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JDRF statement

The Therapeutic Potential of Stem Cells From Umbilical Cord Blood

In the last few decades, the scientific community has recognized the potential of adult and embryonic stem cells to treat diseases and to regenerate new organ tissues for a variety or ailments ranging from type 1 diabetes to Parkinson's disease to spinal cord injuries.

One source of adult stem cells is the umbilical cord blood (UCB) - the blood which is extracted from the umbilical cord and placenta at the time of childbirth, after the cord has been detached from the newborn. UCB contains blood-forming stem cells, similar to those found in bone marrow. Due to its ease of collection and the fact that it used to be a discarded material, UCB presents an appealing, potentially lifesaving source of stem cells. Researchers have recently recovered stem cells from umbilical cord blood which have additional potential to become organs or tissues.

Clinical Use
So far UCB cells have largely been used in clinical transplants as a substitute for bone marrow transplantation to treat malignancies, bone marrow failure, and a few other conditions. There are public cord blood banks (http://www.marrow.org/) that store donated UCB for use by any patient who needs a transplant. In addition, there are private cord blood banks that store UCB for personal use. At present, the true therapeutic applications of UCB cells are yet to be determined.

JDRF Research
JDRF co-funded the first clinical trial evaluating use of autologous UCB infusion to ameliorate the progression of type 1 diabetes. The study results, published in Diabetes Care[1], showed that the treatment is safe. However, this therapeutic approach has yet to demonstrate a clinical benefit. Larger randomized studies are needed to determine the efficacy of autologous UCB for treatment of type 1 diabetes.  

Storing A Child's Cord Blood At Birth
Because of the interest in using UCB in cell therapies, parents frequently ask whether they should store their newborn's UCB cells in a private or public cord blood bank, particularly when a parent or sibling has type 1 diabetes. While this is a matter of personal choice, some information on the state of the science that might be helpful in making a decision includes:

In 2007, the American Academy of Pediatrics issued a policy statement[2] evaluating cord blood storage by both public and private entities: 

  • Private UCB banking businesses store the donors' cells with the possibility of using them to the donors' own benefit in the future. These businesses, however, sometimes appeal to parents' emotional stress and tout unrealistic medical benefits. There is evidence that the UCB from a child with genetic defects also carry the same genetic mutations in its stem cell population, and the potential for children needing transplantation of their own UCB is still doubtful.  However, parents are encouraged to store a newborn's UCB if a first-degree relative has been diagnosed with a disease treatable with a transplant.
  • Public UCB banking[3] initiatives aim to collect large populations of these cells that may cover as many genetic variations as possible to maximize patient matching probabilities. These efforts serve the public interest and the cells stored are not meant to be returned to the donors.

As a result, the American Academy of Pediatrics has made the following recommendations for prospective parents:

  1. UCB donation should be discouraged when its storage is directed for later personal or family use. Most premalignant changes already exist in the UCB, and the transplantation of such UCB may not confer any medical benefits. Direct UCB banking is encouraged when there is a full sibling in the family with a medical condition that can benefit from UCB transplantation.
  2. UCB donation should be encouraged when it is banked for public use. Parents will be notified if abnormalities are found in the UCB during standard laboratory testing. These donations, however, may not be available for future private use.
  3. There is insufficient scientific data to support UCB banking for future private use. Private banking as a "biological insurance" should be discouraged. All UCB banks should be accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) and follow FACT standards.

JDRF's position on storing cord blood
JDRF encourages expecting parents to consider donating their child's UCB for research purposes. Information on UCB donation can be found through the following web site: http://www.marrow.org/HELP/Donate_Cord_Blood_Share_Life/index.html

JDRF cannot encourage UCB storage for therapeutic purposes at this time given the lack of current clinical data demonstrating efficacy of UCB in preserving or restoring c-peptide in patients with type 1 diabetes.



[1] Haller MJ, et. al. Autologous Umbilical Cord Blood Transfusion in Very Young Children with Type 1 Diabetes. Diabetes Care 2009 Nov  32(11):pp-pp. 
[2] http://aappolicy.aappublications.org/cgi/content/full/pediatrics;119/1/165 
[3] http://www.marrow.org/