Diseases Treated with Cord Blood: Cord Blood Uses

Diseases treated with cord blood can be divided into 2 categories:

• Proven Standard Therapies
• Therapies in Clinical Trials

Before choosing the right form of cord blood banking it is also important to distinguish between two modes of treatment:

• Allogeneic - patient receives stem cells from a matching donor, either a sibling or an unrelated donor
• Autologous - patient receives their own stem cells

When parents donate cord blood to a public bank, they are supporting patients around the world who are searching for an unrelated Allogeneic donor. When parents save cord blood in a family bank, they are reserving the options that the baby can use its own stem cells for an Autologous treatment, or an immediate relative (sibling or parents) can use the stem cells for an Allogeneic treatment.

Standard Therapies with blood-forming stem cells

These are diseases for which transplants of blood-forming stem cells (Hematopoietic Stem Cell Transplants, HSCT) are a standard treatment. For some diseases they are the only therapy, and in other diseases they are only employed when front-line therapies have failed or the disease is very aggressive. The lists below include ALL therapies that use blood-forming stem cells, without distinction as to whether the stem cells were extracted from bone marrow, peripheral blood, or cord blood. Most of the diseases for which HSCT is a standard treatment are disorders of blood cell lineage. The proliferation by which blood cells are formed from stem cells is illustrated in the graphic below; you can also read about specific cell types in the immune system in more detail. In the United States, most health insurance providers will pay for a stem cell transplant if it is a "standard therapy" for the patient's diagnosis.

Leukemia is a cancer of the blood immune system, where the cells are called leukocytes or white cells:

• Acute Lymphoblastic Leukemia (ALL) ✔
• Acute Myelogenous Leukemia (AML) ✔
• Acute Biphenotypic Leukemia ✔
• Acute Undifferentiated Leukemia ✔
• Chronic Lymphocytic Leukemia (CLL) ✔
• Chronic Myelogenous Leukemia (CML) ✔
• Juvenile Chronic Myelogenous Leukemia (JCML) ✔
• Juvenile Myelomonocytic Leukemia (JMML) ✔

Myelodysplastic Syndromes are also called pre-leukemia:

• Refractory Anemia ✔ 
• Refractory Anemia with Ringed Sideroblasts (Sideroblastic anemia) ✔ 
•  Refractory Anemia with Excess Blasts ✔ 
•  Refractory Anemia with Excess Blasts in Transformation ✔ 
•  Chronic Myelomonocytic Leukemia (CMML) ✔

Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels:

• Hodgkin's Lymphoma ✔ 
• Non-Hodgkin's Lymphoma (Burkitt's Lymphoma) ✔

Other Disorders of Blood Cell Proliferation

• Aplastic Anemia ✔
• Fanconi Anemia (The first cord blood transplant in 1988 was for FA, an inherited disorder) ✔
• Congenital Dyserythropoietic Anemia ✔
• Paroxysmal Nocturnal Hemoglobinuria (PNH) ✔
• Sickle Cell Disease ✔
• Beta Thalassemia Major (aka Cooley's Anemia) ✔
• Diamond-Blackfan Anemia ✔
• Pure Red Cell Aplasia ✔
• 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 ✔
• Infantile Genetic Agranulocytosis (Kostmann Syndrome) ✔
• Myelokathexis ✔
• Ataxia-Telangiectasia ✔
• Bare Lymphocyte Syndrome ✔
• Common Variable Immunodeficiency ✔
• DiGeorge Syndrome ✔
• Hemophagocytic Lymphohistiocytosis ✔
• Leukocyte Adhesion Deficiency ✔
• Lymphoproliferative Disorders ✔
• Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus) ✔
• 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 ✔

Transplants for Inherited Disorders of the Immune System & Other Organs

• Cartilage-Hair Hypoplasia ✔
• Erythropoietic Porphyria ✔
• Hermansky-Pudlak Syndrome ✔
• Pearson's Syndrome ✔
• Shwachman-Diamond Syndrome ✔
• Systemic Mastocytosis ✔

Transplants for Inherited Metabolic Disorders

• 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 (MPS-VII) (beta-glucuronidase deficiency) ✔
• Mucolipidosis II (I-cell Disease) ✔
• Adrenoleukodystrophy (ALD) ✔
• Krabbe Disease(Globoid Cell Leukodystrophy) ✔
• Metachromatic Leukodystrophy ✔
• Pelizaeus-Merzbacher Disease ✔
• Niemann-Pick Disease ✔
• Sandhoff Disease ✔
• Wolman Disease ✔
• Lesch-Nyhan Syndrome ✔
• Osteopetrosis ✔

Solid tumors not originating in the blood or immune system

• Neuroblastoma ✔
• Medulloblastoma ✔
• Retinoblastoma ✔

Diseases and Disorders that have been in Clinical Trials with Cord Blood or Cord Tissue Cells

A "clinical trial" is a study in human patients for an emerging therapy that has not been adopted as standard therapy. This website has pages that enable patients to search worldwide for currently recruiting clinical trials with ether cord blood or umbilical cord tissue MSC (mesenchymal stem cells). The table below checks off all diagnoses that have ever been treated in clinical trials with cord blood or cord tissue, regardless of whether the trials are still open. The phases of U.S. clinical trials in humans are defined as follows (in other countries phases may be different): 

• Phase 1: Safety study to see if the procedure or drug is well-tolerated. 
• Phase 2: Larger study to measure effectiveness of the new treatment against a control group. 
• Phase 3: Even larger study to compare the effect of various parameters such as dose and administration, and to monitor side effects prior to market release. 
• Phase 4: Post-marketing studies to learn even more about risks, benefits, and optimal use.

Neurologic Disorder

• Alzheimer's disease ✔
• Autism ✔
• Cerebral Palsy ✔
• Encephalopathy ✔
• Global Developmental Delay ✔
• Hearing Loss (acquired sensorineural) ✔
• Intraventricular Hemorrhage ✔
• Parkinson's disease ✔
• Spinal Cord Injury ✔
• Stroke ✔
• Traumatic Brain Injury ✔

Auto-Immune Disorders

• Alopecia Areata ✔
• Amyotrophic Lateral Sclerosis (ALS) ✔ 
• Crohn's Disease ✔
• Eczema (Atopic Dermatitis) ✔
• Graft-versus-Host Disease (GvHD) ✔ 
• Lupus ✔ 
• Multiple Sclerosis ✔ Psoriasis ✔
• Rheumatoid Arthritis ✔ 
• Scleroderma
• Systemic Sclerosis ✔
• Ulcerative Colitis ✔

Cardiovascular

• Acute Myocardial Infarction (Heart Attack) ✔
• Cardiomyopathy ✔ 
• Critical Limb Ischemia (CLI) ✔ 
• Heart Failure ✔ 
• Peripheral Arterial Disease (PAD) ✔ 
• Surgery for Congenital Heart Defects ✔

Diabetic

• Diabetes, Type 1 (this is Auto-Immune) ✔ 
• Diabetes, Type 2 ✔ 
• Diabetic Foot Ulcer ✔
• Diabetic Peripheral Neuropathy ✔

Genetic and/or Metabolic Disorders 

• Aging Frailty ✔ ✔ 
• Duchenne Muscular Dystrophy ✔ 
• Epidermolysis Bullosa ✔ 
•  Hereditary Ataxia ✔ 
• Lysosomal Storage Diseases ✔ 
•  Metabolic Syndrome ✔ 
• Severe Combined Immunodeficiency ✔ 
•  Spinal muscular atrophy ✔ 
• Tay-Sachs Disease ✔ 

Orthopedic 

• Ankylosing Spondylitis ✔ 
• Cartilage Injury ✔ ✔ 
• Cleft Palate Repair ✔ 
• Non-Union Fractures ✔ 
• Osteoarthritis ✔ ✔ 
• Osteochondral Lesion ✔ 
•  Spinal Fusion Surgery ✔ 

Others

• Acute Respiratory Distress Syndrome (ARDS) ✔ 
• BronchoPulmonary Dysplasia (BPD) (lung disorder due to premature birth) ✔ 
• Erectile Dysfunction ✔ 
• Eye Diseases ✔ 
• Fistula ✔ 
• HIV ✔ Kidney Failure ✔ 
• Liver Cirrhosis ✔ 
• Liver Failure ✔ 
• Peyronie's Disease ✔ 
• Premature Ovarian Failure ✔ 
• Uterine Scars ✔ 
• Wounds ✔

Ref: ParentsGuideCordBlood.org


Related: Cord Blood Banking in Malaysia