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Diseases Treated with Cord Blood Stem Cells

How Can Stem Cells Be Used To Treat Diseases?

Parents should consider their cord blood banking options while pregnant

Stem cell transplants are currently the standard therapy for patients with bone marrow disease including:

Cancers of the bone marrow

  • Such as leukemia and lymphoma

Inherited Diseases

  • Sickle cell anemiaCharacterized by abnormal hemoglobin that create ‘C-shaped’ cells which move slowly and cause blocked blood vessels; common among African Americans*
  • ThalessemiaCharacterized by reduced or absent amounts of hemoglobin; common among those of Mediterranean ancestry*
  • Krabbe;s DiseaseCharacterized by the abnormal presence of globoid cells, which are globe-shaped cells that usually have more than one nucleus*
  • Hurler’s SyndromeThis is a lysosomal storage easesease which leads to progressive mental deterioration and loss of physical skills*
  • Aplastic anemiaBone marrow does not produce sufficient new cells to replenish blood cells*
  • Acquired bone marrow failure
    As a result of chemotherapy and/or radiation treatments used in cancer treatment. Stem cell transplants act as:
    A replacement therapy:

    • The sick individual’s malignant bone marrow is replaced by the cells provided by a healthy donor.*
    • Sometimes during cancer treatment, the bone marrow is destroyed by radiation or chemotherapy. In this case, a cord blood transplant is required to introduce new stem cells into a person’s body. These new cells will reconstitute the bone marrow.
      A form of immunotherapy: In this case, the donor’s immune system is present in the cord blood sample. Those cells act against the sick recipient’s remaining cancer cells to kill them. This phenomenon is called graft-versus-leukemia effect.*

Allogeneic transplants use stem cells from a donor. The donor can be a sibling, family member or a donor sample from a public bank.

Diseases that Can be Treated by Allogeneic Transplantation:


  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myelogenous Leukemia (AML)
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia
  • Chronic Myelogenous Leukemia (CML)
  • Chronic Lymphocytic Leukemia (CLL)
  • Juvenile Chronic Myelogenous Leukemia (JCML)
  • Juvenile Myelomonocytic Leukemia (JMML)
  • 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)
  • Hodgkin’s Lymphoma
  • Non-Hodgkin’s Lymphoma (Burkitt’s Lymphoma)

Blood Cell Proliferation Disorders

  • Aplastic Anemia
  • Congenital Dyserythropoietic Anemia
  • Fanconi Anemia
  • Paroxysmal Nocturnal – Hemoglobinuria (PNH)
  • Beta Thalassemia Major (Cooley’s Anemia)
  • Diamond-Blackfan Anemia
  • Pure Red Cell Aplasia
  • Sickle Cell Disease
  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Glanzmann Thrombasthenia


  • 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
  • Myelokathexis
  • Ataxia-Telangiectasia
  • Bare Lymphocyte Syndrome
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Hemophagocytic Lymphohisticytosis
  • Leukocyte Adhesion Deficiency
  • Lymphoproliferative Disorders (LPD)
  • Lymphoproliferative Disorder (also known as Epstein-Barr Virus Sesceptibility)
  • Wiskott-Aldrich Syndrome
  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (Myelofibrosis)
  • Polycythemia Vera
  • Essential Thrombocythemia
  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Neutrophil Actin Deficiency
  • Reticular Dysgenesis
  • Multiple Myeloma
  • Plasma Cell Leukemia
  • Waldenstrom’s Macroglobulinemia

Inherited Disorders

  • Cartilage-Hair Hypoplasia
  • Erythropoietic Porphyria
  • Hermansky-Pudlak Syndrome
  • Adrenoleukodystrophy (ALD)
  • Krabbe Disease (Globoid Cell Leukodystrophy)
  • Metachromatic Leukodystrophy
  • Pearson’s Syndrome
  • Shwachman-Diamond Syndrome
  • Pelizaeus-Merzbacher Disease
  • Systemic Mastocytosis
  • Niemann-Pick Disease
  • Sandhoff Disease
  • Wolman Disease
  • Lesch-Nyhan Syndrome
  • Osteopetrosis
  • Hurler Syndrome (MPS-IH)
  • Scheie Syndrome (MPS-IS)
  • Hunter Syndrome (MPS-II)
  • Sanfilippo Syndrome (MPS-III)
  • Morquio Syndrome (MPS-IV)
  • Maroteaux-Lamy Syndrome (MPS-VI)
  • Sly Syndrome, Beta-Glucuronidase Deficiency
  • Mucolipidosis II (I-cell Disease)

Diseases in Clinical Trial

  • Amyotrophic Lateral Sclerosis (ALS or “Lou Gehrig’s disease)
  • Crohn’s Disease
  • Diabetes, Type I (formerly Juvenile Diabetes)
  • Graft-versus-Host Disease (GvHD)
  • Kidney Plus Stem Cell Transplant
  • Lupus
  • Rheumatoid Arthritis
  • Scleroderma
  • Critical Limb Ischemia
  • Ischemic Stroke
  • Cerebral Palsy (CP)
  • Spinal Cord Injury (SCI)
  • Cleft Palate Repair (alveolar)
  • Cartilage Repair
  • Epidermolysis Bullosa
  • Lysosomal Storage Disease

Autologous treatments use a person’s own stem cells.

Diseases that Can be Treated by Autologous Transplantation:

Diseases Treated

  • Neuroblastoma
  • Medulloblastoma
  • Retinoblastoma

Diseases in Clinical Trial*

  • Autism
  • Cerebral Palsy
  • Hearing Loss (acquired sensorineural)
  • Hypoxic Ischemic Encephalopathy (HIE)
  • Amyotrophic Lateral Sclerosis (ALS)
  • Crohn’s Disease
  • Diabetes, Type 1
  • Lupus
  • Multiple Sclerosis
  • Scleroderma
  • Support for Misc. Open Cardiac Surgery
  • Hypoplastic Left Heart Syndrome (HLHS)
  • Grow Vascular Graft
  • Critical Limb Ischemia
  • Compartment Syndrome (Battlefield Trauma)
  • Ischemic Stroke
  • Ischemic Heart Disease
  • Myocardial Infarction
  • Cardiomyopathy
  • Alveolar Cleft Palate Repair
  • Knee Cartilage Repair

* Source Parentsguidetocordblood.org

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Toll Free: 1.877.235.1997

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