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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

DiagnosisAllogeneicAutologous
Acute Lymphoblastic Leukemia (ALL)X 
Acute Myelogenous Leukemia (AML)X 
Acute Biphenotypic LeukemiaX 
Acute Undifferentiated LeukemiaX 
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

DiagnosisAllogeneicAutologous
Refractory AnemiaX 
Refractory Anemia with Ringed Sideroblasts (Sideroblastic anemia)X 
Refractory Anemia with Excess BlastsX 
Refractory Anemia with Excess Blasts in TransformationX 
Chronic Myelomonocytic Leukemia (CMML)X 

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

DiagnosisAllogeneicAutologous
Hodgkin's LymphomaX 
Non-Hodgkin's Lymphoma (Burkitt's Lymphoma)X 

Other Disorders of Blood Cell Proliferation

DiagnosisAllogeneicAutologous
Anemias are deficiencies or malformations of red cells  
Aplastic Anemia XX
Fanconi Anemia
(The first cord blood transplant in 1988 was for FA, an inherited disorder)
Xsee gene therapy trials
Congenital Dyserythropoietic AnemiaX 
Paroxysmal Nocturnal Hemoglobinuria (PNH)X 
Inherited Red Cell Abnormalities
Red cells contain hemoglobin and carry oxygen to the body
  
Sickle Cell DiseaseX 
Beta Thalassemia Major (aka Cooley's Anemia)X 
Diamond-Blackfan AnemiaX 
Pure Red Cell AplasiaX 
Inherited Platelet Abnormalities
Platelets are blood cells needed for clotting
  
Amegakaryocytosis / Congenital ThrombocytopeniaX 
Glanzmann Thrombasthenia X 
Inherited Immune System Disorders: Severe Combined Immunodeficiency  
SCID with Adenosine Deaminase Deficiency (ADA-SCID)X 
SCID which is X-linkedX 
SCID with absence of T & B CellsX 
SCID with absence of T Cells, Normal B CellsX 
Omenn SyndromeX 
Inherited Immune System Disorders: Neutropenias  
Infantile Genetic Agranulocytosis (Kostmann Syndrome)X 
MyelokathexisX 
Inherited Immune System Disorders: Other  
Ataxia-TelangiectasiaX 
Bare Lymphocyte SyndromeX 
Common Variable ImmunodeficiencyX 
DiGeorge SyndromeX 
Hemophagocytic LymphohistiocytosisX 
Leukocyte Adhesion DeficiencyX 
Lymphoproliferative DisordersX 
Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus)X 
Wiskott-Aldrich Syndrome X 
Myeloproliferative Disorders  
Acute MyelofibrosisX 
Agnogenic Myeloid Metaplasia (Myelofibrosis)X 
Polycythemia VeraX 
Essential ThrombocythemiaX 
Phagocyte Disorders
These are immune system cells that engulf and kill foreign organisms
  
Chediak-Higashi SyndromeX 
Chronic Granulomatous DiseaseX 
Neutrophil Actin DeficiencyX 
Reticular DysgenesisX 
Bone Marrow Cancers  
Multiple MyelomaXX
Plasma Cell LeukemiaXX
Waldenstrom's MacroglobulinemiaXX

Transplants for Inherited Disorders of the Immune System & Other Organs

DiagnosisAllogeneicAutologous
Cartilage-Hair HypoplasiaX 
Erythropoietic PorphyriaX 
Hermansky-Pudlak SyndromeX 
Pearson's SyndromeX 
Shwachman-Diamond SyndromeX 
Systemic MastocytosisX 

Transplants for Inherited Metabolic Disorders

DiagnosisAllogeneicAutologous
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 LeukodystrophyX 
Pelizaeus-Merzbacher DiseaseX 
Lysosomal Storage Diseases  
Niemann-Pick DiseaseX 
Sandhoff DiseaseX 
Wolman DiseaseX 
Other Inherited Metabolic Disorders  
Lesch-Nyhan SyndromeX 
OsteopetrosisX 

Solid tumors not originating in the blood or immune system

DiagnosisAllogeneicAutologous
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. ClinicalTrials.gov 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

DiagnosisAllogeneicAutologous
Autism Phase 2
Phase 1
Cerebral PalsyPhase 2Phase 2
Phase 2
Phase 1
Phase 2
Hearing Loss (acquired sensorineural) Phase 2
Hypoxic Ischemic Encephalopathy (HIE) Phase 1
Spinal Cord Injurycase report
Phase 2
 

Auto-Immune Disorders

DiagnosisAllogeneicAutologous
Amyotrophic Lateral Sclerosis (ALS)case report
Phase 1
Phase 1
Crohn's DiseasePhase 2 placenta
Phase 3 Prochymal
Phase 2 Athersys
Phase 3 ASTIC
Diabetes, Type 1Phase 2Phase 1
Graft-versus-Host Disease (GvHD)Phase 3 Prochymal kids
Phase 3 Prochymal adult
 
Kidney plus stem cell transplantPhase 2 
LupusPhase 1review of trials
Phase 2
Multiple Sclerosis review of trials
Phase 1, news
Rheumatoid ArthritisPhase 2 placenta 
SclerodermaPhase 1case report
Phase 2 SCOT
Phase 2 CD34+

Cardiovascular

DiagnosisAllogeneicAutologous
Congenital Defects  
Support for Misc. Open Cardiac Surgery Phase 1
Hypoplastic Left Heart Syndrome (HLHS) Phase 1
Grow Vascular Graft Phase 1, story
Ischemia  
Critical Limb IschemiaPhase 2Phase 2
Phase 2
Phase 3 BMAC
Compartment Syndrome (Battlefield Trauma) Phase 1
Ischemic StrokePhase 2 placentaPhase 3
Ischemic Heart Disease Meta-Analysis
Cardiac Repair  
Myocardial Infarction Cell Trials blog
Phase 3 BAMI
Cardiomyopathycase reportPhase 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.)

DiagnosisAllogeneicAutologous
Adrenoleukodystrophy Phase 2 bluebird bio
Chronic Granulomatous Disease (X-linked) Phase 1
Fanconi Anemia Phase 1,
news
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 UCLA
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

Orthopedic

DiagnosisAllogeneicAutologous
Alveolar Cleft Palate RepairGintuit FDA approval (NOT blood-forming stem cells)publication
Phase 1
Knee Cartilage RepairCartistem KFDA approvalPhase 2 IMPACT

Miscellaneous

DiagnosisAllogeneicAutologous
Bronchopulmonary Dysplasia (BPD)Phase 2 Pneumostem 
Epidermolysis Bullosapaper, story 
HIV aka "AIDS"Phase 1 Hutch
Berlin patient PubMed & NPR
 
Lysosomal Storage Diseasesin 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.