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Treatable Diseases Using Stem Cells

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Sararajput
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Treatable Diseases Using Stem Cells

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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) 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

  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 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

  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 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

  7. 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

  8. 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"

  9. 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

  10. Phase 3 Traumatic Brain Injury publication

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