuring yourself against getting run over by a herd of elephants in the middle of Bethesda," said Dr. Paul McCurdy, director of the blood resources program for the National Institutes of Health in Bethedsda, Md. "But it's not quite unlike it."

Dr. Joanne Kurtzberg can juggle three conversations, grab a ringing phone and examine slides of blood cells all at once. The pediatric oncologist directs Duke's pediatric bone marrow transplant unit, which is buzzing these days.

Patients, mostly very sick children, come to Kurtzberg's basement clinic at Duke South Hospital from all across the country. So do doctors, who want to see for themselves whether she can help kids they don't have the means to save at home.

While research is still in its infancy, scientists say cord blood's unique qualities may help more patients, particularly children, beat a long list of devastating diseases, including leukemia, severe anemia and inherited immune deficiencies. Cord blood is easier to harvest than bone marrow and cheaper. Marrow must be extracted with needles from a donor's back, a costly and lengthy procedure that requires anesthesia. Like bone marrow, cord-blood transplants take best when the donor is a relative with compatible tissue type.

But cord blood appears to work better between unrelated people than bone marrow does. That's important because every year people who might be helped from a transplant die while waiting for a marrow match.

Scientists didn't know about the riches of cord blood until the late 1980s, when new technology enabled them to better compare blood-making cells in marrow and cord blood, Kurtzberg said. They were thrilled about what they found.

"It turns out that it contains the same cells bone marrow has to regrow blood," Kurtzberg said.

Fewer than 400 cord-blood transplants have been performed throughout the world since 1988. Kurtzberg has done the most. She performed the world's first transplant using blood from an unrelated donor in 1993 and has done that procedure 45 more times. Fifty-four percent of those patients are still alive.

While it is not clear whether small amounts of cord blood hold the same promise for adults as they do for children, Kurtzberg has had some success with adults.

But many questions about the experimental treatment remain. No large-scale studies have been performed to show which diseases respond best to the treatment. And there are unknowns about the blood itself. No mandatory standards have been established for its collection, storage or freezing. It's not clear how long it can safely be preserved. And some wonder whether blood from donors too young to have a medical history may carry undetected disease.

Still, the NIH clearly sees potential in this new therapy. It will invest more than $24 million during the next five years to create at least two public cord-blood banks and eight transplant centers. Kurtzberg's clinic is among the finalists for the funding.

At the same time, the FDA has proposed designating cord blood an investigational new drug. That means companies would have to be licensed to preserve the material but could not make a profit until it was approved for the marketplace.

"We've received a lot of serious complaints about commercial companies from consumers, scientists and physicians," said Leana Harvis, chief of cellular hematology for the FDA. "This is being offered as a type of life insurance before there is sufficient data to support such a claim."

Kurtzberg thinks that public banks eventually will meet the needs of all people looking for cord blood. But she doesn't fault parents for paying to have their children's blood frozen and stored, as long as they know how long the odds are that they'll ever need it. Its full potential, she said, hasn't been tapped.

"It's reasonable if they have the resources and won't have to choose between a college fund or preserving cord blood," she said.

But Dr. Jeremy Sugarman, a medical ethicist at Duke who is trying to generate a national discussion about the ethics of using cord blood, says making a profit off a body part contradicts an important tradition in modern medicine.

"All other body parts, including blood and bone marrow, are not viewed as a commodity but as something for the public good," Sugarman said. "Something not to be bartered and sold, but something to be shared."

Most hospitals still throw cord blood away. At Duke, cords are saved when mothers give their permission.

Cynthia Fisher, ViaCord president, won't disclose how many customers have paid her company to preserve the blood. Most, she said, have a sick child who could use a transplant from a relative or have an inherited disease in their family.

Fisher welcomes regulations and wants more public cord-blood banks. So far there is only one in New York.

But she doesn't think companies should be kept out of the cord-blood business, even if the blood becomes available through public banks.

Two University of North Carolina physicians, obstetrician Jeffrey Kuller and bone marrow transplant specialist Joseph Wiley, have written a paper to prepare doctors for questions from parents-to-be about whether they should preserve the cord blood.

The chances a single child will need a bone marrow transplant, or one day a cord-blood transplant, are very low, they note.

Only one in 8,000 kids will need a transplant to fight acute leukemia, they estimate. One in 24,000 would need it to fight lymphoma and one in 16,000 would need it for marrow failure.

Also, some children with blood-borne diseases, such as leukemia, couldn't use their own blood for a transplant because it might reintroduce the illnesses.

The UNC doctors wrote that parents likely will use emotion more than wisdom in trying to decide whether to preserve the blood.

(Distributed by Scripps Howard News Service.)