Cord Blood and Tissue in the Treatment of Disease
Cord blood, rich in hematopoietic (blood-forming) cells, has established uses in treating nearly 80 diseases and conditions12, including various blood cancers, inherited blood disorders, metabolic conditions, and immune deficiencies through transplantation. Most of the current approved treatments require donor (allogeneic) cells from a matched sibling or unrelated donor. Transplantations are characterized as either autologous (cells of the donor) or allogeneic (cells of a matched sibling or unrelated donor). Transplants using allogeneic cord blood cells have been performed worldwide for decades, primarily in pediatric patients, with ongoing research exploring applications in regenerative medicine.
Cord tissue is a rich source of mesenchymal stem cells (MSCs), which are being explored in regenerative medicine research for their potential to regulate the body’s immune response, support the tissue repair mechanism, and reduce inflammation.80
Researchers are currently conducting clinical trials with cord blood and cord tissue stem cells to investigate their potential for conditions like autism, cerebral palsy, stroke, and more. Below you’ll find a partial list of diseases and conditions involved in these studies.
Diseases Treated by Umbilical Cord Blood Transplantation
| Diagnosis |
Possible use by the baby (autologous) |
Possible use by sibling or other family member (allogeneic) |
Blood Cancers
(187,000 Diagnoses Each Year) |
| Acute Biphenotypic Leukemia |
|
✓ |
| Acute Lymphoblastic Leukemia (ALL) |
|
✓ |
| Acute Myelogenous Leukemia (AML) |
|
✓ |
| Acute Undifferentiated Leukemia |
|
✓ |
| Adult T-Cell Leukemia/Lymphoma |
|
✓ |
| Acute Myelogenous Leukemia (AML) |
|
✓ |
| Chronic Lymphocytic Leukemia (CLL) |
|
✓ |
| Chronic Myelogenous Leukemia (CML) |
|
✓ |
| Chronic Myelomonocytic Leukemia (CMML) |
|
✓ |
| Hodgkin Lymphoma |
|
✓ |
| Juvenile Chronic Myelogenous Leukemia (JCML) |
|
✓ |
| Juvenile Myelomonocytic Leukemia (JMML) |
|
✓ |
| Medulloblastoma |
✓ |
|
| Multiple Myeloma |
|
✓ |
| Neuroblastoma |
✓ |
|
| Non-Hodgkin Lymphoma |
|
✓ |
| Plasma Cell Leukemia |
✓ |
|
| Retinoblastoma |
✓ |
|
| Waldenstrom's Macroglobulinemia |
✓ |
|
| Blood Disorders |
| Acquired Aplastic Anemia |
|
✓ |
| Acute Myelofibrosis |
|
✓ |
| Agnogenic Myeloid Metaplasia |
|
✓ |
| Amegakaryocytosis / Congenital Thrombocytopenia |
|
✓ |
| Beta Thalassemia Major (also known as Cooley’s Anemia) |
|
✓ |
| Blackfan-Diamond Anemia |
|
✓ |
| Congenital Dyserythropoietic Anemia |
|
✓ |
| Fanconi Anemia |
|
✓ |
| Glanzmann Thrombasthenia |
|
✓ |
| Myelodysplastic Syndrome |
|
✓ |
| Paroxysmal Nocturnal Hemoglobinuria (PNH) |
|
✓ |
| Polycythemia Vera |
|
✓ |
| Pure Red Cell Aplasia |
|
✓ |
| Refractory Anemia (RA) |
|
✓ |
| Refractory Anemia with Ringed Sideroblasts (RARS) |
|
✓ |
| Refractory Anemia with Excess Blasts (RAEB) |
|
✓ |
| Refractory Anemia with Excess Blasts in Transformation (RAEB-T) |
|
✓ |
| Sickle Cell Disease |
|
✓ |
| Immune Disorders |
| Ataxia–Telangiectasia |
✓ |
|
| Bare Lymphocyte Syndrome |
|
✓ |
| Cartilage–Hair Hypoplasia |
|
✓ |
| Chediak–Higashi Syndrome |
|
✓ |
| Chronic Granulomatous Disease |
|
✓ |
| Common Variable Immunodeficiency Syndrome |
|
✓ |
| Gunther’s Disease (Erythropoietic Porphyria) |
|
✓ |
| Hermansky–Pudlak Syndrome |
|
✓ |
| Hemophagocytic Lymphohistiocytosis |
|
✓ |
| Hemophagocytosis Langerhans Cell Histiocytosis (Histiocytosis X) |
|
✓ |
| Infantile Genetic Agranulocytosis (Kostmann Syndrome) |
|
✓ |
| Leukocyte Adhesion Deficiency |
|
✓ |
| Lymphoproliferative Disorders (LPD) |
|
✓ |
| Neutrophil Actin Deficiency |
|
✓ |
| Omenn Syndrome |
|
✓ |
| Pearson’s Syndrome |
|
✓ |
| Reticular Dysgenesis |
|
✓ |
| SCID with Adenosine Deaminase Deficiency (ADA-SCID) |
|
✓ |
| SCID with Purine Nucleoside Phosphorylase Deficiency (PNP SCID) |
|
✓ |
| Shwachman–Diamond Syndrome |
|
✓ |
| Systemic Mastocytosis |
|
|
| Wiskott–Aldrich Syndrome |
|
✓ |
| Metabolic Disorders |
| Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN) |
|
✓ |
| Hunter’s Syndrome (MPS–II) |
|
✓ |
| Hurler’s Syndrome (MPS–IH) |
|
✓ |
| Krabbe Disease (Globoid Cell Leukodystrophy) |
|
✓ |
| Lesch–Nyhan Syndrome |
|
✓ |
| Maroteaux–Lamy Syndrome (MPS–VI) |
|
✓ |
| Metachromatic Leukodystrophy |
|
✓ |
| Mucolipidosis II (I–cell Disease) |
|
✓ |
| Mucopolysaccharidoses (MPS) |
|
✓ |
| Neuronal Ceroid Lipfuscinosis (Batten Disease) |
|
✓ |
| Niemann–Pick Disease |
|
✓ |
| Osteopetrosis |
|
✓ |
| Pelizaeus–Merzbacher Disease |
|
✓ |
| Sandhoff Disease |
|
✓ |
| Sanfilippo Syndrome (MPS–III) |
|
✓ |
| Sly Syndrome, Beta–Glucuronidase Deficiency (MPS–VII) |
|
✓ |
| Sly Syndrome (MPS-VIII) |
|
✓ |
| Tay–Sachs Disease |
|
✓ |
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General disclaimers:
The information provided above should not be used for diagnosing or treating a health problem or disease, and those seeking personal medical advice should consult with a licensed physician.
1) Cord blood use is determined by the physician and depends on the patient’s health, cord blood unit characteristics, and the donor match. While the use of cord blood in transplantation is well established, its regenerative medicine use is still being researched. No guarantees exist for future treatments currently under study.
2) Please be aware that the U.S. Food and Drug Administration (FDA) has not yet granted approval for the clinical use of any products manufactured from cord tissue. Furthermore, there is no guarantee that any clinical applications using cord tissue will receive FDA approval in the future.