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Diseases Treated

This website was the first in 2003 to sort diseases treated with blood-forming stem cells into 3 categories:

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

It is also important to distinguish between 2 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 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.

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.

This web page was researched by Frances Verter, PhD, Alexey Bersenev, MD PhD, and Pedro Silva Couto, MSc ©2016. Sources of disease information are publications in the medical literature that were found by keyword searches of PubMed and Google Scholar.

Standard Therapies

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.

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

Diagnosis Allogeneic Autologous
Acute Lymphoblastic Leukemia (ALL) X  
Acute Myelogenous Leukemia (AML) X  
Acute Biphenotypic Leukemia X  
Acute Undifferentiated Leukemia X  
Chronic Lymphocytic Leukemia (CLL) X  
Chronic Myelogenous Leukemia (CML) X  
Juvenile Chronic Myelogenous Leukemia (JCML) X  
Juvenile Myelomonocytic Leukemia (JMML) X  

Myelodysplastic Syndromes are also called pre-leukemia

Diagnosis Allogeneic Autologous
Refractory Anemia X  
Refractory Anemia with Ringed Sideroblasts (Sideroblastic anemia) X  
Refractory Anemia with Excess Blasts X  
Refractory Anemia with Excess Blasts in Transformation X  
Chronic Myelomonocytic Leukemia (CMML) X  

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

Diagnosis Allogeneic Autologous
Hodgkin's Lymphoma X  
Non-Hodgkin's Lymphoma (Burkitt's Lymphoma) X  

Other Disorders of Blood Cell Proliferation

Diagnosis Allogeneic Autologous
Anemias are deficiencies or malformations of red cells    
Aplastic Anemia X X
Fanconi Anemia
(The first cord blood transplant in 1988 was for FA, an inherited disorder)
X see gene therapy trials
Congenital Dyserythropoietic Anemia X  
Paroxysmal Nocturnal Hemoglobinuria (PNH) X  
Inherited Red Cell Abnormalities
Red cells contain hemoglobin and carry oxygen to the body
Sickle Cell Disease X  
Beta Thalassemia Major (aka Cooley's Anemia) X  
Diamond-Blackfan Anemia X  
Pure Red Cell Aplasia X  
Inherited Platelet Abnormalities
Platelets are blood cells needed for clotting
Amegakaryocytosis / Congenital Thrombocytopenia X  
Glanzmann Thrombasthenia X  
Inherited Immune System Disorders: Severe Combined Immunodeficiency    
SCID with Adenosine Deaminase Deficiency (ADA-SCID) X  
SCID which is X-linked X  
SCID with absence of T & B Cells X  
SCID with absence of T Cells, Normal B Cells X  
Omenn Syndrome X  
Inherited Immune System Disorders: Neutropenias    
Infantile Genetic Agranulocytosis (Kostmann Syndrome) X  
Myelokathexis X  
Inherited Immune System Disorders: Other    
Ataxia-Telangiectasia X  
Bare Lymphocyte Syndrome X  
Common Variable Immunodeficiency X  
DiGeorge Syndrome X  
Hemophagocytic Lymphohistiocytosis X  
Leukocyte Adhesion Deficiency X  
Lymphoproliferative Disorders X  
Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus) X  
Wiskott-Aldrich Syndrome X  
Myeloproliferative Disorders    
Acute Myelofibrosis X  
Agnogenic Myeloid Metaplasia (Myelofibrosis) X  
Polycythemia Vera X  
Essential Thrombocythemia X  
Phagocyte Disorders
These are immune system cells that engulf and kill foreign organisms
Chediak-Higashi Syndrome X  
Chronic Granulomatous Disease X  
Neutrophil Actin Deficiency X  
Reticular Dysgenesis X  
Bone Marrow Cancers    
Multiple Myeloma X X
Plasma Cell Leukemia X X
Waldenstrom's Macroglobulinemia X X

Transplants for Inherited Disorders of the Immune System & Other Organs

Diagnosis Allogeneic Autologous
Cartilage-Hair Hypoplasia X  
Erythropoietic Porphyria X  
Hermansky-Pudlak Syndrome X  
Pearson's Syndrome X  
Shwachman-Diamond Syndrome X  
Systemic Mastocytosis X  

Transplants for Inherited Metabolic Disorders

Diagnosis Allogeneic Autologous
Mucopolysaccharidosis (MPS) Storage Diseases    
Hurler Syndrome (MPS-IH) X  
Scheie Syndrome (MPS-IS) X  
Hunter Syndrome (MPS-II) X  
Sanfilippo Syndrome (MPS-III) X  
Morquio Syndrome (MPS-IV) X  
Maroteaux-Lamy Syndrome (MPS-VI) X  
Sly Syndrome (MPS-VII) (beta-glucuronidase deficiency) X  
Mucolipidosis II (I-cell Disease) X  
Leukodystrophy Disorders    
Adrenoleukodystrophy (ALD) X  
Krabbe Disease(Globoid Cell Leukodystrophy) X  
Metachromatic Leukodystrophy X  
Pelizaeus-Merzbacher Disease X  
Lysosomal Storage Diseases    
Niemann-Pick Disease X  
Sandhoff Disease X  
Wolman Disease X  
Other Inherited Metabolic Disorders    
Lesch-Nyhan Syndrome X  
Osteopetrosis X  

Solid tumors not originating in the blood or immune system

Diagnosis Allogeneic Autologous
Neuroblastoma   X
Medulloblastoma   X
Retinoblastoma   X

Therapies in Clinical Trials

A "clinical trial" is a study in human patients for an emerging therapy that has not been adopted as standard therapy. provides a searchable database of all trials in the United States, and holds many international trials as well.

The phases of U.S. clinical trials in humans are defined as follows:

  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

Diagnosis Allogeneic Autologous
Autism   Phase 2
Phase 1
Cerebral Palsy Phase 2 Phase 2
Phase 2
Phase 1
Phase 2
Hearing Loss (acquired sensorineural)   Phase 2
Hypoxic Ischemic Encephalopathy (HIE)   Phase 1
Spinal Cord Injury case report
Phase 2

Auto-Immune Disorders

Diagnosis Allogeneic Autologous
Amyotrophic Lateral Sclerosis (ALS) case report
Phase 1
Phase 1
Crohn's Disease Phase 2 placenta
Phase 3 Prochymal
Phase 2 Athersys
Phase 3 ASTIC
Diabetes, Type 1 Phase 2 Phase 1
Graft-versus-Host Disease (GvHD) Phase 3 Prochymal kids
Phase 3 Prochymal adult
Kidney plus stem cell transplant Phase 2  
Lupus Phase 1 review of trials
Phase 2
Multiple Sclerosis   review of trials
Phase 1, news
Rheumatoid Arthritis Phase 2 placenta  
Scleroderma Phase 1 case report
Phase 2 SCOT
Phase 2 CD34+


Diagnosis Allogeneic Autologous
Congenital Defects    
Support for Misc. Open Cardiac Surgery   Phase 1
Hypoplastic Left Heart Syndrome (HLHS)   Phase 1
Grow Vascular Graft   Phase 1, story
Critical Limb Ischemia Phase 2 Phase 2
Phase 2
Phase 3 BMAC
Compartment Syndrome (Battlefield Trauma)   Phase 1
Ischemic Stroke Phase 2 placenta Phase 3
Ischemic Heart Disease   Meta-Analysis
Cardiac Repair    
Myocardial Infarction   Cell Trials blog
Phase 3 BAMI
Cardiomyopathy case report Phase 1 Texas Heart Inst.
Phase 3

Gene Therapy for Inherited Disorders

Note this list only includes gene therapy that is delivered with blood-forming stem cells. There are many more gene therapy clinical trials, some for the same diseases, that have a different route of delivery (muscle injections, intercerebral injections, etc.)

Diagnosis Allogeneic Autologous
Adrenoleukodystrophy   Phase 2 bluebird bio
Chronic Granulomatous Disease (X-linked)   Phase 1
Fanconi Anemia   Phase 1,
HIV   Phase 1 Calimmune
Phase 1 Hutch
Metachromatic Leukodystrophy   Phase 1 Milan
Severe Combined Immunodeficiency   Phase 1 X-linked SCID Boston
Phase 1 X-linked SCID St. Jude
Phase 1 X-linked SCID Paris
Phase 1 ADA-SCID Duke
Sickle Cell   planned trial
Thalassemia   case report
Phase 1 St. Jude
Phase 1 MSK
Phase 1 bluebird bio
Wiskott-Aldrich Syndrome   Phase 1
Phase 1 Milan


Diagnosis Allogeneic Autologous
Alveolar Cleft Palate Repair Gintuit FDA approval (NOT blood-forming stem cells) publication
Phase 1
Knee Cartilage Repair Cartistem KFDA approval Phase 2 IMPACT


Diagnosis Allogeneic Autologous
Bronchopulmonary Dysplasia (BPD) Phase 2 Pneumostem  
Epidermolysis Bullosa paper, story  
HIV aka "AIDS" Phase 1 Hutch
Berlin patient PubMed & NPR
Lysosomal Storage Diseases in utero cord blood transplant  

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.