Umbilical Cord Blood Banking: Insurance Against Future Diseases?

USA Today (Magazine)
March 01, 2000

CINDY MONROE was pregnant with her first child when doctors found a malignant tumor on the tissue between her lungs. She had non-Hodgkin's lymphoma. What followed was an end to the pregnancy, six months of chemotherapy, and nearly a dozen radiation treatments.

She hit the five-year remission mark in May, 1999. Now 34, she and her husband, Chuck, have a four-year-old daughter and a two-year-old son. The birth of their son, Brent, provided the Monroes with a promise of "biological insurance" in case Cindy's cancer comes back or another family member develops one of myriad problems.

This biological insurance is a blood sample from Brent's umbilical cord which has been cryogenically stored at -385 deg F in liquid nitrogen at a University of Arizona blood bank. It will remain there until it is needed. Cindy Monroe didn't think twice about saving her son's blood after she learned about the Cord Blood Registry (CBR)-a private cord blood bank based in San Bruno, Calif. -via a "Dear Abby" letter.

In most cases, placentas and umbilical cords were routinely tossed in the trash after a baby was born. Now, more than 10 years and over 1-500 transplants later, stem cells from umbilical cord blood seem like the wonder drug for treating many diseases. Expectant parents are increasingly faced with saving, rather than throwing away, this valuable resource.

Studies have shown the success of using umbilical cord stem cells to treat diseases such as leukemia, lymphoma, various anemias, and genetic disorders. Umbilical cord blood is a hematopoietic tissue, meaning it contains a mother lode of cells which give rise to the blood cells that carry oxygen, fight infections, and form clots in injured sites. Umbilical cord blood has even been billed as "an effective alternative to bone marrow transplant therapy" by the privately run New England Cord Blood Bank in Boston.

It almost seems too simple: Save a byproduct of the birth process and potentially save a life. Theoretically, it is simple. In practice, however, umbilical cord use, collection, and storage are still experimental and raise many controversial issues and questions. Should the blood be stored only for potential use by a family member or donated to a public bank? What is the likelihood of a family ever using its own sample?

What about the costs that are involved?

Scientists in the early 1980s began studies that used umbilical cord blood stem cells in tab animal transplants. In 1988, a six-year-old boy from North Carolina with a rare, inherited blood disorder called Fanconi's anemia was the first person in the world to receive an umbilical cord stem cell transplant. His parents had conceived another child in hopes of producing a positive bone marrow match, but researchers gave the parents another altemative-use umbilical cord stem cells from his healthy sister. The transplant was successful.

A study on using umbilical cord blood for transplants appeared in the fall of 1998 in the New England Journal of Medicine. Scientists from the New York Blood Center and the Medical Centers at Duke University, New York's Mount Sinai Hospital, and Brooklyn (N.Y.) Hospital found that placental blood is a useful source of allogeneic hematopoietic stem cells for bone marrow reconstitution. This means that the recipient and the donor do not necessarily need to be related to each other to ensure a successful transplant.

Each year, 30,000 patients are diagnosed with a disease that could be treated with a bone marrow transplant, according to the National Marrow Donor Program in Minneapolis. The chances of finding a viable donor match vary because individual tissue types differ. Unlike bone marrow transplants, studies suggest that the stem cells from a cord blood donor sample need not perfectly match the recipient.

With umbilical cord blood banking, families can store the blood - for a price - for their own use, donate it, or choose neither. Family banking fees average $1-500, plus an annual storage fee of about $100, according to the American College of Obstetricians and Gynecologists. That seems a small price to pay for the potential benefits, private cord blood banks argue, but what are the chances a child or family member will ever use a banked sample? They are slim, says the CBR, but it is a kind of biological reassurance that the companies sell and the parents seek.

"It's a peace-of-mind issue," indicates Scotty Kaesshaefer, communications manager for the CBR. "Ninety-seven percent of the families who bank with us do it just in case something does happen in the future."

For a young family to shell out $1,500 on top of preparing for a baby can be a burden, however. The costs of testing alone can run from $700 to $ 1,000, notes Kevin Long, manager of the American Red Cross' Ross Cord Blood Bank in Columbus, Ohio. Some tests are quite sophisticated, such as the DNA based one that determines the type of six different proteins-called human leukocyte antigens (HLAs) on the cell. A person's HLAs determine his or her tissue type and, therefore, compatibility with another person's tissues.

The idea behind donating to a public bank is that it increases the pool of tissue types. Donation, while free, does not guarantee the sample will be available if the donating family should need it, though. That creates a debate of ownership.

When a person donates a pint of blood to the American Red Cross, that blood goes into a public bank. With cord blood, parents have a choice whether to store their infant's sample or publicly donate it. Throughout the cord blood literature, researchers continuously ask if the blood belongs specifically to the infant or the family, or should be public property.

Finding a bone marrow match can be difficult, especially for minorities. African-Americans, who generally have less-common tissue types, have a 59% chance of coming up with one match in the national bone marrow bank of 3,000,000 donors. Caucasians with common tissue typing have an 85% chance of finding a compatible bone marrow donor.

Public cord blood banks generally don't store a sample for a specific family. The idea here is to generate a variety of samples. "With our program, the stem cell unit likely won't help the family, but it could help somebody else," Long points out. "One reason cord bloods are so useful is because minorities are underrepresented on the National Bone Marrow Registry."

Although the number of transplants using umbilical cord stem cells has risen in the last 10 years, it's still an "experimental procedure," says Larry Lasky, associate professor of pathology and internal medicine at Ohio State University and director of the Cord Blood Program at the Ross Cord Blood Bank. "The need for cord blood cells is not like the considerable need for red blood cells. Using cord blood stem cells is just one of the many treatments for cancer. It doesn't get at the cause of cancer; it just fights the disease down."

Still, collecting a cord blood sample is a lot less painful than donating bone marrow and significantly less time-consuming than searching for a bone marrow donor match. For certain diseases, cord blood stem cells may be the therapeutic answer, It is a small price for this kind of insurance, the CBR maintains.

Because Cindy Monroe is a cancer survivor, she qualified for CBR's Designated Transplant Program. The bank collected and processed her son's blood for free and will store it for four years. After that, the Monroes will pay $95 a year for storage. If Cindy or one of her immediate family members needs the sample, it is readily available. "We would be glad to pay for it forever to keep it stored," she insists.

Some cord blood banks, such as the CBR, rely on the attending physician to collect the blood via a syringe while the placenta is still in utero and after the umbilical cord is clamped. The Ross Cord Blood Bank, however, waits for the placenta to be delivered. Tissue procurement technicians then collect the umbilical cord blood by placing the placenta on an absorbent sheet of paper that soaks up the blood covering the placenta. The cord, which hangs through a hole in the absorbent sheet, is swabbed with alcohol. The technician extracts the blood with a syringe. Both methods yield from 60 to 120 milliliters-equivalent to about one-half to one cup. The chances of the mother's blood mixing with her infant's blood are slim to none, according to Long. "There's less than a one percent chance -like 0.02%."

Once the cord blood is collected, it is shipped to a lab for testing and processing. Lab technicians run the blood through a battery of tests, extract the stem cells from the sample, and prepare the sample for the freezer, where the cells remain until they are needed.

The umbilical cord contains two arteries and one vein, all connected to the placenta. Here, in the umbilical cord and through the placenta, is where nutrients and waste travel between the fetus and the mother. The placenta is the interface between the mother and the developing baby. It serves as a temporary lung, intestine, and kidney, supplying the growing baby with food, water, oxygen, and waste removal throughout fetal development. The placenta also serves as a barrier, making sure maternal and fetal blood never mix. This protects the fetus from a reaction by the mother's immune system.

Cord blood stem cells are naive-even more so than bone marrow stem cells. This means they don't know "self"; that is, the immune system hasn't developed enough for recognition. This makes sense, since it takes about six to nine months for the immune system to develop fully after a baby is born.

Also, using umbilical cord stem cells in transplants seems to decrease the occurrence of graft-versus-host disease (GVHD), a condition that happens when the transplanted tissue, or graft, attacks the recipient, or host. "Since the lymphocytes in cord blood have not yet developed a strong sense of self, they tend not to produce graft-versus-host disease at the same rate unrelated marrow does," Lasky explains. Lymphocytes are white blood cells that help the body fight infections.

In addition to less incidence of GVHD, there seems to be less of a need to precisely match the six HLAs, while "bone marrow transplants have a poor chance of success unless at least four of the six major HLAs . . . are alike in donor and recipient," according to the Journal of the National Cancer Institute.

Transplant success

Studies within the past five years have shown success in using cord blood stem cells in allogeneic transplants. Allogeneic transplants mean the tissue comes from a person other than the recipient-either a sibling, another relative, or a non-relative. Conversely, an autologous transplant means the patient uses his own tissue.

A 1998 New England Journal of Medicine study on utilizing allogeneic umbilical cord blood stem cells reported that, during the first few years of using these cells in transplants, "the placental blood was obtained from siblings, but now recipients of transplants from unrelated donors . . . account for almost all patients who receive transplants." The researchers followed 562 children who had received non-related cord blood in a transplant.

In the study, information concerning acute-or short-term-GVHD was available for 399 of the 562 patients. Of those 399 patients, 118 (30%) had no GVHD, while 42 (11%) had severe GVHD. Of the 562 patients in the study, 378 (67%) had leukemia or lymphoma; 137 (24%) had a genetic disease; and 47 (eight percent) had an acquired disease.

By the time the study was published in November, 1998, 51 (14%) of the patients with leukemia had relapsed, as did two of the 13 patients with lymphoma. Of the 218 patients who died, GVHD was cited as the cause of death in 11%, while most (47%) died from infection.

The researchers concluded that "placental-- blood transplants regularly engraft, cause GVHD at a relatively low rate, and produce survival rates similar to those with transplantation of bone marrow from unrelated donors." The majority of patients (137) were between six and 11 years old. Most patients in the study weighed from 44 to 85 pounds.

"The chances of graft failure increase as a person weighs more," cautions Lasky, whose lab is working on expanding the amount of stem cells in a sample. In fact, one of the main disadvantages to using cord blood stem cells in transplants is the finite amount of material in each unit. "Once harvested, there's no cord to go back to for more. If stem cell expansion becomes successful, then the restriction of sample size may not apply."

This kind of research, although experimental, gives private cord blood banks even more reason to claim that not banking cord blood amounts to throwing away a valuable resource that could be used by that child later in life. Conscientious parents are faced with determining the likelihood their offspring or family will ever need a stored sample, and physicians are encouraged to embrace a new technique that the CBR calls "integral to a well-rounded obstetrical and prenatal program.

The number of storage facilities cropping up worldwide-from India to Australia, Europe to Canada-echoes the promise of umbilical cord blood in treating various diseases. At least a dozen public and private cord blood banks are in business in the U.S. According to Long, the Red Cross has plans to establish five or six more cord blood banks. Even with the proliferation of storage facilities and the promise that cord blood stem cells may be the next best thing in curing certain diseases, patients and doctors should realize that using these cells is still experimental.

Yet, with all the promise of healing and money-making surrounding cord blood, it seems logical that some entity would step in to regulate the tissue. The Food and Drug Administration (FDA) regulates other human tissues, such as red blood cells, but doesn't have much of a role in overseeing cord blood banking. Why is this so?

"That's the $64,000 question," indicates Lorrie Harrison of the FDA's Center for Biologics Evaluation and Research. "At this point, there is little data to support whether or not stem cells will work down the road; if they're being properly processed; if there will really be a need for them; if people are just throwing their money away. Those are questions we can't answer right now because we just don't have scientific data."

The Ross Cord Blood Bank was the first American Red Cross chapter to earn FDA permission to proceed with collecting, processing, and storing stem cells. In November, 1998, it collected its first donor stem cell sample from Colin Ohr, born Nov. 3. His mother, Misty, works as a bone marrow transplant nurse for The Ohio State University Hospitals. "Bone marrow transplantation is near and dear to my heart," she said. "I've seen patients who couldn't find a donor. Cord blood is such a precious resource. I can't imagine just throwing it into a trash can."

The Ross Cord Blood Bank aims for 1-500 to 2,000 cord blood samples within three to five years, Long says. Of the 10,000 privately stored stem cell units at the CBR, seven have been used for same-family transplants. None of the recipients has been the child who donated. This begs the obvious question surrounding family banking: Why bother? "The stem cell sample is for the whole family-- brothers and sisters, fathers and mothers and grandparents," Kaesshaefer points out.

According to Lasky, more than 25 transplants are done each month with allogeneic cord blood. "A large number of these transplants are among siblings. If it's a HLA-identical sibling, the transplanted cord blood probably has a better chance of engrafting than would any other tissue, including bone marrow."

A 1997 study, published in the New England Journal of Medicine, found that the one-year survival rate in a group of 78 patients receiving cord blood from a relative was 63%, while the survival rate among 65 patients receiving cord blood from a non-relative was 29%.

Understanding options

Parents need to understand their options, notes Judy Hill, an Ohio regional cord blood educator for the CBR who teaches parents and physicians about cord blood storage. The public Ross Cord Blood Bank also does its share to educate the public.

"We make sure parents know there are other options," Long explains. "When a family decides they want to store a unit in our bank, they go through an informed consent procedure. Once they consent, the cord blood becomes property of our bank-the parents relinquish all control." Unlike donating a pint of blood through a private blood bank, where donors are usually paid, parents wishing to store the blood strictly for family use need to go through a private bank.

Cindy Monroe embraces the peace of mind the private banks sell. "The cord blood is now available to me in case I relapse and need a bone marrow transplant. Brent may save my life one day."

Had Cindy Monroe not had cancer, she likely never would have donated the blood, much less had it stored for future family use. After all, she didn't have a family history of cancer.

It is hard to say if any of her immediate family members will ever need the stem cell sample. There are no conclusive data on how long stem cells last in cold storage. Besides, the likelihood of Brent using his own cord blood sample seems small. Depending on which study you read, the chance of a child using his or her own stem cells is estimated at between one in 1,000 and one in 200,000 by age 18, according to the American College of Gynecologists and Obstetricians. Moreover, for youngsters who develop diseases, their own cord blood may not be the best option. "If a child develops a leukemia and uses his own cord blood, chances are that the cancerous genes are going to be in that cord cell unit and you'll be transplanting another leukemic cell line into that child," Long warns.

The Ross Cord Blood Bank does not routinely bank blood specifically for a family's use, he says. Yet, if a physician determines that the cord blood should be collected for an established family medical need, the bank would save the blood for the family. With bone marrow, potential donors sign up with the National Bone Marrow Registry, but years can go by before the registry contacts them. In the meantime, the donor may have moved, changed his mind, or developed a disease. Nevertheless, a stem cell sample may be readily available and provide the recipient with immediate access to the stem cells. "It's in the freezer, ready to go," Lasky points out.

While Brent Monroe's stem cells wait in a freezer in case his family needs them and Colin Ohr's stem cells remain part of the growing pool of samples in the American Red Cross public cord blood bank, researchers continue to seek answers to the scientific and ethical questions surrounding this new promise of providing another means for life and hope. "Using cord blood stem cells in transplants is still such a new field," Long cautions. "It's hard to tell what the future holds."

Copyright (c) 2000 Bell & Howell Information and Learning Company. All rights reserved. Copyright Society for Advancement of Education Mar 2000

Holly Wagner is a science writer in the Office of University Communications, Ohio State University, Columbus.

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