For those who can’t get enough of Clay Jones, he is now available in multimedia through the magic of podcasts! Dr. Jones was interviewed for The Prism blog last Monday, discussing the general topic of alternative medicine and pediatrics, followed by a dive into fluoride and cavities in kids. It is available for your listening pleasure at their website or on iTunes. Next step, a semi-hostile takeover of Mark Crislip’s multimedia empire – Ed
A family has many choices to make as the arrival of a new baby approaches. What will they name their child? Will they breast or bottle feed? Should they use cloth of disposable diapers? What about circumcision? Will they vaccinate or not? Some of these choices are relatively minor while some may significantly impact the health of their child for years to come. A fairly recent addition to the long list of choices that parents are burdened with, thanks to a push from reputable organizations like the American Academy of Pediatrics, as well as private companies looking to turn a profit, is what to do with the blood in their newborn infant’s umbilical cord.
Currently the most commonly-chosen option remains to simply leave it in there. In that case, it will be discarded along with the mother’s placenta or even occasionally eaten although that is a topic for another post perhaps. Another option is to have blood from the umbilical cord donated to a public cord blood bank. These have been popping up all over the place and public banking is currently recommended by the AAP whenever possible. The final option, which is by far the most controversial (and expensive), is paying to have the umbilical cord blood banked privately for personal use by the donating child or a family member. As I will explain, while not entirely without potential benefit, the private banking of cord blood is probably not a good idea and the thousands of dollars that it costs might be better spent elsewhere. Unfortunately, because of the fear of making a wrong choice, many parents are vulnerable to being persuaded by the calculated misinformation produced by these companies.
Why umbilical cord blood?
There is a very good reason why the question of banking umbilical cord blood is such an important one. The use of cord blood, or more accurately the multipotent (possibly pluripotent) hematopoietic stem cells which are present within cord blood, is a modern medical advance that has greatly improved outcomes in a subset of very ill patients. These are patients with a variety of life threatening conditions including cancers and other diseases of the blood, genetic disorders and inborn errors of metabolism.
At times, either because of a side effect of treatment or the purposeful intent of it, a patient’s own bone marrow is wiped out and must be replaced with stem cells in order to regain function – the function in question being the patient’s immune system and ability to produce red blood cells and platelets. This is known as a hematopoietic stem cell transplant (HSCT) or perhaps still more commonly, a bone marrow transplant. These stem cells can come from either the patient themselves, if harvested prior to the initiation of therapy, or from a donor. Whether their own (autologous) stem cells, or from a relative or unrelated donor (allogenic), historically stem cells were harvested from bone marrow or peripheral blood. The use of cord blood stem cells has, in more recent years, added a third option that is better in some instances and less helpful in others.
HSCT is very risky, with mortality rates as high as 10%, and many more patients must live with chronic side effects after the procedure. Life threatening infections and debilitating breakdown of a patient’s mucous membranes are probably the two most well-known examples of possible morbidity, but severe liver disease, complications from bleeding and graft-versus-host disease (GVHD) are also common. GVHD occurs when the patient’s new immune system targets a variety of tissues in the body. It can be quite severe and it can become chronic. There is even the increased risk of other cancers down the road. With this in mind, HSCT is only attempted after a thorough assessment of the risks and benefits and it is never something that is taken lightly by those who provide care for these patients.
During our training, every pediatric resident quickly learns a healthy respect for HSCT on their first Heme/Onc rotation. The patients are challenging to say the least, and we’ve all seen kids saved from their cancer only to suffer greatly from the side effects of the cure. Any advancement that might lead to an improvement in outcomes is celebrated, and the use of cord blood stem cells is one of those advances.
What are the benefits of HSCT with cord blood?
We know that the likelihood that transplanted stem cells will take hold and begin producing progenitors, a process called engraftment, and the ultimate survival rates of patients undergoing HSCT, are better when donor and patient are genetically matched. This is easier said than done unfortunately, particularly in the case of patients that aren’t of northern European descent. Avoiding the issue altogether by simply using the patient’s own stem cells is preferred when possible, but there are many instances where it isn’t a good idea, such as when the disease being treated involves that patient’s bone marrow.
Full siblings are a great option when available, but each has only a 25% chance of being a full match. Next on the list of preference would be an unrelated but fully-matched donor. There is a national registry of altruistic potential donors but the process takes time and often there isn’t an available match. Many patients have died while going through this system. Use of a mismatched donor is done, but it is considerably more risky with much higher rates of morbidity, in particular GVHD, and mortality. This is where cord blood has come into play.
The potential benefits of using cord blood are impressive, and these days there are almost always available units for children who need it. First off, it is available relatively quickly because testing for safety and genetics has already been done. There is no need for the preparatory regimens required for harvesting peripheral blood from a donor, which does have risk, or even the painful procedure of obtaining stem cells from a donor’s bone marrow. In fact, there is no risk to baby or mother with procurement of cord blood at the time of birth.
Cord blood transplant is also considerably less likely to transmit any blood-borne infection. There is also the well-established benefit of less risk of GVHD compared to equally mismatched bone marrow or peripheral blood stem cells from a donor. The donor pool of cord blood is also much more easily expanded, and in fact there already is greater coverage of ethnic minorities in public cord blood banks than on bone marrow donor registries. It does not require any commitment from potential donors and there is no donor attrition. Most importantly, survival is better and morbidity less when compared to HSCT using equally mismatched bone marrow or peripheral blood stem cells in some conditions.
Is cord blood too good to be true?
The benefits of the appropriate use of cord blood as a source of stem cells for HSCT are rock solid, but there are downsides. Unfortunately, the risk of engraftment failure is higher with cord blood stem cells, and it can take longer for the new immune system to function. This allows more time for serious infection to occur. Cord blood is also limited by how much is available. There is a finite amount from any one donor, and often one aliquot, typically around 50ml of cord blood, is not enough for HSCT in larger children and adults. This issue has recently begun to be dealt with by using units from multiple donors, and the data is good so far. There are also certain indications for HSCT where use of cord blood stem cells may be less desirable because the patient’s new immune system might be less likely to attack any residual cancer (graft-versus-tumor-effect) and increase the chance of relapse.
Private vs. public banking
There are two ways for cord blood to be stored for potential future use. Parents can donate their child’s cord blood to a public bank or privately store it for use by that child or a family member. There is some overlap because public banks will often do directed cord blood banking for expecting families who have another child with a condition potentially treatable with HSCT. Private banks will also often provide this particular service free of charge, a practice which in my opinion is more for marketing purposes than out of the kindness of their hearts. I’ll explain why soon.
Public banking involves the collection and storage of cord blood units which are then entered into public databases and made available for anyone. Though in the past they were few in number, making availability a limiting factor, now they are fairly widespread. Also cord blood can now be shipped to central locations, opening up most of the country to public banking. They are typically non-profit. Private banks are for-profit businesses that collect and store cord blood units for possible future use by the donating child or a family member. Costs average about $3,000 to $3,500.
Since cord blood stem cells have such a potential for improving outcomes, it is easy to see why parents would be interested in private banking. They may believe that they are taking out biological insurance for their child. They might feel that the peace of mind afforded by private banking is worth the high price. Unfortunately, they are often misled by shady marketing techniques and blatant misinformation.
What is wrong with private banking?
So why does the AAP discourage private banking for future use? Why does the American College of Obstetricians and Gynecologists warn its members to provide “balanced and accurate” information on the subject to avoid misleading emotionally vulnerable parents? Essentially because the likelihood of a patient ever using a privately banked unit of their own cord blood is extremely remote, it is very expensive and there is a lot of bad information out there. There is a greater likelihood, although still quite small, that a unit of privately banked cord blood will be used for a family member, typically a sibling. These make up the majority of private bank success stories, but this is a service also frequently provided by public banks for free. Overall, the chance of a physician calling up a private bank for a child is somewhere around 1 out of 2,700.
There are limitations of using personal cord blood. There is only evidence to support its potential use for specific genetic, hematologic and oncologic conditions. There is insufficient evidence to support claims that privately stored cord blood will one day be used to cure diabetes, heart disease, or autism for just a few examples of what is often included in the marketing hype. The AAP and other organizations, such as the National Cord Blood Program, specifically speak out against speculation on future uses and against banking for “biological insurance.”
Private cord blood bank marketing
It’s hard to know where to begin when discussing the marketing schemes of private cord blood banks. There are just so many examples of fear mongering, use of deceptive testimonials, exaggerating the likelihood of ever needing to retrieve the donated unit, and much more. There are several large private banks, but I will focus on Cord Blood Registry (Cbr) because it is the biggest.
I first became aware of the shady practices of Cbr while my wife was pregnant with our second child. I was sitting in her obstetrician’s waiting room and picked up what appeared to be a generic parenting magazine. It was full of the standard boilerplate on how to prepare for a new arrival, but I found it odd that there were four separate references to private cord blood banking in the content, and a full page ad for Cbr. One such reference came in the form of a “top ten things for every expecting family to check off their list”. On the list was private cord blood banking. There was also an article promoting private banking by Dr. Robert Sears, a fellow pediatrician who I had not yet lost all respect for back in 2008. Sears is a medical consultant for Cbr by the way.
After discovering the outside back page to be a full-page ad for Cbr, I scoured the magazine and finally discovered in the fine print that Cbr had also published the magazine. The entire thing was nothing but an advertisement. I didn’t know that much about private banking at the time, but that experience inspired me to investigate further. Here are a few examples of their deceptive marketing techniques:
1. Testimonials, celebrity endorsements and arguments from authority
The Cbr website and print media contain many testimonials about successful use of cord blood stem cells, complete with videos of grateful families relating their tragic story and subsequent happy ending thanks to Cbr. They use quotes from physicians and researchers, even from a celebrity physician:
My patients and their families mean the world to me and I want to make sure they have the best treatment possible. Saving cord blood is something every expectant family should know about and consider because the potential therapeutic uses continue to expand. Research in the use of cord blood is very important and cannot continue if parents do not consider banking.
- Dr. Jennifer Arnold, neonatologist, member of the American Academy of Pediatrics and star of TLC’s The Little Couple.
Notice she didn’t say anything about private banking being a benefit, or that personal use of cord blood is likely to be recommended. It is true that parents need to consider banking, but in a public program. This is their angle with these quotes though. Almost all of these quotes apply just as well if not better to HSCT with allogenic cord blood obtained from a public bank, which costs a family nothing and is almost always an available option. Here is another example:
I strongly recommend that all expectant parents ask their doctor about the lifesaving potential of cord blood… Today, cord blood is being used to treat such life-threatening diseases as leukemia and other cancers, as well as certain blood and immune disorders that formerly necessitated a bone marrow transplant.
- Dr. Marianne Neifert, Pediatrician, Baby Talk Magazine, October 1997
Again, this actually supports public banking even more so than private and certainly doesn’t support autologous transplant with cord blood stem cells, which would not be used for cancers of the blood or immune disorders. One more:
Recipients of cord-blood transplants from HLA-identical siblings have a lower incidence rate of acute and chronic GVHD than recipients of bone marrow transplants from HLA-identical siblings.
- Drs. Vanderson Rocha, John E. Wagner, Jr., Eliane Gluckman, et. al. New England Journal of Medicine, June 22, 2000
Yes, this is true. And this is a service that is regularly provided by public cord blood banks, for free. Private banks also don’t tend to charge for storing the cord blood of siblings of a child with a potentially transplant treatable condition, but they do so because they really really need to be able to use them for marketing. If not for the use of sibling cord blood, they would almost never use any of their stored units.
2. Speculation in order to prey on parental fear of making a bad decision
The folks at Cbr love to speculate about the future benefits of stem cells. Don’t we all. But it is inappropriate to use this speculation to take advantage of a vulnerable population. They also frequently discuss the future potential of stem cells in general, leaving the parent to assume that the benefit would only apply to cord blood derived stem cells. If we are talking about benefits decades down the line, who is to say that we won’t be able to simply use peripheral stem cells or make those harvested from skin pluripotent. Here are two examples:
Just like we didn’t envision the powers of PCs thirty years ago, we may not be seeing all the uses of cord blood yet.
- Dr. LeeAnn Jensen, Immunologist, National Institutes of Health
A really exciting area that’s just now in the basic science lab, is the area where stem cells…can be used for going over to muscle cells like your heart muscle if you had a heart attack…or brain cells if you had Parkinson’s Disease…Hopefully, in 20 or 30 or 40 years…these might be potential applications for stem cells including umbilical cord blood stem cells.
- Dr. Stanton Goldman, Pediatric Hematologist Oncologist and stem cell transplant physician, Medical City, Dallas, Texas
3. Using unpublished ongoing clinical trials and experimental treatments as marketing
This video from the Cbr website involves an infant who had injury to her brain following removal of a brain tumor. In addition to intensive therapy, she received an infusion of her own cord blood stem cells. She improved and her mother is now pleading for more parents to take out biological insurance like you would have dental insurance. There is even a disclaimer that the results were not published and may be simply due to her other interventions. There are many more like this.
Also on the website are links to a number of clinical trials that are ongoing and involve the infusion of autologous cord blood stem cells. In this example, the trial is looking at the possible benefit of cord blood stem cells on language in children with autism. There is no guarantee that any of these trials will pan out. In fact, they almost certainly won’t. It is shameful to use this as a marketing ploy to scare parents into buying a service like this.
4. Exaggerating the need and ignoring the limitations
Cbr and the other private banks claim that cord blood stem cells will still be viable even decades later, and this is likely true. What they conveniently leave out is the fact that once a child is over about 50 kg, the amount of stem cells in their privately banked unit would likely be insufficient. In adults, this is dealt with by batching multiple units of donor cord blood. If they are banking on future advances in technology, then why not just figure that cord blood stem cells will be obsolete in 50 years anyway? I don’t see the difference.
The evidence to date shows that the chance of an individual one day benefiting from HSCT using cord blood stem cells is about 1 out of 2,700. But Cbr and the other private banks state the following: “1 in 217 – for an individual (by age 70), using his or her own stem cells or someone else’s”. Even if true, the majority would not require privately banked cord blood. But this claim, which comes from a 2008 publication in Biology of Blood and Marrow Transplantation is highly speculative. The authors admit that many assumptions were made to come up with their final results. It is almost certainly overestimating the likelihood.
5. Bait and switch
Cbr and its ilk frequently point out the many conditions that doctors have attempted to treat with HSCT. Naturally they include references to how cord blood stem cells may lead to improved outcomes for some of these conditions compared to bone marrow or peripheral blood derived stem cells. What they frequently omit is the fact that most of these conditions would never be treated with a child’s own hematopioetic stem cells because that is where the disease process originated from in the first place. HSCT transplant would only be performed using donor stem cells. A good example is none other than acute lymphocytic leukemia, the most common cancer in childhood.
What do pediatricians have to say about cord blood banking?
The stance of the AAP is reasonable and based on good evidence, but it doesn’t do much good if the pediatricians practicing on the front lines aren’t aware of it. So what is the state of understanding of cord blood banking among pediatricians? Periodically the AAP surveys its members to answer such questions. One such survey, in this case examining counseling practices and attitudes regarding cord blood banking, was initiated by the AAP Section on Hematology/Oncology and ran from October 2007 to March 2008.
Just over 1,100 pediatricians responded, and this was whittled down to the 700 that actually provide health supervision. The results were discouraging. Most of the respondents weren’t familiar with the AAP statement, with more than half being completely unaware of its existence or only vaguely familiar. Only 4% claimed to have a good working knowledge of the AAP’s stance on cord blood banking.
Most pediatricians weren’t aware of the options for cord blood banking in their area of practice. Not surprisingly, cord blood banking does not appear to be something that is commonly discussed with expectant mothers, and it is almost always brought up by the parents rather than the physician. Equally unsurprising is that almost a third of pediatricians reported that they were aware of parents in their practice being solicited by private cord blood banking companies.
Pediatricians likely do not discuss cord blood banking with families for a variety of reasons. Almost all practicing pediatricians do see expectant mothers (84% of survey respondents), so it isn’t because of a lack of opportunity. Many simply aren’t comfortable bringing it up. Six out of ten respondents reported that they lack confidence in their ability to discuss the science behind cord blood banking. Only 18% reported that they think they have a good understanding and are comfortable talking about it.
Stem cells derived from cord blood collected at the time of birth are a wonderful addition to science-based treatment of a variety of life threatening conditions, including some of the most common causes of cancer in children. The benefits of their use are substantial, but they are far from perfect. Just what to do with a child’s cord blood is controversial, but it shouldn’t be. Public cord blood banking is increasingly available and is recommended by the majority of experts because it helps make sure that a match is always available for every child who would benefit from cord blood-derived stem cells.
The chance that privately-banked cord blood will ever be used by the donating child is extremely small. And both public and private systems allow for free storage of cord blood when there is a family member with a condition that might require stem cell transplant. If you have the money, it isn’t wrong to use a private bank, just don’t believe the marketing hype put out by private cord blood banks. Pediatricians should serve as a source of unbiased information but unfortunately it seems that most have not kept up to date with this issue.