Treated Diseases

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Acute Leukemias
Acute Biphenotypic Leukemia
Acute Lymphocytic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Acute Undifferentiated Leukemia

Chronic Leukemias
Chronic Lymphocytic Leukemia (CLL)
Chronic Myelogenous Leukemia (CML)
Juvenile Chronic Myelogenous Leukemia (JCML)
Juvenile Myelomonocytic Leukemia (JMML)

Myelodysplastic Syndromes
Amyloidosis
Chronic Myelomonocytic Leukemia (CMML)
Refractory Anemia (RA)
Refractory Anemia with Excess Blasts (RAEB)
Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
Refractory Anemia with Ringed Sideroblasts (RARS)

Stem Cell Disorders
Aplastic Anemia (Severe)
Congenital Cytopenia
Dyskeratosis Congenita
Fanconi Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Myeloproliferative Disorders
Acute Myelofibrosis
Agnogenic Myeloid Metaplasia (Myelofibrosis)
Essential Thrombocythemia
Polycythemia Vera

Lymphoproliferative Disorders
Hodgkin’s Disease
Non-Hodgkin’s Lymphoma
Prolymphocytic Leukemia

Phagocyte Disorders
Chediak-Higashi Syndrome
Chronic Granulomatous Disease
Neutrophil Actin Deficiency
Reticular Dysgenesis

Liposomal Storage Diseases
Adrenoleukodystrophy
Gaucher’s Disease
Hunter’s Syndrome (MPS-II)
Hurler’s Syndrome (MPS-IH)
Krabbe Disease
Maroteaux-Lamy Syndrome (MPS-VI)
Metachromatic Leukodystrophy
Morquio Syndrome (MPS-IV)
Mucolipidosis II (I-cell Disease)
Mucopolysaccharidoses (MPS)
Niemann-Pick Disease
Sanfilippo Syndrome (MPS-III)
Scheie Syndrome (MPS-IS)
Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)
Wolman Disease

Histiocytic Disorders
Familial Erythrophagocytic Lymphohistiocytosis
Hemophagocytosis
Histiocytosis-X
Langerhans’ Cell Histiocytosis

Inherited Erythrocyte Abnormalities
Beta Thalassemia Major
Blackfan-Diamond Anemia
Pure Red Cell Aplasia
Sickle Cell Disease

Congenital (Inherited) Immune System Disorders
Absence of T & B Cells SCID
Absence of T Cells, Normal B Cell SCID
Ataxia-Telangiectasia
Bare Lymphocyte Syndrome
Common Variable Immunodeficiency
DiGeorge Syndrome
Kostmann Syndrome
Leukocyte Adhesion Deficiency
Omenn’s Syndrome
Severe Combined Immunodeficiency (SCID)
SCID with Adenosine Deaminase Deficiency
Wiskott-Aldrich Syndrome
X-Linked Lymphoproliferative Disorder

Other Inherited Disorders
Cartilage-Hair Hypoplasia
Ceroid Lipofuscinosis
Congenital Erythropoietic Porphyria
Glanzmann Thrombasthenia
Lesch-Nyhan Syndrome
Osteopetrosis
Tay Sachs Disease

Inherited Platelet Abnormalities
Amegakaryocytosis / Congenital Thrombocytopenia

Plasma Cell Disorders
Multiple Myeloma
Plasma Cell Leukemia
Waldenstrom’s Macroglobulinemia

Other Malignancies
Brain Tumors
Ewing Sarcoma
Neuroblastoma
Ovarian Cancer
Renal Cell Carcinoma
Small-Cell Lung Cancer
Testicular Cancer

Autoimmune Diseases
Evan Syndrome
Multiple Sclerosis (Experimental)
Rheumatoid Arthritis (Experimental)
Systemic Lupus Erythematosus (Experimental)

Emerging Clinical Stem Cell Applications

Alzheimer’s Disease

Parkinson’s Disease

Stroke
Doctors are focusing on the ability of the stem cell to differentiate into nerve cells. The approach to treatment would be to regenerate new and healthy nerve cells (tissue) to reduce, alleviate, or eliminate the symptoms or effects of diseases like Alzheimer’s and Parkinson’s and repair damage caused by stroke.

      Sanchez-Ramos, J., Song, S., Kamath, S., et al. Expression of neural markers in human umbilical cord blood. Experimental Neurology. 2001;171:109-115.
    Chen, J., Sanberg, P., Li, Y., et al. Intravenous administration of human umbilical cord blood reduces behavioral deficits after stroke in rats. Stroke. 2001;32:2682-2688.

Diabetes
Success using stem cells to regenerate pancreas function and insulin production. May help insulin dependent patients become free of adjunctive therapy (such as insulin injections).

    Domenick, M., Ildstad, S. Impact of bone marrow transplantation on type I diabetes. World Journal of Surgery. 2001;25:474-480.

Heart Disease
Success regenerating heart tissue and blood vessels for treatment of heart disease or traumatic injury to the heart.

    Assmus, B., Schachinger, V., Teupe, C., et al. Transplantation of progenitor cells and regeneration enhancement in acute myocardial infarction (TOPCARE-AMI). Circulation. 2002;106:3009-3017.

Liver Disease
Stem cells differentiated into liver cells in mice. Future applications may include repairing liver damage caused by cirrhosis, viral infection, trauma, chemotherapy, or radiation therapy.

    Wang X., Ge S., McNamara G., et al. Albumin-expressing hepatocyte-like cells develop in the livers of immune-deficient mice that received transplants of highly purified human hematopoietic stem cells. Blood. 2003;101(10):4201-4208.

Lupus
Following stem cell transplantation patients with severe lupus have been able to regenerate blood cells that are completely free of the disease.

    Brunner, M., Greinix, H., Redlich, K., et al. Autologous blood stem cell transplantation in refractory systemic lupus erythematosus with severe pulmonary impairment. Arthritis & Rheumatism. 2002;46(6):1580-1584.

Multiple Sclerosis
Stem cell transplantation has stopped and even reversed disease progression for some patients with multiple sclerosis.

    Kozak, T. Hematopoietic stem cell transplantation in the treatment of autoimmune diseases. The Middle European Journal of Medicine. 2002;114(1-2; 7-13)

Muscular Dystrophy
Stem cells have been proven to fuse into skeletal muscle cells in patients with muscular dystrophy. Transplantation may help slow or stop the progression of muscle wasting.

    Gussoni, E., Bennett, R., Muskiewicz, K., et al. Long term persistence of donor nuclei in a duchenne muscular dystrophy patient receiving bone marrow transplantation, The Journal of Clinical Investigation, 2002;110(6):807-814.

Spinal Cord Injury
In animal studies cord blood stem cells are beneficial in helping to reverse paralysis caused by spinal cord injury. Cord blood-derived stem cells were shown to migrate to and participate in the healing of neurological defects caused by traumatic assault.

    Saporta, S., Kim, J., Willing, A., et al. Human umbilical cord blood stem cells infusion in spinal cord injury: engraftment and beneficial influence on behavior. Journal of Hematotherapy & Stem Cell Research. 2003; 12:271-278.