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Treatable Diseases Using Stem Cells

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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

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


Treatable diseases using stem cells

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)

see gene

therapy

trials

X

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


Treatable diseases using stem cells

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


Treatable diseases using stem cells

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

X

X

Waldenstrom’s Macroglobulinemia

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


Treatable diseases using stem cells

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.


Treatable diseases using stem cells

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

Phase 2

Phase 2

Phase 1

Phase 2

Cerebral Palsy

Phase 2

Hypoxic Ischemic Encephalopathy (HIE)

Phase 1

Traumatic Brain Injury

animal study

Phase 1

case report

Phase 2

Spinal Cord Injury

Autism

Phase 2

Hearing Loss (acquired sensorineural)

Phase 2

Auto-Immune Disorders

Diagnosis

Allogeneic

Autologous

case report

Phase 1

Amyotrophic Lateral Sclerosis (ALS)

Phase 1

Phase 2 placenta

Phase 3 Prochymal

Phase 2 Athersys

Crohn's Disease

Phase 3 ASTIC

Diabetes, Type 1

Phase 2

Phase 1

Phase 3 Prochymal

kids

Phase 3 Prochymal

adult

Graft-versus-Host Disease (GvHD)

Kidney plus stem cell transplant

Phase 2

review of trials

Phase 2

Lupus

Phase 1

review of trials

Phase 1, news

Multiple Sclerosis


Treatable diseases using stem cells

Rheumatoid Arthritis

Phase 2 placenta

case report

Phase 2 SCOT

Phase 2 CD34+

Scleroderma

Phase 1

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

Phase 2

Phase 2

Phase 3 BMAC

Critical Limb Ischemia

Phase 2

Compartment Syndrome (Battlefield Trauma)

Phase 1

Ischemic Stroke

Phase 2 placenta

Phase 3

Ischemic Heart Disease

Meta-Analysis

Cardiac Repair

Cell Trials blog

Phase 3 BAMI

Myocardial Infarction

Phase 1 Texas Heart

Inst.

Phase 3

Cardiomyopathy

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


Treatable diseases using stem cells

Phase 1,

news

Fanconi Anemia

Phase 1 Calimmune

Phase 1 Hutch

HIV

Metachromatic Leukodystrophy

Phase 1 Milan

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

Severe Combined Immunodeficiency

Sickle Cell

planned trial

case report

Phase 1 St. Jude

Phase 1 MSK

Phase 1 bluebird bio

Thalassemia

Phase 1

Phase 1 Milan

Wiskott-Aldrich Syndrome

Orthopedic

Diagnosis

Allogeneic

Autologous

Gintuit FDA

approval(NOT blood-

forming stem cells)

publication

Phase 1

Alveolar Cleft Palate Repair

Cartistem KFDA

approval

Knee Cartilage Repair

Phase 2 IMPACT

Miscellaneous

Diagnosis

Allogeneic

Autologous

Bronchopulmonary Dysplasia (BPD)

Phase 2 Pneumostem

Epidermolysis Bullosa

paper, story

Phase 1 Hutch

Berlin

patient PubMed &NPR

HIV aka "AIDS"


Treatable diseases using stem cells

in utero cord blood

transplant

Lysosomal Storage Diseases

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


Treatable diseases using stem cells

Phase 3

Traumatic Brain Injury

publication


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