Treatable Diseases using Stem Cells

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