Diseases Treated with Stem Cells and Its Potential Applications

www.cordlife.com stem cells (Haematopoietic StemCell),stem cells(Haematopoietic andperipheralblood. suchasbonemarrow withcordbloodandothersources ofsimilartype beentreated have isalistofsomethediseasesthat The following Diseases Treated withStemCells Potential Applications Diseases Treated withStemCellsandits BLOOD CANCERS Neuroblastoma* Langerhans’ CellHistiocytosis* Hodgkin Lymphoma* SOLID TUMORS Acute Lymphoblastic Leukaemia* Acute BiphenotypicLeukaemia* LEUKAEMIA Wilms Tumor Medulloblastoma Retinoblastoma Non HodgkinLymphoma Multiple Myeloma OTHER BLOODCANCERS Chronic Myelomonocytic Leukaemia* with Anaemia Refractory Refractory Anaemia withExcess with Anaemia Refractory Anaemia* Refractory MYELODYSPLASTIC SYNDROMES Polycythemia Vera Essential Thrombocythemia Agnogenic MyeloidMetaplasia Acute Myelofibrosis MYELOPROLIFERATIVE NEOPLASMS Juvenile ChronicMyelogenous Juvenile Myelomonocytic Leukaemia* Chronic Lymphocytic Leukaemia* Chronic MyelogenousLeukaemia* Leukaemia* Acute Undifferentiated Acute MyelogenousLeukaemia* Plasma CellLeukaemia Waldenstrom’s Macroglobulinemia Histiocytic Neoplasms STANDARD THERAPIES (Burkitt’s Lymphoma)* Anaemia)* Ringed Sideroblasts(Sideroblastic in Transformation*Blasts Excess Blasts* Leukaemia Allogeneic x x x x x x x x x x x x x x x x x x x x x x x x Autologous x x x x x x x x x DISORDERS NON MALIGNANTBLOOD Diamond BlackfanSyndrome* FAILURE SYNDROMES HEREDITARY BONEMARROW Paroxysmal Nocturnal Hemoglobinuria Fanconi’s Anaemia* Anaemia Dyserythropoietic Congenital Anaemia* Aplastic MALFORMATIONS OFREDCELLS) ANAEMIAS (DEFICIENCIESOR SCID withabsenceof T Cells, SCID withabsenceof T &BCells* SCID whichisXlinked* SCID with Adenosine Deaminase Deficiency Neutrophil Actin Reticular Dysgenesis Omenn Syndrome* Bare Lymphocyte Syndrome* DEFICIENCY (SCID) SEVERE COMBINEDIMMUNE IMMUNE DISORDERS Dyskeratosis Congenita* Dyskeratosis Shwachman DiamondSyndrome* Pearson’s Syndrome Glanzmann’s Thrombasthenia* Thrombocytopenia Congenital Amegakaryocytosis PLATELETINHERITED ABNORMALITIES Cooley’s Anaemia Beta Thalassemia Major/ Cell Anaemia* Sickle Pure RedCell Aplasia ABNORMALITIES INHERITED REDCELL STANDARD THERAPIES Normal BCells* Deficiency (ADASCID)* 24-Hour Hotline 62380808 Allogeneic x x x x x x x x x x x x x x x x x x x x x Autologous x x x 1 DCR no. 2770 Version E, QR 8.1‐8‐5‐e, July 2016 2 www.cordlife.com Pelizaeus-Merzbacher Disease leukodystrophy* Metachromatic Krabbe Disease Adrenoleukodystrophy* LEUKODYSTROPHY DISORDERS METABOLIC DISORDERS Ataxia-Telangiectasia X linkedHyperIgMSyndrome X linkedLymphoproliferative Disease Syndrome* Wiskott Aldrich Deficiency*Leukocyte Adhesion IPEX Syndrome* IKK GammaDeficiency Hemophagocytic Lymphohistiocytosis Evans Syndrome* DiGeorge Syndrome* Immunodeficiency*Common Variable SYSTEM DISORDERS OTHER INHERITEDIMMUNE Systemic Mastocytosis* Gunther’s Disease(Congenital HairHypoplasia Cartilage IMMUNE SYSTEM &OTHER ORGANS INHERITED DISORDERSOF THE Disease* Chronic Granulomatous Chediak HigashiSyndrome* PHAGOCYTE DISORDERS Myelokathexis* Kostmann Syndrome NEUTROPENIAS STANDARD THERAPIES (Globoid CellLeukodystrophy)* (Duncan’s Syndrome)* (NEMO Deficiency)* Protoporphyria)* Erythropoietic Genetic Agranulocytosis)* (Infantile Allogeneic x x x x x x x x x x x x x x x x x x x x x x Autologous physician. bedeterminedbythetreating stem cellswillultimately situation. inevery provide acureorbeapplicable The useof thecellswill Banking cordblooddoesnotguaranteethat Hermansky-Pudlak Syndrome Osteopetrosis* Lesch–Nyhan Syndrome* OTHER METABOLIC DISORDERS Sly Syndrome(betaglucuronidase Scheie Syndrome* Sanfilippo Syndrome* Morquio Syndrome Mucolipidosis II(I-cellDisease)* Maroteaux LamySyndrome* Hurler Syndrome* Hunter Syndrome* STORAGE DISEASES MUCOPOLYSACCHARIDOSIS (MPS) Wolman Disease* Sandhoff Disease Niemann PickDisease Gaucher’s Disease Alpha Mannosidosis* LYSOSOMAL STORAGE DISEASES STANDARD THERAPIES deficiency) Diseases Treated withStemCellsandits 24-Hour Hotline 62380808 Potential Applications (cont’d) Potential Applications Allogeneic x x x x x x x x x x x x x x x x Autologous

DCR no. 2770 Version E, QR 8.1‐8‐5‐e, July 2016 Clinical Trials With the advancement of stem cell+ research, the potential for future use of stem cell grows. Below is a list of diseases currently under Clinical Trials. These are diseases for which stem cell+ treatments appear to be 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 help effect a cure, but further research is needed to determine the best candidate patients for stem cell therapy, the optimum stem cell dosage, the optimum method of cell delivery, etc.

For some patients, clinical research trials represent an avenue for receiving promising new therapies that would not otherwise be available. Patients with difficult to treat or currently “incurable” diseases, such as AIDS or certain types of cancer, may want to pursue participation in clinical research trials if standard therapies are not effective. Clinical research trials are sometimes lifesaving.

CLINICAL TRIALS Allogeneic Autologous Alzheimer’s Disease* x Amyotrophic Lateral Sclerosis x x Autism* x Brain Tumour x Cardiomyopathy x x Cartilage repair x x Cerebral palsy* x Cleft Palate Repair (Alveolar) x Compartment Syndrome (Battlefield Trauma) x Critical Limb Ischemia x x Crohn's disease x x Diabetes Type 1* x x Epidermolysis Bullosa* x Ewing Sarcoma x Graft versus Host Disease (GvHD) x Hearing Loss (acquired sensorineural)* x HIV* x Huntington’s Disease x Hypoplastic Left Heart Syndrome* x Hypoxic Ischemic Encephalopathy (HIE)* x Ischemic Heart Disease x x Ischemic Stroke x Kidney plus stem cell transplant x Liver cirrhosis* x Lupus x Multiple Sclerosis x Myocardial Infarction x Open cardiac surgery for congenital heart diseases x Ovarian Cancer (Link to clinical trials) x Parkinson’s Disease x Rhabdomyosarcoma x Rheumatoid Arthritis x Scleroderma x x Spinal cord injury* x x Testicular Tumour x Tissue Engineered Vascular Grafts for cardiac defects x Traumatic Brain Injury x *Treated with Cord Blood +Stem cells mentioned here comprises of other cell lines such as Mesenchymal stem cells. The clinical trials and experimental treatments listed above may be using other lines of stem cells, and not only haematopoietic stem cells. References: • Diseases treated page. Parent’s Guide to Cord Blood Foundation. http://parentsguidecordblood.org/diseases.php. Accessed Dec 2015. • Stem cell treatment page. Cord Blood Registry Website. http://www.cordblood.com/benefits-cord-blood/umbilical-cord-stem-cells/stem-cell-treatments. Accessed Dec 2015.

• Clinical trial registry page. ClinicalTrials.gov. http://clinicaltrials.gov. Accessed Dec 2015. July 2016 QR 8.1‐8‐5‐e, E, Version 2770 DCR no.