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Diseases Treated

This website was the first in 2003 to sort diseases treated with cord blood into 3 categories:

  1. Standard Therapies
  2. Therapies in Clinical Trials
  3. Experimental Treatments

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.

This web page was researched by Frances Verter, PhD, Alexey Bersenev, MD PhD, and Pedro Silva Couto, MSc ©2016-2018. Sources of information about established therapies were publications in the medical literature found via PubMed and Google Scholar. Sources of clinical trials were searches of, Chinese Clinical Trial Registry (ChiCTR), Japan University hospital Medical Information Network Clinical Trial Registry (UMIN-CTR), Japan Medical Association Clinical Trial Registry (JMA-CTR), Clinical Research Information Service from South Korea (CRiS), EU Clinical Trials Register (EudraCT), World Health Organization International Clinical Trials Registry Platform (ICTRP), Netherlands Trial Register (NTR), Australian New Zealand Clinical Trial Registry (ANZCTR), Clinical Trials Registry-India (CTRI), German Clinical Trials Register (DRKS), and Iranian Registry of Clinical Trials (IRCT).

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 side graphic (click on the image to expand it); 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

Anemias are deficiencies or malformations of red cells:......
Aplastic Anemia  
Fanconi Anemia
(The first cord blood transplant in 1988 was for FA, an inherited disorder)
Congenital Dyserythropoietic Anemia 
Paroxysmal Nocturnal Hemoglobinuria (PNH) 
Inherited Red Cell Abnormalities
Red cells contain hemoglobin and carry oxygen to the body:
Sickle Cell Disease 
Beta Thalassemia Major (aka Cooley's Anemia) 
Diamond-Blackfan Anemia 
Pure Red Cell Aplasia 
Inherited Platelet Abnormalities
Platelets are blood cells needed for clotting:
Amegakaryocytosis / Congenital Thrombocytopenia 
Glanzmann Thrombasthenia  
Inherited Immune System Disorders: Severe Combined Immunodeficiency:......
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 
Inherited Immune System Disorders: Neutropenias......
Infantile Genetic Agranulocytosis (Kostmann Syndrome) 
Inherited Immune System Disorders: Other......
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  
Myeloproliferative Disorders:......
Acute Myelofibrosis 
Agnogenic Myeloid Metaplasia (Myelofibrosis) 
Polycythemia Vera 
Essential Thrombocythemia 
Phagocyte Disorders
These are immune system cells that engulf and kill foreign organisms:
Chediak-Higashi Syndrome 
Chronic Granulomatous Disease 
Neutrophil Actin Deficiency 
Reticular Dysgenesis 
Bone Marrow Cancers:......
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

Mucopolysaccharidosis (MPS) Storage Diseases:......
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) 
Leukodystrophy Disorders:......
Adrenoleukodystrophy (ALD) 
Krabbe Disease(Globoid Cell Leukodystrophy) 
Metachromatic Leukodystrophy 
Pelizaeus-Merzbacher Disease 
Lysosomal Storage Diseases:......
Niemann-Pick Disease 
Sandhoff Disease 
Wolman Disease 
Other Inherited Metabolic Disorders:......
Lesch-Nyhan Syndrome 

Solid tumors not originating in the blood or immune system


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. 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):

  1. Phase 1: Safety study to see if the procedure or drug is well-tolerated.
  2. Phase 2: Larger study to measure effectiveness of the new treatment against a control group.
  3. 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.
  4. Phase 4: Post-marketing studies to learn even more about risks, benefits, and optimal use.


Neurologic Disorder

DiagnosisCord Blood
Cord Blood
Cord Tissue
Alzheimer's disease 
Cerebral Palsy
Global Developmental Delay 
Hearing Loss (acquired sensorineural)  
Intraventricular Hemorrhage 
Parkinson's disease 
Spinal Cord Injury 
Traumatic Brain Injury

Auto-Immune Disorders

DiagnosisCord Blood
Cord Blood
Cord Tissue
Alopecia Areata  
Amyotrophic Lateral Sclerosis (ALS) 
Crohn's Disease  
Eczema (Atopic Dermatitis)  
Graft-versus-Host Disease (GvHD) 
Multiple Sclerosis  
Rheumatoid Arthritis 
Systemic Sclerosis  
Ulcerative Colitis  


DiagnosisCord Blood
Cord Blood
Cord Tissue
Acute Myocardial Infarction (Heart Attack)  
Critical Limb Ischemia (CLI) 
Heart Failure  
Peripheral Arterial Disease (PAD) 
Surgery for Congenital Heart Defects  


DiagnosisCord Blood
Cord Blood
Cord Tissue
Diabetes, Type 1
(this is Auto-Immune)
Diabetes, Type 2
Diabetic Foot Ulcer  
Diabetic Peripheral Neuropathy  

Genetic and/or Metabolic Disorders

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


DiagnosisCord Blood
Cord Blood
Cord Tissue
Ankylosing Spondylitis  
Cartilage Injury 
Cleft Palate Repair  
Non-Union Fractures  
Osteochondral Lesion  
Spinal Fusion Surgery  


DiagnosisCord Blood
Cord Blood
Cord Tissue
Acute Respiratory Distress Syndrome (ARDS)  
BronchoPulmonary Dysplasia (BPD)
(lung disorder due to premature birth)
Erectile Dysfunction  
Eye Diseases 
Kidney Failure  
Liver Cirrhosis 
Liver Failure 
Peyronie's Disease  
Premature Ovarian Failure 
Uterine Scars  

Experimental Treatments

These are diagnoses for which stem cell treatments are being studied either in the laboratory with cell cultures or in animals that mimic the human disease. The experimental therapies are not yet in human clinical trials. In experimental research, it is often not clear whether an eventual therapy, if developed, would be Autologous or Allogeneic.

Due to the explosion of stem cell research and the difficulty keeping track of which studies are promising, this section does not have a list.