FREE ESSAY ON TREATING DISEASE WITH STEM CELLS

TREATING DISEASE WITH STEM CELLS

Treating Disease with Stem Cells
This article was written by Dr. Gregory Hale, professor of Pediatrics at the University
of Kentucky College of Medicine, in response to questions posed by Scientific American
Magazine regarding the treatment of certain diseases with cord blood stem cells. There is
some additional information provided by Viacord, a medical service company that provides
private family cord blood banking, processing, and research. Dr. Hale discussed the
advantages of cord blood stem cell transplants, the results of several transplants, as
well as the research that on-going in the field of stem cell transplantation.
The blood that remains in the human umbilical cord blood following birth contains a rich
source of hematopoietic progenitor cells known as "stem cells." These stem cells are the
master cells of the blood. They divide to make new red cells that supply oxygen, white
blood cells that fight disease and infection, and platelets that facilitate healing.
Doctors now have three sources of stem cells available to them: bone marrow, mobilized
bone marrow or peripheral blood, and umbilical cord blood. Bone marrow has traditionally
been used as a source of stem cells, but research is proving that cord blood may be an
excellent alternative source. Cord blood can be utilized for the treatment of many
diseases, including leukemia, sickle cell anemia, and Hodgkin's disease. The first
successful cord blood transplant took place in 1988 when a newborn's cord blood was used
in a life saving stem cell transplant for her older brother who had Fanconi anemia.
Since then, there has been much research into the advantages of cord blood stem cells.
Cord blood is much more readily available and poses no donor risk. The blood is simply
collected from the placenta and umbilical cord after the baby is born and stored. The
potential of expanding the ethnic diversity of the donor pool is greatly increased, since
minorities are considerably under-represented in bone marrow transplant pools. Cord blood
stem cells may also pose less risk of graft-versus-host disorder, or GVHD, than stem
cells from bone marrow. GVHD occurs when the donor's immune cells make antibodies against
the host's tissues, resulting in serious complications.
In the last ten years, several studies of cord blood transplants have taken place and
their results have been published in science and medical journals. In 1996, the New
England Journal of Medicine reported the results of 25 consecutive cord blood
transplantations from August 1993 to November 1995 by Dr. Joanne Kurtzberg at Duke
University. After bone marrow searches were unsuccessful for 17 patients, cord blood was
collected from unrelated donors at the New York Blood Center. Nineteen patients had
malignant diseases and four had nonmalignant conditions. The patients' average age was
seven years and the average weight was 19.4 kilograms. Twenty cord blood units were
slightly HLA-mismatched and one was HLA-identical. The HLA, or Human Leukocyte Antigen,
induces the formation of antibodies because it is recognized by the immune system as a
threat. Engraftment was successful in 23 of 25 patients, and only two patients had severe
graft-versus-host disease. In contrast, bone marrow transplants in children have a 30
percent incidence of graft-versus-host-disease. Seven of the patients with malignant
disease and 6 with nonmalignant conditions were alive one year after transplantation. The
report concluded that "HLA-mismatched cord blood is an alternative source of stem cells
for transplantation in children."
In another study by Dr. John Wagner at the University of Minnesota Hospital, 13 patients
with malignant leukemia diseases and five with nonmalignant conditions received cord
blood transplants between July 1994 and December 1995. The average age of these patients
was 2.7 years and the average weight was 15.4 kilograms. Seven patients received
HLA-matched blood and 11 received varying degrees of HLA-mismatched grafts. Six months
after transplantation, 65 percent of the patients were alive and only two developed
severe graft-versus-host disease. The authors stated that the benefits of cord blood
transplantation included the "low rate of graft-versus-host disease, rapid availability
of blood, lessened donor risk, and a low risk of transmissible infections such as
cytomegalovirus and Epstein Barr virus." 
Even with these and other successful transplantations, there are still questions to be
answered. For example, what is the minimal cell dose? Can an adult recipient be
effectively transplanted? Because the volume of umbilical blood collected at birth is
between 80 and 100 milliliters, the majority of transplants have been pediatric cases.
Bone marrow, on the other hand, gives the doctor a large supply of stem cells.
Researchers are now seeking methods to increase the number of cord blood cells for use in
larger adult patients.
Further basic science and clinical research will answer these and other dilemmas such as
the graft versus leukemia effect, how long cord blood can be preserved and utilized, and
the degree of graft-versus-host disease. There is additional research going on now to
investigate the feasibility of inserting genes into cord blood stems cells and using them
for gene therapy. The possibilities have sparked increased interest into doctors and
scientists worldwide.
With this interest comes the need for both private family and public cord blood banks.
The National Institutes of Health (NIH) funded the first donor cord blood band and is
also funding two more in California. Fortunately, the number of insurance companies who
will pay for cord blood storage is increasing. The $1,500 for the initial service and $95
per year storage fee saves the insurer thousands of dollars compared to a bone marrow
harvest, which costs between $5,000 and $10,000, or an unrelated donor's bone marrow
which can cost up to $30,000.
According to Dr. Hale, the future holds great promise for the use of cord blood stem
cells. The results of these studies must be compared with the anticipated high mortality
rate without transplantation. The number of cord blood transplants is expected to
increase significantly in the near future and further research will continue to improve
knowledge and increase survival rates.
Diseases that are currently treatable with Stem Cells*:
Malignant Diseases
? Acute Lymphoblastic Leukemia
? Acute myeloblastic leukemia
? Chronic myelogenous leukemia
? Hodgkin's disease
? Multiple myeloma
? Non-Hodgkin's lymphoma
Nonmalignant Diseases
? Aplastic anemia
? Sickle cell anemia
? Osteopertrosis
? Globoid cell leukodystrophy
? Severe combined immunodeficiency
? Wiskott-Aldrich syndrome
? X-linked lymphoproliferative syndrome
? Hunter's syndrome
? Hurler's syndrome
? Lesch Nyhan syndrome
? Beta thalassemia
*Scientific American, January 1998
Bibliography
Gregory Hale, Ph.D.
Scientific American Magazine Online
January 1998
http://www.sciam.com/medicine/medicine29.html