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1. Diseases Treated
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
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

2. 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

3. 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

4. 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

5. 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. 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.

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

7. Rheumatoid Arthritis Phase 2 placenta
Scleroderma Phase 1
case report
Phase 2 SCOT
Phase 2 CD34+
Cardiovascular
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
Ischemia
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

8. 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
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
Miscellaneous
Diagnosis Allogeneic Autologous
Bronchopulmonary Dysplasia (BPD) Phase 2 Pneumostem
Epidermolysis Bullosa paper, story
HIV aka "AIDS"
Phase 1 Hutch
Berlin
patient PubMed &NPR

9. 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.
Clinical Trials with stem cells from Cord Tissue
In January 2014 a review article found that 70% of clinical trials using Mesenchymal Stem Cells (MSC)
from umbilical cord tissue are in China.
Diagnosis Allogeneic Autologous
Alzheimer's Disease Phase 2 Neurostem
Aplastic Anemia publication
Ataxia (Hereditary) publication
Autism publication
Cardiomyopathy Phase 2
Cerebral Palsy case report
Diabetes, Type 1 publication
Graft-versus-Host Disease (GvHD) publication
Hypoxic Ischemic Encephalopathy (HIE) Phase 1
Liver Cirrhosis publication
Lupus publication
Multiple Sclerosis case report
Rheumatoid Arthritis publication
Spinal Cord Injury publication

10. Phase 3
Traumatic Brain Injury publication