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Treating Diseases with Stem Cell Transplant


What stem cells can do today opens doorways to even more, tomorrow…

Cord blood stem cell transplants have already changed and saved thousands of lives around the world. Science is developing other miraculous uses for these precious cells, potentially impacting countless numbers of lives in the future.

How are cord blood stem cells saving lives now?


Cord blood stem cells have been used to treat nearly 80 diseases, including numerous types of malignancies, anemias, inherited metabolic disorders and deficiencies of the immune system. The majority of cord blood transplants to date have been performed in patients younger than 18 years old. However, with the advancement in regenerative medicine, it is foreseeable that individuals of all ages can benefit from stem cell therapy in the near future. The source of cord blood used in transplants can be autologous (self) or allogeneic (such as a sibling or an unrelated third party).  

Graft-versus-host disease, a complication associated with stem cell transplant therapy, occurs less frequently with umbilical cord stem cells vs. other types of stem cells; and, it is even rarer when the cord stem cells come from a blood related family member.

Below are some diseases currently being treated with stem cells. Although many cord blood stem cell treatments today are allogeneic (non-self), leading scientists believe that autologous (self) cord blood will have a role in treating Type I diabetes, other autoimmune diseases, and brain and cardiac injuries.

Leukemias
Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells (all therapies are allogeneic)

  • Acute Leukemia
    • Acute Lymphoblastic Leukemia (ALL)
    • Acute Myelogenous Leukemia (AML)
    • Acute Biphenotypic Leukemia
    • Acute Undifferentiated Leukemia
       
  • Chronic Leukemia
    • Chronic Myelogenous Leukemia (CML)
    • Chronic Lymphocytic Leukemia (CLL)
    • Juvenile Chronic Myelogenous Leukemia (JCML)
    • Juvenile Myelomonocytic Leukemia (JMML)
       
  • Myelodysplastic Syndromes (sometimes called pre-leukemia)
    • Refractory Anemia (RA)
    • Refractory Anemia with Ringed Sideroblasts (RARS)
    • Refractory Anemia with Excess Blasts (RAEB)
    • Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
    • Chronic Myelomonocytic Leukemia (CMML)

Lymphomas
Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels (all therapies are allogeneic)
  • Hodgkin’s Lymphoma
  • Non-Hodgkin’s Lymphoma (Burkitt’s Lymphoma)

Other Disorders of Blood Cell Proliferation (all therapies are allogeneic)
  • Anemias - Anemias are deficiencies or malformations of red cells (all therapies are allogeneic)
    • Aplastic Anemia
    • Congenital Dyserythropoietic Anemia
    • Fanconi Anemia (Note: the first cord blood transplant in 1988 was for FA, an inherited disorder)
    • Paroxysmal Nocturnal Hemoglobinuria (PNH)
    • Pure Red Cell Aplasia
       
  • Inherited Red Cell Abnormalities - Red cells contain hemoglobin and carry oxygen to the body
    • Beta Thalassemia Major (also known as Cooley’s Anemia)
    • Blackfan-Diamond Anemia
    • Pure Red Cell Aplasia
    • Sickle Cell Disease
       
  • Inherited Platelet Abnormalities - Platelets are blood cells needed for clotting
    • Amegakaryocytosis / Congenital Thrombocytopenia
    • Glanzmann Thrombasthenia
       
  • Inherited Immune System Disorders - Severe Combined Immunodeficiency (SCID)
    • SCID with Adenosine Deaminase Deficiency (ADA-SCID)
    • SCID which is X-linked
    • SCID with absence of T & B Cells
    • SCID with absence of T Cells, Normal B Cells
    • Omenn Syndrome
       
  • Inherited Immune System Disorders - Neutropenias
    • Kostmann Syndrome
    • Myelokathexis
       
  • Inherited Immune System Disorders - Other
    • Ataxia-Telangiectasia
    • Bare Lymphocyte Syndrome
    • Common Variable Immunodeficiency
    • DiGeorge Syndrome
    • Leukocyte Adhesion Deficiency
    • Lymphoproliferative Disorders (LPD)
    • Lymphoproliferative Disorder, X-linked (also known as Epstein-Barr Virus Susceptibility)
    • Wiskott-Aldrich Syndrome
       
  • Myeloproliferative Disorders
    • Acute Myelofibrosis
    • Agnogenic Myeloid Metaplasia (Myelofibrosis)
    • Polycythemia Vera
    • Essential Thrombocythemia
       
  • Phagocyte Disorders - Phagocytes are immune system cells that can engulf and kill foreign organisms
    • Chediak-Higashi Syndrome
    • Chronic Granulomatous Disease
    • Neutrophil Actin Deficiency
    • Reticular Dysgenesis
       
  • Bone Marrow Cancers (Plasma Cell Disorders)
    • Multiple Myeloma
    • Plasma Cell Leukemia
    • Waldenstrom’s Macroglobulinemia

Transplants for Inherited Disorders affecting the Immune System & Other Organs (all therapies are allogeneic)
  • Cartilage-Hair Hypoplasia
  • Gunther’s Disease (Erythropoietic Porphyria)
  • Hermansky-Pudlak Syndrome
  • Pearson’s Syndrome
  • Shwachman-Diamond Syndrome
  • Systemic Mastocytosis

Transplants for Inherited Metabolic Disorders (all therapies are allogeneic)
  • Mucopolysaccharidoses (MPS) Storage Diseases
    • Mucopolysaccharidoses (MPS)
    • Hurler’s Syndrome (MPS-IH)
    • Scheie Syndrome (MPS-IS)
    • Hunter’s Syndrome (MPS-II)
    • Sanfilippo Syndrome (MPS-III)
    • Morquio Syndrome (MPS-IV)
    • Maroteaux-Lamy Syndrome (MPS-VI)
    • Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)
    • Mucolipidosis II (I-cell Disease)
       
  • Leukodystrophy Disorders
    • Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)
    • Krabbe Disease (Globoid Cell Leukodystrophy)
    • Metachromatic Leukodystrophy
    • Pelizaeus-Merzbacher Disease
       
  • Lysosomal Storage Diseases
    • Gaucher Disease
    • Niemann-Pick Disease
    • Sandhoff Disease
    • Tay-Sachs Disease
    • Wolman Disease
       
  • Inherited Metabolic Disorders - Other
    • Lesch-Nyhan Syndrome
    • Osteopetrosis
       
Solid tumors not originating in the blood or immune system (Autologous therapy)
  • Neuroblastoma
  • Retinoblastoma
Request more information about treating diseases, today!

Autologous stem cells may not be useful in the treatment for certain diseases listed above - www.parentsguidecordblood.org/diseases.php

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