Diseases Treated by Blood Stem Cells

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1 Diseases Treated by Blood Stem Cells This is the only website to group hematopoietic (blood) stem cell treatments according to whether they are standard, in trial, or experimental. (Courtesy of Parents Guide to Cord Blood Banks see ) Hematopoietic stem cells are capable of evolving into all the specific cell types in the blood and immune system. They can be found in people of all ages. The three sources of hematopoietic stem cells which are routinely used for medical treatments are: 1. the bone marrow of an adult person 2. the peripheral blood of an adult person 3. the umbilical cord blood of a newborn baby When a patient requires a Hemaotpoietic Stem Cell Transplant (HSCT), the treating physician will decide which source of stem cells to use. This will depend on several factors, including but not limited to: the degree of match between donor and patient (sometimes the donor and patient are one and the same person), the expected speed of engraftment, and the amount of time available to search for a perfectly matching donor. Categories below: Standard Therapies Therapies in Clinical Trials Experimental Treatments

2 Image courtesy of Dr. Jeff Drew next step... Standard Therapies These are diseases for which 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 (see figure below), ranging from the stem cells in the bone marrow down to specific cell types in the blood. In the United States, most health insurance providers will only pay for a stem cell transplant if it is a "standard therapy" for the patient's diagnosis. As an example, for illustrative purposes only, here is the clinical policy bulletin of Aetna, one of the nation's largest health insurance providers, regarding stem cell transplants. Note that they cover various expenses regarding the storage and testing of cord blood if a relative of the newborn requires a transplant.

3 Recommended Resources: Cancer.gov National Cancer Institute database on cancer Association of Cancer Online Resources is a clearinghouse for both medical information and patient support groups Oncolink is one of the best cancer guides on the internet; it is produced by the University of Pennsylvania Leukemias (Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells) Acute Leukemia Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Acute Biphenotypic Leukemia Acute Undifferentiated Leukemia Chronic Leukemia Chronic Myelogenous Leukemia (CML) Chronic Lymphocytic Leukemia (CLL) Juvenile Chronic Myelogenous Leukemia (JCML) Juvenile Myelomonocytic Leukemia (JMML) Myelodysplastic Syndromes ( Myelodysplasia is sometimes called pre leukemia) Refractory Anemia (RA) Refractory Anemia with Ringed Sideroblasts (RARS) Refractory Anemia with Excess Blasts (RAEB) Refractory Anemia with Excess Blasts in Transformation (RAEB T) Chronic Myelomonocytic Leukemia (CMML) Lymphomas

4 ( Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels) Hodgkin's Lymphoma Non Hodgkin's Lymphoma Burkitt's Lymphoma Inherited Red Cell (Erythrocyte) Abnormalities (Red cells contain hemoglobin and carry oxygen to the body) Beta Thalassemia Major (also known as Cooley's Anemia) Blackfan Diamond Anemia Pure Red Cell Aplasia Sickle Cell Disease Other Disorders of Blood Cell Proliferation Anemias (Anemias are deficiencies or malformations of red cells) severe Aplastic Anemia Congenital Dyserythropoietic Anemia Fanconi Anemia (Note: the first cord blood transplant in 1988 was for this disease) Paroxysmal Nocturnal Hemoglobinuria (PNH) Pure Red Cell Aplasia Inherited Platelet Abnormalities (Platelets are small blood cells needed for clotting) Amegakaryocytosis / Congenital Thrombocytopenia Glanzmann Thrombasthenia Myeloproliferative Disorders Acute Myelofibrosis Agnogenic Myeloid Metaplasia (Myelofibrosis) Polycythemia Vera Essential Thrombocythemia

5 Inherited Immune System Disorders Severe Combined Immunodeficiency (SCID) 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 Kostmann Syndrome Myelokathexis Inherited Immune System Disorders Other Ataxia Telangiectasia Bare Lymphocyte Syndrome Common Variable Immunodeficiency DiGeorge Syndrome Leukocyte Adhesion Deficiency Lymphoproliferative Disorders (LPD) Lymphoproliferative Disorder, X linked (also known as Epstein Barr Virus Susceptibility) Wiskott Aldrich Syndrome Phagocyte Disorders ( Phagocytes are immune system cells that can engulf and kill foreign organisms) Chediak Higashi Syndrome Chronic Granulomatous Disease Neutrophil Actin Deficiency Reticular Dysgenesis Cancers in the bone marrow (Plasma Cell Disorders) Multiple Myeloma Plasma Cell Leukemia Waldenstrom's Macroglobulinemia

6 Other cancers (Not originating in the blood system) Neuroblastoma Retinoblastoma next step... Therapies in Clinical Trials These are diseases for which stem cell treatments have been shown beneficial, but have not been adopted as standard therapy. For some of these diseases, stem cell transplants only slow the progression of the disease, but do not produce a cure. For other diseases, stem cell treatments may effect a cure, but the optimum dosage and usage of the stem cells is still under investigation. In the United States, patients with these diagnoses can usually only get access to a stem cell treatments if they are enrolled in a clinical trial. Patients seeking a trial for their situation should consult the first resource below, ClinicalTrials.gov. Recommended Resources: ClinicalTrials.gov Transplants for Cancerous Tumors NIH National Library of Medicine central resource for information about clinical trials. Search for a trial that is enrolling patients for a specific condition. Unfortunately, many clinical trials are not incuded in this list. National Marrow Donor Program (NMDP) has a Patient Resources page on learning about clinical trials which apply stem cell transplants. National Institute of Neurological Disorders and Stroke (NINDS) offers an index covering a long list of neurological diseases. National Organization for Rare Disorders (NORD) has compiled an index of rare diseases, which includes alternate disease names and disorder subdivisions. Breast Cancer Ewing's sarcoma Renal cell carcinoma

7 Transplants for Inherited Disorders effecting the Immune System & Other Organs Cartilage Hair Hypoplasia Gunther's Disease (Erythropoietic Porphyria) Hermansky Pudlak Syndrome Pearson's Syndrome Shwachman Diamond Syndrome Systemic Mastocytosis Transplants for Inherited Metabolic Disorders Mucopolysaccharidoses (MPS) Storage Diseases Mucopolysaccharidoses (MPS) Hurler's Syndrome (MPS IH) Scheie Syndrome (MPS IS) Hunter's Syndrome (MPS II) Sanfilippo Syndrome (MPS III) Morquio Syndrome (MPS IV) Maroteaux Lamy Syndrome (MPS VI) Sly Syndrome, Beta Glucuronidase Deficiency (MPS VII) Mucolipidosis II (I cell Disease) Leukodystrophy Disorders Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN) Krabbe Disease (Globoid Cell Leukodystrophy) Metachromatic Leukodystrophy Lysosomal Storage Diseases Gaucher Disease Niemann Pick Disease Sandhoff Disease Tay Sachs Disease Wolman Disease

8 Inherited Disorders Other Lesch Nyhan Syndrome Osteopetrosis Transplants for Disorders of Cell Proliferation Histiocytic Disorders Familial Erythrophagocytic Lymphohistiocytosis Hemophagocytosis Langerhans Cell Histiocytosis (LCH; formerly called Histiocytosis X) Transplants for diseases of the Central Nervous System Multiple Sclerosis (MS) Gene Therapy (ie: Transplanting genetically altered stem cells) Glanzmann Thrombasthenia Severe Combined Immunodeficiency (SCID) SCID with Adenosine Deaminase Deficiency (ADA SCID) SCID which is X linked Cellular Cardiomyoplasty (ie: Strengthening damaged heart muscle by infusing stem cells or promoting their growth) autologous stem cell transplantation drug enhanced stem cell proliferation next step...

9 Experimental Treatments These are diseases for which stem cell treatments have not been proven to have any efficacy in human beings. In a "Phase I" clinical trial, the purpose of the study is to find out if the therapy makes any difference in the course of the disease, as compared to a control group. Some doctors may also be giving stem cells to patients in experiments outside of clinical trials, but in the United States such experiments are subject to limitations imposed by FDA regulations. This category also covers experiments in the laboratory, either with cell cultures or animals. Auto Immune Diseases Arthritis, Juvenile Arthritis, Rheumatoid Crohn's Disease Diabetes, Type I Evan Syndrome Juvenile Dermatomyositis Scleroderma Lupus Gene Therapy (ie: Transplanting genetically altered stem cells) Fanconi's Anemia Metabolic Disorders (Leukodystrophy Diseases, Storage Disorders, etc.) Parkinson's Disease

10 Nerve cell repair Diseases of the Central Nervous System Amyotrophic Lateral Sclerosis (ALS, or "Lou Gehrig's disease") Alzheimer's Disease Huntington's Disease Parkinson's Disease Traumatic injury Spinal cord injury Stroke recovery Organ repair Kidney Combined transplant of kidney plus hematopoietic stem cells Growth of renal cells from hematopoietic stem cells Liver Growth of liver cells from hematopoietic stem cells Last modified: Monday, 20 Nov :29:41 EST Copyright Frances Verter