Cord Blood Application

Current clinical applications for cord blood

• Stem cell source for
  • Allogeneic hematopoietic stem cell transplantation
  • Autologous transplantation
    • Back-up source of hematopoietic stem cells should the child develop aplastic anemia or high risk cancer/leukemia
    • Vehicles to deliver genes(gene therapy)
• Autologous tranfusion of red cells to premature infants(under study)

Source of hematopoietic stem cells for allogeneic transplants

• Bone Marrow
• Peripheral blood stem cells
• Umbilical cord blood

Hematopoietic stem cell transplantation

Indications	Principle
Malignancies - leukemia, solid tumors, lymphomas, neuroblastoma	Eradicate "resistant cancer with high dose chemotherapy
Immunde Disorders - Severe Combined Immune Deficiency	Provide functional immune system

Hematopoietic stem cell transplantation

Indications	Principle
Genetic diseases - Sickle cell disease, Thalassemia	Provide normal hemoglobin and red blood cells
Metabolic disorders - Hurlers syndrome, Adrenoleukodystrophy(ALD)	HSC serve as source of enzyme, cross areas of need
Bone marrow failure disorders - aplastic anemia, Fanconi's anemia, Dyskeratosis congenita, Shwachman Diamond Syndrome	Provide new source of hematopoietic stem cells

Umbilical Cord Blood Transplantation

• Over 3,000 transplants to date using cord blood, mostly "unrelated"
• Donor pool is expanding
  • Over 200,000 potential donor cords frozen
• New data showing advantages of cord blood transplant versus bone marrow

Unrelated Donor Transplantation in Children: Stem Cell Source Utilization

U of I Hematopoietic Stem Cell Bank

• Started in 2004 with Foundation funding
• Collection and storage of umbilical cord blood for Research Only
• Provide cord blood for investigators in Iowa and other research centers
• Only bank that provides stem cells that are fresh or frozen

Diseases where gene therapy has been or is being considered*

• Severe immune deficiencies
  • Severe Combined Immuno Deficiency(SCIDs), Adenosine Deaminase (ADA) deficiency, Common Granulomatous Disease(CGD)
• Hematopoietic stem cell usage in genen therapy trials
  • SCIDs
  • ADA deficiency
  • ILz gamma common chain deficiency
  • CGD
  • Fanconi's anemia
  • Dyskeratosis congenita