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

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)

Autologous

Myelodysplastic Syndromes are also called pre-leukemia

Diagnosis

Allogeneic

Refractory Anemia

Autologous

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

Diagnosis

Allogeneic

Hodgkin's Lymphoma

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

Autologous

Other Disorders of Blood Cell Proliferation

Diagnosis

Allogeneic

Autologous

Aplastic Anemia

Fanconi Anemia
(The first cord blood transplant in 1988 was for FA, an inherited
disorder)

see gene
therapy
trials

Congenital Dyserythropoietic Anemia

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Anemias are deficiencies or malformations of red cells

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)

Myelokathexis

Inherited Immune System Disorders: Other

Ataxia-Telangiectasia

Bare Lymphocyte Syndrome

Common Variable Immunodeficiency

DiGeorge Syndrome

Hemophagocytic Lymphohistiocytosis

Leukocyte Adhesion Deficiency

Lymphoproliferative Disorders

Lymphoproliferative Disorder, X-linked (Susceptibility to EpsteinX

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

Waldenstroms Macroglobulinemia

Transplants for Inherited Disorders of the Immune System & Other Organs

Diagnosis

Allogeneic

Cartilage-Hair Hypoplasia

Erythropoietic Porphyria

Hermansky-Pudlak Syndrome

Pearson's Syndrome

Shwachman-Diamond Syndrome

Systemic Mastocytosis

Autologous

Transplants for Inherited Metabolic Disorders

Diagnosis

Allogeneic

Mucopolysaccharidosis (MPS) Storage Diseases

Hurler Syndrome (MPS-IH)

Scheie Syndrome (MPS-IS)

Hunter Syndrome (MPS-II)

Sanfilippo Syndrome (MPS-III)

Autologous

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

Osteopetrosis

Solid tumors not originating in the blood or immune system

Diagnosis

Allogeneic

Autologous

Neuroblastoma

Medulloblastoma

Retinoblastoma

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 Injury

Diagnosis

Cerebral Palsy

Allogeneic

Autologous

Phase 2

Phase 2
Phase 2
Phase 1
Phase 2

Hypoxic Ischemic Encephalopathy (HIE)

Phase 1

Traumatic Brain Injury

animal study

Spinal Cord Injury

case report
Phase 2

Phase 1

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

Multiple Sclerosis

review of trials
Phase 2
review of trials
Phase 1, news

Rheumatoid Arthritis

Phase 2 placenta

Scleroderma

Phase 1

case report
Phase 2 SCOT
Phase 2 CD34+

Allogeneic

Autologous

Cardiovascular

Diagnosis
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 3 BMAC

Phase 2

Compartment Syndrome (Battlefield Trauma)

Ischemic Stroke

Phase 1
Phase 2 placenta

Ischemic Heart Disease

Phase 3
Meta-Analysis

Cardiac Repair
Cell Trials blog
Phase 3 BAMI

Myocardial Infarction

Cardiomyopathy

Phase 1 Texas Heart

Inst.
Phase 3

case report

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,
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 bloodforming 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

Miscellaneous

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

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

Autologous

Phase 3
Traumatic Brain Injury

publication