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)
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
Autologous
Myelodysplastic Syndromes are also called pre-leukemia
Diagnosis
Allogeneic
Refractory Anemia
X
Autologous
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
Hodgkin's Lymphoma
X
Non-Hodgkin's Lymphoma (Burkitt's Lymphoma)
X
Autologous
Other Disorders of Blood Cell Proliferation
Diagnosis
Allogeneic
Autologous
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
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
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 EpsteinX Barr virus) 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
Cartilage-Hair Hypoplasia
X
Erythropoietic Porphyria
X
Hermansky-Pudlak Syndrome
X
Pearson's Syndrome
X
Shwachman-Diamond Syndrome
X
Systemic Mastocytosis
X
Autologous
Transplants for Inherited Metabolic Disorders
Diagnosis
Allogeneic
Mucopolysaccharidosis (MPS) Storage Diseases Hurler Syndrome (MPS-IH)
X
Scheie Syndrome (MPS-IS)
X
Hunter Syndrome (MPS-II)
X
Sanfilippo Syndrome (MPS-III)
X
Autologous
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.
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