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Treatment Possibilities


Transplantation of hematopoietic stem cells (found in bone marrow, cord blood, or embryos in their first days of existence) is already approved as standard therapy for several diseases. For some, transplantation is the only treatment; for others, stem cells are used in especially aggressive disease processes or when traditional medicine has reached its limits.

In other diseases, stem cell transplants would lead to improvements in the patients, but the treatment is not yet recognized as a standard therapy. In still other cases, a transplant would only slow the disease but not cure it. These kinds of treatments are still at the clinical studies stage and include the use of mesenchymal stem cells, also found in the umbilical cord tissue.

Experimental therapies test whether stem cell transplantation is an effective, safe treatment for a specific disease. These are "Phase I" clinical studies, in which researchers test whether the new treatment is an improvement on existing treatments.

Download list of diseases in pdf format

Standard Therapies

Leukemias and Lymphomas

  • Acute Lymphoblastic Leukemia
  • Acute Myelogenous Leukemia
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia
  • Adult T-cell Leukemia/Lymphoma
  • Hodgkin’s Lymphoma
  • Non-Hodgkin’s Lymphoma
  • Chronic Lymphocytic Leukemia
  • Prolymphocytic Leukemia
Myelodysplastic/Myeloproliferative Disorders

  • Myelodysplastic Syndromes, including:
  • Myelodysplastic Syndromes, including:
    Refractory Anemia (RA)
    Refractory Anemia with Ringed Sideroblasts (RARS)
    Refractory Anemia with Excess Blasts (RAES)
    Refractory Anemia with Excess Blasts in Transformation (RAES-T)
    Chronic Myelomonocytic Leukemia
    Juvenile Myelomonocytic Leukemia
    Refractory Cytopenia
  • Philadelphia chromosome-positive Chronic Myelogenous Leukemia
  • Idiopathic Myelofibrosis
  • Polycythemia Vera
  • Essential Thrombocythemia
Plasma Cell Disorders

  • Multiple Myeloma
  • Plasma Cell Leukemia
  • Waldenstrom’s Macroglobulinemia
  • Amyloidosis
Bone marrow failure syndromes

  • Aplastic Anemia
  • Fanconi Anemia 
  • Dyskeratosis congenita
  • Paroxysmal Nocturnal Hemoglobinuria
  • Blackfan-Diamond Anemia
  • Congenital Dyserythropoietic Anemia
  • Pure Red Cell Aplasia
  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Inherited Platelet Abnormalities (Bernard-Soulier Syndrome, Glanzmann Thrombasthenia)
  • Kostmann Syndrome
  • Shwachman–Diamond Syndrome
Hemoglobin Diseases

  • Beta Thalassemia
  • Sickle Cell Disease
  • Selected cases of transfusion-dependent Piruvato-Kinasi Deficit 
Histiocytosis

  • Familial Hemophagocytic Lymphohistiocytosis
  • Griscelli Syndrome
  • Chédiak-Higashi Syndrome
  • Langerhans Cell Histiocytosis (Histiocytosis X)
Inherited Immune System Disorders

  • Chronic Granulomatous Disease
  • Leukocyte Adhesion Deficiency
  • Severe Combined Immunodeficiency (SCID) including:
  • SCID with Adenosine Deaminase Deficiency
    HLA Class I and Class II Molecules Deficiency
    Zap70 Deficiency
    Omenn Syndrome
    Purine Nucleoside Phosphorylase Deficiency
    Reticular Dysgenesis
    Defects Gamma-Chain common to Multiple Cytokine
    JAK3 Deficiency
  • Hyper-IgM Syndrome
  • Wiskott-Aldrich Syndrome
  • X-linked Lymphoproliferative Syndrome (Duncan)
  • Cartilage-Hair Hypoplasia
  • DiGeorge Syndrome
  • IPEX (Immunodysregulation Polyendocrinopathy Enteropathy X-linked) Syndrome
Inherited Metabolic Disorders

  • Hurler’s Syndrome (MPS-IH)
  • Scheie Syndrome (MPS-IS)
  • Maroteaux-Lamy Syndrome (MPS-VI)
  • Sly Syndrome (MPS-VII)
  • Adrenoleukodystrophy (ALD)
  • Fucosidosis
  • Gaucher Disease
  • Krabbe Disease (Globoid Cell Leukodystrophy)
  • Mannosidosis
  • Metachromatic Leukodystrophy
  • Mucolipidosis II (I-cell Disease)
  • Batten Disease
  • Sandhoff Disease
  • Osteopetrosis
Osteogenesis imperfecta

Other Inherited Disorders

  • Gunther’s Disease (Erythropoietic Porphyria)
Other Neoplasms

  • Ewing’s Sarcoma
  • Neuroblastoma
  • Renal Cell Carcinoma
  • Rhabdomyosarcoma
Others

  • Evans syndrome
  • Autoimmune Lymphoproliferative Syndrome
  • Progressive Systemic Sclerosis
  • Pediatric Neoplasms treated with chemo/radiotherapy

Therapies in Clinical Trials



The therapies in clinical trials are a mix of Allogeneic and Autologous modes of treatment.

Auto-Immune

Cardiovascular

Neurologic

Orthopedic

Stem Cells & Gene Therapy for Inherited Disorders (all therapies are Autologous)

Stem Cell Transplantation for Additional Inherited Disorders (all therapies are Allogeneic)

Stem Cell Transplatation for New Indications (all therapies are Allogeneic)


Experimental Treatments


Gene Therapy & Stem Cells (all therapies are Autologous)

Neurologic Disorders


Regenerative Medicine

Heart Liver

Clinical Trials with stem cells from Cord Tissue


Auto-Immune

Cardiovascular

Neurologic

Miscellaneous Regenerative Medicine

Liver Burns
Source: http://www.parentsguidecordblood.org/diseases.php
Copyright: Frances Verter, PhD.
Page created on: 03/09/2012
Last modified on: 24/10/2012
 
 
 
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