Skip to comments.Blood Treatment's Promise Mired in Bureaucracy
Posted on 05/29/2004 1:08:18 AM PDT by neverdem
Megan Cardy is one of the lucky ones. Her doctors found a match.
Megan was 14 when her leukemia returned. Having already endured chemotherapy, she dreaded the prospect of another long course of treatment. So Megan and her parents decided on a bone-marrow transplant that would provide blood stem cells to enable her body to produce healthy blood.
After her doctors spent six months searching the donor lists for stem cells close enough to Megan's own tissue, she got the stem cells she needed but not from a bone-marrow donor. Instead, the source was umbilical cord blood, a byproduct of normal childbirth that is rich in stem cells that are more adaptable than those in bone marrow. Three years later, Megan's leukemia remains in remission.
"Cord blood is literally a lifesaver," said Megan's mother, Suzanne.
Unfortunately, doctors say, thousands of others in the United States with leukemia, lymphoma or other diseases who might benefit from a stem-cell transplant will die this year, waiting for a match. Despite cord blood's promise as a source of stem cells and its advantage of being much easier to collect than cells from a living donor there is not yet enough stored cord blood available. And while every childbirth in this country is a potential source of donation, efforts to create a readily accessible national supply of cord blood are being held back by financial and bureaucratic snags, and by a turf war between the field's two leading nonprofit players.
The stem cells at issue are not the human-embryo cells used in medical research that have become embroiled in the national abortion debate. Instead the cells in transplants like Megan Cardy's are blood stem cells primitive cells that are capable of growing into the red blood cells, white blood cells and platelets that make up healthy blood.
To provide matches for successful transplants in the 80 percent or so of the patients who might benefit, the country would need some 150,000 units of cord blood, said Dr. Pablo Rubinstein, a pioneer in the field. (A unit is the amount collected from a single cord.) By one estimate, however, there may be only 20,000 to 25,000 usable units available. Many others in storage are of questionable quality because there are no federal standards for collecting and storing the blood. And those that do exist are scattered among cord-blood banks around the country, with no central registry and database to make them easy for a doctor to find.
Experts say none of the problems are unsolvable. But in the absence of federal coordination, the industry seems unable to organize itself. So far, a lack of political consensus on the matter has kept the government from nurturing a national cord-blood system one that could function until there are enough transplants taking place to make the system self-supporting. Meanwhile, in a kind of Catch-22, the lack of a fully functioning system has curbed demand by transplant doctors for umbilical cord blood, leaving the blood banks without the money to collect and store significantly more units.
As a result, only 400 or so transplants using cord blood are now taking place in the United States each year instead of the thousands that medical experts say are needed. Compare that with Japan, where government financing has helped create a more fully functioning national cord-blood donor system; about 43 percent of stem-cell transplants in Japan were via cord blood last year.
One of the biggest impediments in this country is the turf battle between the field's major players: the nonprofit National Marrow Donor Program, based in Minneapolis, and the New York Blood Center, a nonprofit blood bank.
The Minneapolis group, which already oversees the nation's main registry of blood and marrow donors, wants to also lead the cord-blood effort. The New York Blood Center is resisting that group's oversight, arguing that the government should pick a small group of blood banks that it would finance directly.
Meanwhile, transplant doctors are growing increasingly frustrated that lives are being lost as the face-off, which began last year, drags on with no end yet in sight. "It's been a tremendous waste of time and energy arguing about it," said Dr. Joanne Kurtzberg, a leading transplanter at Duke University Medical Center.
Over Bone Marrow
Cord blood's advantages over adult bone marrow include an easier donor process . In the case of marrow, a potential volunteer donor provides a blood sample, and the tissue type is kept on file for matching purposes. But when a potential recipient turns up, that donor must then be tracked down typically for a medical procedure.
Donating cord blood is much simpler. The stem cells are collected at virtually no risk to a mother and baby at birth, when the umbilical cord and placenta would otherwise be discarded, once the mother has agreed to donate the blood. Although many units that are collected are later discarded, because they test positive for certain diseases or are too small to be of use, the blood found suitable can be stored for use whenever needed, with no need for further involvement by the donor.
The cells Megan Cardy received, through an intravenous procedure lasting less than an hour, had been frozen for two and a half years.
But if the donating is easy, collecting, processing, testing, freezing and storing the blood is expensive $1,200 or more per unit. And public cord-blood banks, most of which are nonprofit, generally receive no revenue from a donation until they sell a unit to a transplant center for about $20,000. Few organizations have the upfront money to build a substantial inventory. And many, like the American Red Cross, have significantly scaled back efforts to collect more cord blood.
The gulf between cord blood's promise and the market realities explains the desperation of people like the parents of Kailee Wells. Kailee, a 7-year-old girl adopted from China, suffers from aplastic anemia, a rare disorder in which the patient's bone marrow cannot produce blood cells. The Wellses, who are from Albuquerque but spend most of their time in Milwaukee where Kailee is being treated, have been seeking a stem-cell donor for more than two years.
"The doctors have told us Kailee needs a transplant this summer," said Owen Wells, Kailee's father, who runs a Web site, www.kaileegetwells.com, to help with the donor search.
Finding matches for ethnic minorities is particularly hard, doctors and researchers say, because there are too many people of similar background in the existing donor pool. Cord blood could help ease the scarcity. Because its stem cells are immature, they are more adaptable, resulting in fewer cases where the stem cells attack the patient's body, even when the tissue is less closely matched.
A ready national database with enough usable cord-blood units might greatly improve the odds of finding a match for sick children like Kailee Wells. In the absence of such an inventory, the one matched cord-blood unit Kailee's doctors could find had too few cells to be usable. Now Mr. Wells is desperately seeking a suitable living bone-marrow donor by setting up donor drives, asking people to have their blood tested voluntarily for a potential tissue match. It is a long shot. "We knew it was a needle in a huge haystack," he said.
In the few hundred cord-blood transplants that do take place in this country each year, children are the most frequent recipients, because of the relatively small quantity of stem cells typically yielded by a single umbilical cord. But some adults like Richard Wendland, 6-foot-2 and 185 pounds, have benefited from the treatment.
Mr. Wendland, who lives in Little Canada, Minn., near St. Paul, learned in 2000 that he had non-Hodgkin's lymphoma. He took chemotherapy and went into remission, he said, only to find out in early 2002 that the cancer was "rearing its ugly head again."
While his doctors said he would probably live about eight more years with the lymphoma, Mr. Wendland, who was 46, opted for a transplant even though he was given only 50-50 odds of surviving the transplant in the first year. Mr. Wendland's doctors were able to find a match that summer a donor unit that was large enough and a close-enough match that his doctors chose to proceed.
He received the transplant in October 2002, after a grueling round of chemotherapy and radiation intended to rid his body of any diseased cells in preparation for the transplant. The procedure itself "was anticlimactic," he said.
His doctors described it as an unusually quick recovery: Mr. Wendland was back at home in a week and returned to his job as a computer consultant for US Bank in February 2003. "I am cancer-free," he said.
A Lack of Standards
And of Regulation
The lack of standards for cord blood raises real concerns about the quality of the collecting and processing now taking place. Dr. Wagner recalls the time a couple of years ago when he received a unit of cord blood that turned out to have been misidentified by the bank.
"Fortunately, we caught it right before the transplant," said Dr. Wagner, who added that the patient would have probably died if the mistake had not been discovered.
In another case last summer that involved a different bank and is still under investigation, a transplant simply failed, possibly because that bank's units had too few of a certain kind of cell that makes a transplant successful. "Not all banks are created equal," Dr. Wagner said. "We need to have greater oversight."
The government's relative lack of involvement in the cord-blood issue includes its virtual absence as a regulator. Earlier this year the Food and Drug Administration did begin to require groups that collect cord blood to register, and the agency recently issued rules about who may donate. But there are no hard rules for the way banks collect and store the blood, and regulators perform no inspections of the operations.
"All of this tells us there is a need to have the F.D.A. involved with or without legislation," said Dr. Rubinstein, who argues that the government should set tough standards and select a handful of cord-blood banks that can meet them. The F.D.A. says it is continuing work on regulations governing the banks.
The National Marrow Donor Program, which works with a network of 12 banks, says it already oversees the quality of the units in its registry. The group says that it regularly inspects the banks and that it tracks the results of transplants using cells from individual banks to make sure there are no quality-control problems.
"I'm very confident about the quality of the units listed by the National Marrow Donor Program," said Dr. Dennis Confer, the program's chief medical officer. Even Dr. Confer, though, does not dismiss the need for greater federal oversight and the continuing development of standards. "The doctors don't want to worry about the product," he said. "They have enough to worry about already."
Some transplanters say the efforts by an existing standards group, the Foundation for the Accreditation of Cellular Therapy, appear to be the most promising. So far, the foundation has accredited three of the seven cord-blood banks it has inspected, one of them the New York Blood Center.
But as things stand in the United States, Dr. Wagner and other transplanters say they are left largely on their own in determining the quality of a bank. Dr. Wagner says he personally interviews officials at all the banks he uses. "I have to trust the blood," he said.
The Major Players
Remain at Odds
Only the field's major players currently have the ability to finance significant new collections. The National Marrow Donor Program recently said it would spend $8 million from its reserves to help the banks in its network add units. And the New York Blood Center, which relies heavily on philanthropic efforts to build its inventory, recently expanded its collection efforts to Cleveland.
But more money is needed, and Dr. Rubinstein, who runs the New York center, pushed hard last year for federal financing to develop the national inventory.
Bills were introduced in the both the House and Senate, but the fight between the New York Blood Center and the National Marrow Donor Program over who should lead a national effort fractured any chance for Congressional consensus. Although Congress did agree to provide incremental financing of $20 million over the next two years for the collection of cord blood, that financing is now in limbo, with lawmakers unwilling to put money behind the effort until administrative issues are resolved. Congress plans to spend $1 million on a study of the issue by the Institute of Medicine, which advises the government on health issues and plans to hold a meeting on the topic on this Wednesday.
An additional $20 million or so annual federal dispersal that has traditionally gone to the National Marrow Donor Program to oversee its existing registry is also uncertain.
Dr. Rubinstein and Dr. Cladd Stevens, his wife and the medical director for the New York Blood Center's program, continue to argue that the National Marrow Donor Program should not oversee a national cord-blood system. "We need federal money to build the inventory," Dr. Stevens said. "We don't think it needs to be a huge monolithic organization."
She and her husband argue that blood banks should compete for any federal financing, forming a loose network that could be overseen by a board representing doctors, patients and others.
But officials at the National Marrow Donor Program argue that theirs is the logical entity to serve as the single body overseeing all sources of stem cells for transplant, whether cord blood or bone marrow. "We don't believe they are competing technologies," said Dr. Jeffrey Chell, the program's chief executive, who also noted that the program was active in educating doctors and patients. "There would be no additional cost to a federal program to take advantage of this."
Some doctors and patient advocates agree with that position, saying the creation of a new separate network does not make sense. "It's going to duplicate resources," said Dr. Kurtzberg of Duke, who has worked closely with Dr. Rubinstein but favors a more coordinated approach.
With apparently little chance that the two sides will reach a compromise anytime soon, transplanters and doctors are waiting for the Institute of Medicine to clarify what kind of system should be used. But meanwhile, in the view of patient advocates like Frances Verter, people will continue dying because stem cells will not find their way to the patients who most need them.
Ms. Verter, a scientist at NASA, had a daughter who died of cancer in 1997, and decided when she subsequently gave birth to another daughter to store her cord blood. She now operates a Web site for parents contemplating private storage or donation of cord blood, www.parentsguidecordblood.com.
In the impasse over who should administer a national cord-blood program, Ms. Verter says, "nobody is asking what the patients want."
I just found out that John Kerry and Henry Waxman were recruited by my meds maker Serono to get FDA approval for the meds in this country. It was approved nearly everywhere else: canada, europe, etc.
But Serono has given more money to the Repubs than the Dems, even during the fight with Biogen who made a competing med.
$1200 cost for a $20,000 sale in a couple of years seems like powerful profit incentive to me, with the internet as an easy way to market.
When embryonic stem cell exploitation hit the news a few years back (early in Bush's Presidency) the expectation of some was a system of tissue banks derived from embryonic stem cell lines. The owners of these banks of immune response identified tissues would be the source for stem cell lines closely matched to the immune system of the patient, thus the banks would be tremendously profitable as the source of tissues for treatments. Using adult stem cells taken from the patient's body and coaxed into differentiating into needed tissues takes away the 'banking empowerment' anticipated by business for embryonic stem cells (the body parts of individual humans at their embryo age in their lifetime ended for harvesting purposes ... cannibalism).
There is a little discussed issue which is driving the lust for embryonic stem cell >>>>
While reading this first sentence the same thought came to my mind $$$$$ in NJ, McSleazy is pushing cannibalism big time by streamlining the application process and using public money from the budget about $6 million to start funding the research and set up institutes.
Thanks for the ping!