Stem cells from the umbilical cord blood
What are umbilical cord blood stem cells? What are their features?
Stem cells are primary, non-specialised cells of the human body. They are distinguished by unlimited abilities to divide and develop into any type of cell. The human body is constantly in the process of apoptosis, which consists in the dying of cells. The rebuilding of tissues is possible thanks to stem cells. At first they are universal, and only in the course of further divisions (totipotent stem cells → pluripotent stem cells → multipotent stem cells → unipotent stem cells) they acquire specialisation, thus creating diverse types of cells – of the blood, bone, fat, muscle tissue, as well as cells constituting the so-called bone marrow stroma.
The exceptional properties of stem cells are a proof of their immense potential. Already today, umbilical cord blood stem cells are used in the treatment of many diseases. There are numerous clinical trials underway concerning the use of stem cells in the so-called regenerative medicine – in patients suffering from autism, with cerebral palsy, after strokes, injuries and burns. Doctors still do not know where the possibilities of stem cells end, which makes them an unquestionable hope for medicine..
Sources of stem cells
The easiest, and simultaneously the least invasive, method of obtaining stem cells is umbilical cord blood. The stem cells present in UCB, owing to their special characteristics, have a significant advantage over the cells coming from various different sources.
- bone marrow
- peripheral blood
- umbilical cord blood
- embryos
- other tissues, including: adipose tissue, nervous tissue, intestines
Advantage of umbilical cord blood stem cells
Why is it worth to collect stem cells from umbilical cord blood? Unlike stem cells coming from bone marrow, peripheral blood and adipose tissue, those present in the umbilical cord blood are collected in a non-invasive manner, after the delivery is completed and after umbilical cord separation. Contrary to popular belief, umbilical cord blood is not taken away from the baby. Should it not be collected, it would become medical waste. Collecting umbilical cord blood is a quick and painless procedure that does not require general or local anaesthesia. Stem cells obtained in such way are frozen in cryogenic tanks and immediately available, if needed. The volume of the frozen stem cell deposit is small, but it has a very large amount of stem cells. Umbilical cord blood stem cells most accurately resemble hematopoietic stem cells derived from bone marrow. However, they have a bigger proliferative potential, longer telomeres and a greater ability to produce certain cytokines. Umbilical cord blood stem cells can be used for both autologous (the recipient is the donor) and allogeneic (transplant from another person) transplantations. In the case of an allogeneic transplantation, it is imperative to properly choose both the donor and the recipient in terms of HLA (Human Leukocyte Antigen) antigen matching. Tissue incompatibility between the donor and the recipient may result in rejection of the transplanted cells or GvHD (graft-versus-host disease). The risk of such reactions is lower in the case of use of umbilical cord blood stem cells because umbilical cord blood lymphocytes are less mature and less immunocompetent. That is why in the case of allogenic transplantation of the umbilical cord stem cells, lower HLA matching is accepted, and thus finding a donor is easier.
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Diseases which are treated with stem cells
Acute and chronic leukemias
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Acute Myelogenous Leukemia (AML) | ||
| Acute Lymphoblastic Leukemia (ALL) | ||
| Chronic Lymphocytic Leukemia (CLL) | ||
| Juvenile Myelomonocytic Leukemia (JMML) | ||
| Juvenile Chronic Myelogenous Leukemia (JCML) | ||
| Chronic Myelogenous Leukemia (CML) |
Myelodysplastic syndromes
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Myelodysplastic syndrome | ||
| Chronic myelomonocytic leukemia (CMML) | ||
| Refractory anemia (RA) | ||
| Refractory anaemia with ring sideroblasts (RA-S) | ||
| Refractory anemia with excess of blasts (RAEB) |
Diseases caused by a stem cell defect
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Severe aplastic anemia | ||
| Fanconi anemia | ||
| Paroxysmal nocturnal hemoglobinuria (PNH) |
Other cancers
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Neuroblastoma | Ongoing studies | |
| Brain tumour | ||
| Retinoblastoma | ||
| Bone sarcoma (Ewing's sarcoma) | Ongoing studies | |
| Nephroblastoma (Wilms’ tumor) | Ongoing studies | |
| Germ cell tumour | ||
| Breast cancer | Ongoing studies | Ongoing studies |
| Ovarian cancer | Ongoing studies | Ongoing studies |
| Small cell lung cancer | Ongoing studies | |
| Kidney cancer | Ongoing studies | |
| Glioma – cancer of the central nervous system | Ongoing studies | |
| Ewing's sarcoma | ||
| Rheumatoid arthritis | Ongoing studies | |
| Multiple sclerosis | Ongoing studies | Ongoing studies |
Autoimmune diseases
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Juvenile rheumatoid arthritis | Ongoing studies | |
| Systemic lupus erythematosus | Ongoing studies | |
| Crohn's disease | Ongoing studies | |
| Evans syndrome | Ongoing studies |
Mucopolysaccharidoses
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Hurler syndrome (MPS-IH) | ||
| Maroteaux-Lamy syndrome (MPS-VI) | Ongoing studies |
Hereditary abnormalities of red blood cells
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Beta-thalassemia major | ||
| Sickle cell anemia | ||
| Diamond-Blackfan anemia | ||
| Pure red cell aplasia |
Hereditary disorders of the immune system
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Ataxia-telangiectasia | ||
| Bare lymphocyte syndrome | ||
| DiGeorge syndrome | ||
| Kostmann syndrome | ||
| Common variable immunodeficiency | ||
| Leukocyte adhesion deficiency | ||
| Hyper IgM syndrome | ||
| Omenn syndrome | ||
| Wiskott–Aldrich syndrome | ||
| X-linked lymphoproliferative (XLP) syndrome | ||
| Severe combined immunodeficiency (SCID) | ||
| SCID with the absence of T lymphocyte and B lymphocyte | ||
| SCID with the absence of T lymphocyte and normal number of B lymphocytes | ||
| SCID with adenosine deaminase deficiency |
Other hereditary diseases
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Cartilage-hair hypoplasia | ||
| Glanzmann’s thrombasthenia | ||
| Lesch-Nyhan syndrome | ||
| Osteopetrosis |
Lysosomal storage diseases
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Adrenoleukodystrophy | ||
| Gaucher disease | ||
| Krabbe disease | ||
| Niemann-Pick disease | ||
| Wolman disease | ||
| Metachromatic leukodystrophy | ||
| Mucopolysaccharidosis (MPS) | ||
| Hunter syndrome (MPS-II) | ||
| Morquio syndrome (MPS-IV) | ||
| Steal syndrome, beta-glucuronidase deficiency (MPS-VII) | ||
| Sanfilippo syndrome (MPS-III) | ||
| Scheie syndrome (MPS-IS) | ||
| Sandhoff disease | ||
| Pelizaeus-Merzbacher disease |
Histiocytoses
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Hemophagocytosis | ||
| Histiocytosis X | ||
| Familial hemophagocytic lymphohistiocytosis (FEL) |
Diseases treated with hematopoietic stem cell transplantation in a non-standard way
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Type 1 diabetes | Ongoing studies | |
| Cerebral palsy | Ongoing studies | |
| Paresis of the upper and lower limbs | Ongoing studies | |
| Anoxic brain damage | Ongoing studies | |
| Traumatic brain injury | Ongoing studies |
Lymphoproliferative disorders
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Hodgkin lymphoma | Depends on the level of advancement | |
| Malignant lymphomas | Depends on the level of advancement | Ongoing studies |
Plasma cell diseases
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Multiple myeloma | Ongoing studies |
Phagocyte disorders
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Reticular dysgenesis | ||
| Neutrophil actin deficiency | ||
| Chronic granulomatous disease | ||
| Chediak-Higashi syndrome |
Other diseases
| Name of disease | Autologous transplants (the donor is the recipient) |
Allogeneic transplantation (donor other than recipient) |
|---|---|---|
| Systemic scleroderma | Ongoing studies | Ongoing studies |
| Hearing loss | ||
| Autism spectrum disorders | ||
| Brain and spinal cord injuries | ||
| Developmental delay | ||
| Cerebellar ataxia | ||
| Muscular dystrophies | ||
| Ischemic strokes | ||
| Prolymphocytic leukemia | ||
| Non-Hodkin lymphomas | ||
| Shwachman-Diamond syndrome |
Stem cell
transplantation
Stem cells from the umbilical cord blood (UCB) can transform into blood and immune system elements. That is why they are used in bone marrow transplantations.
The first UCB stem cell transplantation was performed in France in 1988, in a 5-year-old boy with Fanconi anaemia. The cells were obtained from umbilical cord blood collected from the patient’s sister. The transplantation was successful, and the boy remains healthy and does not require blood transfusions. Since then, umbilical cord blood stem cell transplantations have been used in the treatment of numerous diseases of the hematopoietic system, e.g. cancers (leukaemias, lymphomas), diseases related to bone marrow failure (aplastic anaemia) or immune disorders.
Treatment using
stem cells from
umbilical cord blood
The umbilical cord blood stem cells can also be used in the treatment of diseases, such as type 1 diabetes or ischaemic stroke. It is worth noting that in the case of cancer, own umbilical cord blood stem cells cannot be used because they might be burdened with a genetic disorder which caused the malignancy to occur. The same applies to diseases with a known genetic background. In such cases, allogenic transplants are performed which require cells from another donor (e.g. from the patient’s siblings).
Recently intensive clinical trials were conducted on the use of umbilical cord blood stem cells in regenerative medicine, using patient’s own (autologous) cells. Most of the research concerns nervous system diseases, like autism, cerebral palsy, injuries of the brain and spinal cord. Umbilical cord blood stem cell transplantations can lead to inhibition of the pathological inflammatory process. Stem cells have an immunomodulatory effect, stunt apoptosis (cell death), induce migration, proliferation and cell differentiation, and also stimulate angiogenesis (creation of new blood vessels).
It should be noted that umbilical cord blood stem cell use in regenerative medicine is performed only in clinical trials and does not constitute standard treatment. Based on the current research, it seems a very auspicious direction in research.