Privately banked cord blood and placenta-derived stem cells have great potential to expand a family’s treatment options for a variety of serious and often life-threatening diseases. Whether you’ve taken the important step of banking your newborn’s stem cells or are just beginning to research the possibilities, you may be wondering what happens if you really need to use the banked stem cells.

Understanding the transplant process can help you make informed choices should your child (or perhaps another family member) ever need to use the stem cell unit for treatment. To help you learn more, here’s what every parent ought to know about cord blood transplants:

What is a stem cell transplant?

A stem cell transplant is a therapeutic procedure used to treat patients whose stem cells are abnormal, weakened, or destroyed by disease or treatment. Healthy stem cells infused into the body can replenish themselves and transform into other cell types, including red blood cells, white blood cells and platelets, thus enabling them to replace the unhealthy cells. For therapy to be successful, the healthy stem cells must be transplanted and undergo engraftment and differentiation.

Sources of healthy stem cells include umbilical cord blood and the placenta, bone marrow, or peripheral (circulating) blood. A stem cell transplant may use stem cells taken from the patient, a family member or an unrelated donor.

How can a stem cell transplant help my child or other loved ones?

Stem cell transplants have been used to treat serious diseases in children and adults including various cancers such as leukemia, certain lymphomas, and many other diseases. To date, there are more than 80 diseases for which cord blood stem cell transplants have been used to treat patients. (1)

In addition, research is currently underway to find new medical uses for stem cells, including treatments for Alzheimer’s disease, cancer, Parkinson’s disease, stroke, diabetes, Lou Gehrig’s disease (ALS), cardiac repair and spinal cord injuries. In time, through the use of stem cells, beneficial treatments for one or more of these conditions could become a reality.

Why use privately banked stem cells for transplantation, instead of a publicly donated cord blood unit or bone marrow?

Private cord blood banking (also known as family banking) markedly improves the likelihood that a suitable transplant will be immediately available. More than 30% of patients requiring transplant therapy are unable to find a suitable match.(2) The best match is a perfect 6/6 or 5/6 HLA match for the recipient, meaning he or she will have the same six (or five) antigens as the patient. Without a privately banked unit, a search could take months and still be unsuccessful. The benefit of collecting and storing stem cells at birth is that, should the child develop any of certain rare, often fatal diseases, the child’s stem cells will be readily available for transplantation and that may help in restoring the patient’s health. The child’s stem cells will always be a perfect 6/6 match for that child.

Additionally, a cord blood transplant does not have to be a perfect match for the patient. This is because the immune cells in umbilical cord blood are less mature than those in adult bone marrow. This has particular relevance for persons of ethnic or racial minorities, adopted children and others with rare tissue types who may have a harder time finding a transplant match. Patients receiving cord blood stem cells rather than bone marrow stem cells are at lower risk for complications from graft versus host disease (GVHD), a life-threatening immune response to the transplant process. There is also a lower risk of infectious disease transmission from transplant when using umbilical cord blood stem cells compared to bone marrow stem cells.(3)

Family members may benefit from private banking, as there’s a stronger chance of a suitable match for the baby’s siblings, parents, grandparents and other blood relatives compared with the use of stem cells from a public registry. In fact, a study in the New England Journal of Medicine showed that the 1-year survival rate for patients treated with cord blood stem cells from a relative was 63%—compared with only 29% from unrelated donors, and that number increases if the transplant recipient is using his or her own stem cells.(4)

Stem cells from umbilical cord blood and the placenta from a family member also offer the advantage of time. Cord blood stem cells have already been collected and thus are readily available for the patient; simply confirming compatibility between the stem cells and the patient, transporting the cells to the patient’s location, and thawing them are necessary for the transplant. For bone marrow and peripheral blood stem cells, a compatible donor must be identified, which may take months if it is even possible. Cryogenically preserved cord blood stem cells may be successfully transplanted after up to 10 years or more in storage.(5)

If a transplant is ever needed, what should my family do to prepare for the transplant process?

Should your child or another family member ever require a cord blood stem cell transplant, it is important to prepare for treatment by working with your healthcare team. A good way to begin is by making a list of questions and concerns to discuss with the transplant doctor. Possible questions include:

• Do you recommend a transplant using the patient’s own stem cells or those from a donor? Why?

• What are the treatment goals – a long-term remission, a cure, or to alleviate symptoms?

• Are there potential side effects or complications that make this treatment risky?

• How can side effects or complications be prevented or alleviated?

• What is the anticipated length of treatment and hospitalization?

• Are there activities that the patient should avoid during treatment?

• After the transplant occurs, what are the next steps in treatment and recovery?

In addition to talking to your healthcare provider, you can request that your insurance carrier provide a transplant insurance case manager who can answer coverage issues. Assistance may be available to patients who do not have insurance coverage. Patients also may be eligible for government healthcare programs or other forms of financial help.

Should you privately bank your child’s stem cells, you should keep all paperwork associated with the process in a readily available folder or envelope, whether or not a transplant is ever needed. This way, the contact information, stem cell unit number, etc. will be readily available.

How do I release privately banked stem cells for transplant, if needed?

To request your child’s cord blood and placenta-derived stem cells for transplantation, you or the transplant center must first call the cord blood bank to request that your cord blood stem cell units be released. The cord blood bank will then coordinate the details with the transplant center, such as necessary testing and the delivery date. This may take only a few days, or as little as 24 hours in an emergency.

After signatures are obtained from the parent or guardian, the cord blood bank will ship the cord blood stem cell unit(s) to the transplant center via a specialized medical courier. The courier service should use liquid nitrogen dry shippers, which are designed to safely transport stem cell units at cryogenic temperatures. This stands in sharp contrast with the bone marrow transplant process, which requires an average of 4 months to locate a matching unit and transplant the stem cells, assuming a donor is available.

How does the stem cell transplant process work?

A successful stem cell transplant process begins with a conditioning or preparative regimen designed to suppress immune reactions and eradicate the recipient's disease. If the transplant comes from a donor instead of using the recipient's own stem cells, additional immunosuppressive medications may need to be administered.

The stem cells are then infused into the blood stream through an intravenous catheter. The transplanted cells circulate in the blood stream and travel to the bone marrow. Bone marrow transplants utilize a large dose of stem cells. For a cord blood transplant, just few ounces of the stem cell unit need to be used for transplant.(5)

Engraftment takes place as the cells integrate into the surrounding tissue in the bone marrow. At this point, the stem cells will begin to grow and produce new red blood cells, white blood cells, and platelets. Within a few weeks, the need for therapeutic transfusions may be alleviated as blood cell counts rise toward normal. If the transplant is a success, the stem cells will continue to produce new, properly functioning blood cells as needed for the rest of the recipient's life.

You or a loved one may never need a stem cell transplant. However, understanding the transplantation process will help you take steps to protect your family’s future health and will enable you to be better prepared should the need ever arise.

Sources:

(1) The Leukemia & Lymphoma Society. Cord blood stem cell transplantation. Available at: http://www.leukemia-lymphoma.org/attachments/National/br_1128624081.pdf. Accessed October 29, 2007.

(2) Wagner, J, et al: Blood 2000 (5):1611-1618

(3) Lower risk of infectious disease transmission with USB vs BM stem cells. Behzad-Behbahani,A, et al., Transplantation Proceedings.37(7): 3211-3212, 2005.

(4) Gluckman E, Rocha V, Boyer-Chammard A, et al. Outcome of cord blood transplantation from related and unrelated donors. N Engl J Med. 1997; 337:373-381.

(5) The National Cord Blood Program. Comparison Between Bone Marrow or Peripheral Blood Stem Cells and Cord Blood Donated for Transplantation. Available at: http://www.nationalcordbloodprogram.org/qa/comparison.html . Accessed October 29, 2007.