Diseases Treated By Cord Blood Stem Cells
Stem Cells and Cancer
Acute Lymphocytic leukemia (ALL): This type of leukemia is characterized by the rapid production of defective white blood cells. The large amount of the white blood cells blocks the production healthy red and white blood cells as well as platelets.
Acute Myelogenous leukemia (AML): This cancer is marked by the abundance of immature white blood cells which quickly replace other white blood cells in the bone marrow.
Chronic Myelocytic leukemia (CML): This cancer differs from ALL and AML in that it affects mature white blood cells. These cancerous cells rapidly increase and affect the bone marrow. They may also enter the blood stream.
Myelodysplastic syndrome (MDS): People with MDS usually have a shortage of blood caused by their body�s inability to effectively produce blood cells. Those blood cells that are formed are often immature and therefore defective. MDS may progress into an acute leukemia.
Liposarcoma: Cancer of the fat cells that affects soft tissue.
Neuroblastoma: A type of cancer made up of a solid tumor that originates in the nerve tissue of the neck, chest, pelvis or, most commonly, in the adrenal gland tissue found in the abdomen.
Non-Hodgkin�s lymphoma: Cancer of the lymphatic system. Unlike Hodgkin�s disease, Non-Hodgkin�s lymphoma tends to be unpredictable and is much more likely to spread to other areas of the body.
Yolk Sac sarcoma: A type of cancer that usually originates in the testicles before spreading to other areas of the body.
Blood Disorders
Amegakaryocytic thrombocytopenia (AMT): A blood disorder that causes a marked decrease in the production of platelets. This leads to frequent bruising and problems clotting when bleeding.
Aplastic anemia: A type of blood disorder caused by the body�s inability to produce enough blood cells. The lack of blood cells causes sufferers to have a lowered immune system and troubles clotting.
Diamond-Blackfan anemia: Unlike Aplastic anemia, Diamond-Blackfan anemia sufferers have troubles producing only red blood cells. Those affected by Diamond-Blackfan anemia are also likely to have physical deformities, most notably malformed thumbs. They may also be short in stature.
Congenital cytopenia: A hereditary deficiency of the blood cells.
Evan�s syndrome: An autoimmune disorder where the body produces antibodies to attack red and white blood cells along with platelets.
Fanconi�s anemia: A genetic disorder that is marked by the body�s lack of essential bone marrow material including red and white blood cells and platelets. People who suffer from this disorder may have an abnormal heart, kidney and/or skeletal structure. They may also have brown skin discoloration on some parts of their body.
Kostmann�s syndrome: A genetic disorder wherein sufferers have a deficiency of neutrophils, a particular kind of white blood cells. The lack of neutrophils makes it more difficult for sufferers to fight bacterial infections.
Sickle cell anemia: A genetic disease wherein sufferers have misshapen red blood cells. The cells therefore do not work properly and cause small blood clots.
Thalassemia: A genetic blood disorder that is marked by the body�s inability to properly produce hemoglobin. As a result, the red blood cells are under produced yet also destroyed too frequently.
Inherited Metabolic Disorders
Adrenoleukodystrophy: A genetic disorder that is characterized by the body�s inability to breakdown long chain fatty acids due to a lack of a particular enzyme. This disorder can affect the adrenal glands, nervous system and testes. There are seven recognized forms of this disease.
Bare-lymphocyte syndrome: A rare disorder affecting the immune system. People with this disorder are especially vulnerable to viral and bacterial infections, which can cause them to suffer from chronic diarrhea and stunted growth.
Dyskeratosis congenital: A rare disease whereby those affected tend to have premature aging and are more vulnerable to developing cancer due to their bone marrow failure.
Familial erythrophagocytic lymphohistiocytosis: A rare genetic disorder, it is characterized by an over active immune system. T-cells will attack the liver, spleen, bone marrow and central nervous system causing multiple problems in a person.
Gaucher disease: A genetic disorder caused by a person�s lack of the glucocerebrosidase enzyme. This results in toxic fatty materials building up in the liver, spleen and bone marrow.
Gunter disease: Also known as congenital erythropoietic, this genetic disease causes a sufferers skin to be extremely sensitive to sunlight. Exposure to sunlight can result in blistering (with a long healing time), scarring and skin pigmentation changes among other things.
Hunter syndrome: A hereditary disease that results in the body�s inability to properly breakdown a specific chemical. This causes the chemical to build up in different body tissues whereby it can cause damage and inhibit proper organ function.
Hurler syndrome: A genetic disease that is caused by the body�s inability to produce a particular enzyme (lyposomal alpha-L-iduronate) necessary for the breakdown of certain chemicals. This causes the chemicals to build up and affect the internal organs as well as mental development.
Inherited neuronal ceroid lipofuscinosis: A genetic disease that is marked by a build of an abnormal pigment (lipofuscin) in the brain. It is thought to be caused by the brain cells inability to remove or reuse brain proteins.
Krabbe disease: A rare disorder which affects the nervous system. It is caused by the lack of a crucial enzyme required for the proper development of the myelin sheath (a protective coating around the nerve fibers in the brain composed of a fatty covering).
Lanegerhans�-cell histiocytosis: This disorder occurs when there is an abnormal increase in the quantity of particular immune cells. The excessive amount of immune cells can form tumors in different bones possibly resulting in fractures. It can also cause problems with a person�s immune system resulting in rashes, gum problems, and lung problems among other things.
Lesch-Nyhan Disease: A genetic disorder that directly affects the body�s ability to produce and breakdown purine, a chemical that makes up RNA and DNA molecules. It is marked by an increase in blood and uric acid levels in addition to the absence of a particular enzyme (hypoxanthine guanine phosphoribosyltransferase).
Leukocyte adhesion deficiency: A rare genetic disorder that stems from the bodies inability to effectively produce a particular protein, CD18. The lack of CD18 protein inhibits white blood cells from traveling to parts of the body that are infected or injured, leaving a person more susceptible to sicknesses and prolonged healing times.
Osteopetrosis: A rare genetic disorder that causes the density of bones to increase. This can inhibit bone growth; make the bones weaker and more susceptible to breakage; and can also crowd out bone marrow.
Immunodeficiencies
Adenosine deaminase deficiency (ADA or SCID-ADA): A rare genetic disorder that is caused by the body�s inability to adequately produce the adenosine deaminase enzyme, which is responsible for proper functioning of the immune system. The lack of the enzyme seriously compromises the immune system and may cause those affected by it to live in isolation.
Severe combined immunodeficiency (SCID): A genetic disorder that is caused by the body�s inability to efficiently produce T- and B-lymphocytes. This makes people unable to effectively fight off infections and may cause them to live in isolation. SCID is sometimes also known as �Bubble Boy syndrome� in reference to a boy who was affected by SCID and lived in a germ-free plastic bubble for 12 years.
Wiskott-Aldrich syndrome: A hereditary disorder that is marked by defects in the immune systems production of T- and B-lymphocytes as well as platelets. People affected by this disorder tend to be more susceptible to infections, especially those that affect the respiratory tract, as well as have bleeding problems. There is also an increased chance of developing certain cancers.
X-Linked lymphoproliferative disease (XLP): A rare hereditary disorder that is marked by a person�s inability to adequately develop the appropriate antibodies needed to fight off the Epstein-Barr virus (a common herpes virus). XLP sufferers are more susceptible to infections and some forms of cancer as well as possible deficiencies in blood production, low levels of antibodies in their blood and aplastic anemia.
Hyper-IgM immunodeficiency (HIM): This rare disorder is marked by the body�s normal to increased production of poor quality IgM antibody, which is found on the B cell of white blood cells. This hyper production of poor quality cells interrupts the development of other important antibodies. Those afflicted by HIM are vulnerable to bacterial infections as well as autoimmune disorders and cancers.
This is not an exhaustive list. New ways of using cord blood stem cells to treat diseases and disorders are constantly being developed.
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