2012.06.26 - The current uses of stem cells › ... › Baby › baby-stem-cells-uses.pdfMesenchymal...

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Mesenchymal Stem Cells - Contained in the umbilical cord tissue. Haematopoietic Stem Cells - Contained in the umbilical cord blood. UC-MAR-INF-008 2012-06-01 THE CURRENT USES OF STEM CELLS They are: There are two types of stem cells that can be retrieved from the Umbilical Cord Blood and Cord Tissue at birth. HAEMATOPOIETIC STEM CELLS Anaemia (Anaemia's are deficiencies or malformations of red blood cells that are inherited or acquired diseases) Ÿ Severe Aplastic Anemia Ÿ Sickle cell anemia Ÿ Thalassemia Ÿ Fanconi’s Anemia (Note: the first cord blood transplant in 1988 was for this disease) Ÿ Paroxysmal Nocturnal Hemoglobinuria (PNH) Ÿ Pancytopenia Inherited Platelet Abnormalities (Platelets are small blood cells needed for clotting) Ÿ Amegakaryocytosis / Congenital Thrombocytopenia Myeloproliferative Disorders Ÿ Acute Myelofibrosis Ÿ Polycythemia Vera Ÿ Essential Thrombocythemia Inherited Immune System Disorders Ÿ Severe Combined Immunodeficiency (SCID) Metabolic Disorders Ÿ Adrenoleukodystrophy Ÿ Gaucher's disease Ÿ Krabbe disease (globoid cell leakodystrophy) Ÿ Gunther disease Ÿ Hurler syndrome Ÿ Hunter syndrome Ÿ Neimann-Pick Syndrome Ÿ Tay-Sachs disease Cancers in the bone marrow (Plasma Cell disorders) Ÿ Multiple Myeloma Ÿ Waldenstrom's Macroglobulinemia Other cancers (Not originating in the bone marrow or blood system) Ÿ Neuroblastoma Ÿ Retinoblastoma Ÿ Nephroblastoma 1 Umbilical cord blood is rich in haematopoietic stem cells. “Haematopoietic” means “blood forming” and these stem cells are destined (“programmed”) to differentiate into the 3 types of blood cells: red blood cells, white blood cells and platelets. In our bodies, bone marrow is the source of all blood cells. Haematopoietic stem cells are contained in the bone marrow and they continuously make new blood cells to replace old ones. If bone marrow is damaged by disease or drugs, it cannot make these essential blood cells, leading to fatal consequences. Therefore, haematopoietic stem cells must be replaced as part of the treatment. This is done via whole bone marrow transplant or stem cell transplant: . The below lists diseases for which haematopoietic stem cells are a standard treatment. For some diseases they are the only therapy and for others, they are employed when front-line therapies have failed or the disease is very aggressive: Leukemia – Acute and Chronic (Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white blood cells) Myelodysplastic Syndromes (Myelodysplasia is sometimes called pre-leukemia) Lymphomas (Lymphoma is a cancer of the white blood cells that circulate in the blood and lymph vessels)

Transcript of 2012.06.26 - The current uses of stem cells › ... › Baby › baby-stem-cells-uses.pdfMesenchymal...

Page 1: 2012.06.26 - The current uses of stem cells › ... › Baby › baby-stem-cells-uses.pdfMesenchymal Stem Cells - Contained in the umbilical cord tissue. Haematopoietic Stem Cells

Mesenchymal Stem Cells - Contained in the umbilical cord tissue.

Haematopoietic Stem Cells - Contained in the umbilical cord blood.

UC-MAR-INF-0082012-06-01THE CURRENT USES OF STEM CELLS

They are:T here are two types of stem cells that can be retrieved from the Umbilical Cord Blood and Cord Tissue at birth.

HAEMATOPOIETIC STEM CELLS

Anaemia (Anaemia's are deficiencies or malformations of red blood cells that are inherited or acquired diseases)Ÿ Severe Aplastic AnemiaŸ Sickle cell anemiaŸ ThalassemiaŸ Fanconi’s Anemia (Note: the first cord blood

transplant in 1988 was for this disease)Ÿ Paroxysmal Nocturnal Hemoglobinuria (PNH)Ÿ Pancytopenia

Inherited Platelet Abnormalities (Platelets are small blood cells needed for clotting)Ÿ Amegakaryocytosis / Congenital Thrombocytopenia

Myeloproliferative DisordersŸ Acute Myelofibrosis Ÿ Polycythemia Vera Ÿ Essential Thrombocythemia

Inherited Immune System DisordersŸ Severe Combined Immunodeficiency (SCID)

Metabolic DisordersŸ AdrenoleukodystrophyŸ Gaucher's diseaseŸ Krabbe disease (globoid cell leakodystrophy)Ÿ Gunther diseaseŸ Hurler syndromeŸ Hunter syndromeŸ Neimann-Pick SyndromeŸ Tay-Sachs disease

Cancers in the bone marrow (Plasma Cell disorders)Ÿ Multiple Myeloma Ÿ Waldenstrom's Macroglobulinemia

Other cancers (Not originating in the bone marrow or blood system)Ÿ Neuroblastoma Ÿ RetinoblastomaŸ Nephroblastoma

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Umbilical cord blood is rich in haematopoietic stem cells. “Haematopoietic” means “blood forming” and these stem cells are destined (“programmed”) to differentiate into the 3 types of blood cells: red blood cells, white blood cells and platelets. In our bodies, bone marrow is the source of all blood cells. Haematopoietic stem cells are contained in the bone marrow and they continuously make new blood cells to replace old ones. If bone marrow is damaged by disease or drugs, it cannot make these essential blood cells, leading to fatal consequences. Therefore, haematopoietic stem cells must be replaced as part of the treatment. This is done via whole bone marrow transplant or stem cell transplant: .

The below lists diseases for which haematopoietic stem cells are a standard treatment. For some diseases they are the only therapy and for others, they are employed when front-line therapies have failed or the disease is very aggressive:

Leukemia – Acute and Chronic (Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white blood cells)

Myelodysplastic Syndromes (Myelodysplasia is sometimes called pre-leukemia)

Lymphomas (Lymphoma is a cancer of the white blood cells that circulate in the blood and lymph vessels)

Page 2: 2012.06.26 - The current uses of stem cells › ... › Baby › baby-stem-cells-uses.pdfMesenchymal Stem Cells - Contained in the umbilical cord tissue. Haematopoietic Stem Cells

UC-MAR-INF-0082012-06-01

THE CURRENT USES OF STEM CELLS

MESENCHYMAL STEM CELLS

TRANSPLANTATION OF STEM CELLS

[email protected]

0861 NETCELLS

STORAGE OF STEM CELLS

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Mesenchymal stem cells are found in abundance in umbilical cord tissue, although a small amount is found in the cord blood as well. These cells form the various CONNECTIVE tissues of the body: bone, cartilage, tendons, muscle and nerves. They are found in nearly all the organs of the body but are difficult to harvest from most sites. Apart from the umbilical cord tissue, mesenchymal stem cells can be harvested from the bone marrow and adult adipose (fat tissue).These cells can be easily multiplied and stored for future use. Their applications are far reaching and are currently being researched, tested and used in experimental clinical applications for:Ÿ Wound healingŸ Skin regenerationŸ Cartilage and bone repairŸ Heart muscle repairŸ Nerve repair

Allogeneic transplantation This type of transplant is a transplant between two genetically non-identical individuals. These individuals may be related family members or completely unrelated. Allogeneic transplants (allograft) may be used in the treatment of inherited and non- inherited disorders. Before doing an allograft, the recipient needs to find a matching donor.

Stem cells can be retrieved from 3 types of donors:Ÿ The patient themselves (autologous transplant)Ÿ A related donor i.e. a family member Ÿ An unrelated donor (allogeneic transplant)

Autologous transplantation Autologous transplantation means that the donor and the recipient is the same person. This type of transplantation refers to the situation where the child's own stem cells are used for themselves.It needs to be noted that autologous transplantation cannot be used in the treatment of inherited disorders. The reason being that the genes that code for the inherited disorder are carried in every cell i.e. they will also be found in the person's stem cells. So, we cannot return cells containing genes for a disorder back into the person after we have treated them for the disorder. In that case, an allogeneic donor will be used.

HLA matching necessary for transplantsPrior to allogeneic stem cell transplantation, HLA – type matching needs to take place. Human leukocyte antigens (HLA) are proteins on the surface of cells that determines the tissue type of a person. The immune system uses the HLA's to differentiate its own cells from that of other (non-self) cells. To successfully receive tissue from another person, the tissue type has to be the same otherwise the recipients system will see it as foreign and it will reject the donated tissue. A major advantage of cord blood and cord tissue is that it does not have to be a 100% match for transplant, opening the door to many more useable donors. HLA typing is done by analysing a sample of blood from the donor and one from the recipient.

If you can get Haematopoietic stem cells and Mesenchymal stem cells from adults, why store at birth?

The stem cells that are collected from a new born baby are at the beginning stages of their life span.Stem cells start showing age related changes as they get older: they start to divide slower and their reparative function starts to decrease. These changes are precipitated by the shortening of the telomeres (strands) of the chromosomes with each division of the cells. Therefore, umbilical cord stem cells have a “youthful” phenotype that is able to be expanded easily and retain the properties of stem cells. It is postulated that better treatment results are achieved with stem cells collected at birth. Additionally, stem cells are much easier to collect at birth (painless and more cost effective).

Why should you store both Cord Blood and Cord Tissue Stem Cells?

Ÿ Cord blood and cord tissue are used for different things and they are not interchangeable.

Ÿ Cord blood is needed for bone marrow transplants.Ÿ Cord tissue regenerates connective tissue. Although

there are no standard clinical applications at the moment, they have the potential to be extremely useful.