Facts about Umbilical Cord Therapeutics and ALS
Roberta Friedman, Ph.D., Research Department Information Coordinator

Nathalie Kayadjanian, Ph.D., Associate Director of Biomedical Research

The recognition by researchers and subsequently by the public that a baby’s umbilical cord can provide stem cells gives hope for countering disease but also gives pause at the possibilities for misinformation and commercial exploitation. Cord blood banking is already a well established and largely unregulated enterprise. It is an area with ample potential for pitfalls and disappointment as well as for medical advances.

ALS is inevitably fatal, so the possibility that cord blood collected from newborns could provide a therapeutic is understandably compelling for families with an affected loved one. In this document, The ALS Association seeks to provide information to help families make decisions about cord blood banking and to understand the science behind the potential for cord blood to be a source of therapy.

We must emphasize at the outset that no effective treatment for ALS or any human clinical trial of cord blood products in ALS is yet available. All work is taking place in animals and in lab dishes. The ALS Association is funding research into stem cells as well as any other opportunity to provide therapeutics for the disease. Please monitor our web site to keep current with research developments in this and other ALS related fields.

Cord Blood: The Biology

The umbilical cord, cut from the baby and delivered soon after birth with the placenta, is a rich source of many types of cells.

The first use proposed and implemented for cord blood was for cancer. Blood from the cord can serve in the same way as bone marrow to rebuild a patient’s blood and immune cell supply. This allows patients to survive after intensive therapy that destroys a patient’s own bone marrow in the attempt to wipe out cancer cells. A recent study showed that umbilical cord blood transplants may offer blood cancer patients better outcomes than bone marrow transplants (analysis of outcome data performed at the Statistical Center, Center for Blood and Marrow Transplant Research at the Medical College of Wisconsin, Milwaukee).

Stem Cells from Umbilical Cords

Stem cells are of such interest because they can make any type of cell as does the developing embryo, which starts from a fertilized egg to generate all the specialized cell types. This property of stem cells (called pluripotency: see The ALS Association’s stem cell primer at http://www.alsa.org/research/article.cfm?id=715) gives promise for medical therapies to replace or repair dying cells in a variety of disorders including ALS.

So the umbilical cord can be a source of stem cells and because the cord is a byproduct of birth, usually discarded, saving it and its cells can proceed without ethical questions.

The Umbilical Cord --Source of Therapeutics

Besides cancer, cord blood cells are used to treat some immune deficiencies and blood disorders, such as sickle cell anemia and aplastic anemia. More than 8,000 cord cell transplants have been carried out worldwide.

Umbilical cord blood stem cells are more able to match the recipient’s so-called “self” markers to avoid immediate as well as delayed rejection*. They also proliferate in the lab more readily than do similar cells from bone marrow.

[*Details: Human Leukocyte Antigen (HLA)-matching refers to six of the many proteins that appear on the surface of white blood cells and other tissues in the body to mark cells as “self” so they are not attacked and destroyed by the immune system that patrols the bloodstream to counter infection and cancer.

The six HLA genes help determine tissue transplant compatibility between donor and recipient. Cord blood stem cells are immunologically "younger" than bone marrow stem cells. So a greater degree of mismatch between donor and recipient cells is tolerable with cord blood stem cells as compared to bone marrow cells. Because cord blood stem cells are less mature than bone marrow stem cells, they are less likely to attack the patient's body (graft versus host disease, a complication of transplants). Cord blood stem cells from a related donor, such as a brother or sister, provide the greatest likelihood for a match.]

The Future for Therapeutics

Nearly 80 clinical trials studying the uses of cord blood stem cells are currently registered with the FDA. Studies in various stages prior to patient testing are underway in heart disease, diabetes, stroke, Parkinson's, spinal cord injuries, Alzheimer's, muscular dystrophy and liver disease, as well as in ALS.

Potential in ALS

Researchers at the University of South Florida showed that infusion of mononuclear cells from human umbilical cord blood could modestly prolong the lifespan and delay onset of disease in SOD1 mutant mice. The effect due to the cell infusion may be due to changing the activity of the immune system modulation, in addition to anti-inflammatory effects.

But clinical application is not yet around the corner. Researchers working at the University of Ulm, Germany, and colleagues published in the Journal of Neural Transmission that cells derived from human bone marrow called mesodermal stromal cells, as well as umbilical cord blood cells, had little ability to aid in the mouse model of ALS. They directly injected either type of cells into the fluid that bathes the brain and spinal cord. Ten days after the transplant, only a few cells could be seen around the spinal cord. Survival times of the mice did not change with the treatment. Strategies will have to be explored to improve the delivery of transplanted cells into the regions of interest for the treatment of ALS.

Should You Store Your Baby’s Cord Blood?

It is up to parents to decide whether to collect and store a baby’s cord blood. There are no standards or set policies for transport, safety testing, or screening for the presence of communicable diseases. Nor are there any standards established to ensure that cells are collected properly or properly preserved or even how to best reactivate the cells after storage. Perhaps the cells might not be what is expected if one seeks to bring them out of storage at a future time when they would be needed.

Stem Cell “Cures” Not Yet Available

Many ALS patients and their families hope that regenerative medicine investigations, including that using stem cells from umbilical cord, will find a way to heal the damage in the disease. At present (summer 2007), nerve repair has only been partially achieved in animals with certain spinal injury. The Internet contains many posts from patients seeking unregulated treatments and references to clinics which offer them. Some seeking a stem cell "cure" have traveled to countries with lax medical regulations. They may have received infusions of stem cells from cord blood or from aborted fetuses.

Potentially harmful effects have occasionally been documented to follow the treatments. Please discuss any plans to seek such treatment with your care team including your physician specializing in ALS.

The following are several Internet locations that give further information.

A Parent's Guide to Cord Blood Banks provides detailed scientific and medical background on the issue, listings worldwide of cord banks as well a questionnaire for interviewing and evaluating private cord blood banks. Other useful information included is the cost and legal issues. It is addressing mainly the issue of cord blood as a blood cell replacement yet includes areas of current research such as ALS. The strategy is to inform but leave the final decision to the reader.
About Cord Blood Banking gives discussion by a doctor of whether to bank your baby's umbilical cord blood
Cord Blood Donor Foundation provides educational awareness and further research in the use of cord blood stem cells. You can obtain a list of cord blood donor banks in the United States.
Donate Your Baby's Umbilical Cord Blood offers information on cord blood donation from the National Marrow Donor Program (NMDP). Links on this page lead to longer explanations and to an extensive list of cord banks, both NMDP members and others.
Final Thoughts [from A Parent's Guide to Cord Blood Banks:]

It costs about one thousand dollars to process cord blood for storage. A banker of cord blood has budgetary limits on how many donations to accept for free. An income may be necessary to support free collections. This funding could come from federal or other grants, it may come from profits from private deposits or from selling some of the donations to research.

Also, banks which are saving cord blood for transplants can only afford to collect from nearby hospitals during staffed hours. You may have to inquire based on the hospital where you plan to deliver.

Not everyone is eligible to donate cord blood. To learn more about the requirements click on Informed Consent. And inquire with the person who will deliver your baby: many doctors charge over $100 for collecting cord blood, even if the blood is donated for the public good.