.diseases-sub-title{ font-weight: bold; margin-top: 10px; } Over 80 diseases 1 potentially treatable with cord blood stem cells Stem cells are now at the forefront of one of the most fascinating and revolutionary fields in medicine. Blood-forming stem cells, also called Haematopoietic Stem Cells (HSCs), can be found in your baby's cord blood. These cells have the potential to treat more than 80 diseases 1, including some types of cancer like leukaemia, neuroblastoma, and lymphoma. During a stem cell transplant, stem cells are infused into the patient’s bloodstream, where they start to heal damaged cells and tissues. The patient’s blood and immune system are regenerated after the stem cells successfully engraft. DID YOU KNOW? Haematopoietic Stem Cells (HSCs) can potentially be used to treat over 80 diseases1. List of diseases potentially treatable with cord blood stem cells The following diseases can potentially be treated with cord blood stem cell transplants. These cells can be derived from cord blood, bone marrow, or peripheral blood. Blood Cancers Leukaemia STANDARD THERAPIES Allogeneic Autologous Acute Biphenotypic Leukaemia X B-cell Acute Lymphoblastic Leukaemia 2 X T-cell Acute Lymphoblastic Leukaemia 2 X Acute Myelogenous Leukaemia X Acute Undifferentiated Leukaemia X Chronic Myelogenous Leukaemia X Chronic Lymphocytic Leukaemia X Juvenile Myelomonocytic Leukaemia X Juvenile Chronic Myelogenous Leukaemia X Myeloproliferative Neoplasms STANDARD THERAPIES Allogeneic Autologous Acute Myelofibrosis X Agnogenic Myeloid Metaplasia (Myelofibrosis) X Essential Thrombocythemia X Polycythemia Vera X Myelodysplastic Syndromes STANDARD THERAPIES Allogeneic Autologous Refractory Anaemia X Refractory Anaemia with Excess Blasts X Refractory Anaemia with Excess Blasts in Transformation X Refractory Anaemia with Ringed Sideroblasts (Sideroblastic Anaemia) X Chronic Myelomonocytic Leukaemia X Bone Marrow Cancers STANDARD THERAPIES Allogeneic Autologous Multiple Myeloma X X Plasma Cell Leukaemia X X Waldenstrom’s Macroglobulinemia X X Non Malignant Blood Disorders Anaemias (Deficiencies or Malformations of Red Cells) STANDARD THERAPIES Allogeneic Autologous Aplastic Anaemia X Congenital Dyserythropoietic Anaemia X Fanconi Anaemia 3 X Paroxysmal Nocturnal Hemoglobinuria X Inherited Red Cell Abnormalities STANDARD THERAPIES Allogeneic Autologous Pure Red Cell Aplasia X Sickle Cell Anaemia X Beta Thalassemia Major / Cooley’s Anaemia X Diamond Blackfan Anaemia X Inherited Platelet Abnormalities STANDARD THERAPIES Allogeneic Autologous Amegakaryocytosis / Congenital Thrombocytopenia X Glanzmann’s Thrombasthenia X Immune Disorders Severe Combined Immune Deficiencies (SCID) STANDARD THERAPIES Allogeneic Autologous Omenn Syndrome X 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 Inherited Disorders of the Immune System & Other Organs STANDARD THERAPIES Allogeneic Autologous Cartilage Hair Hypoplasia X Erythropoietic Porphyria X Hermansky Pudlak Syndrome X Pearson’s Syndrome X Shwachman Diamond Syndrome X Systemic Mastocytosis X Phagocyte Disorders STANDARD THERAPIES Allogeneic Autologous Chediak Higashi Syndrome X Chronic Granulomatous Disease X Neutrophil Actin Deficiency X Reticular Dysgenesis X Neutropenias STANDARD THERAPIES Allogeneic Autologous Kostmann Syndrome (Infantile Genetic Agranulocytosis) X Myelokathexis X Other Inherited Immune System Disorders STANDARD THERAPIES Allogeneic Autologous Bare Lymphocyte Syndrome X Common Variable Immunodeficiency X DiGeorge Syndrome X Hemophagocytic Lymphohistiocytosis X IKK Gamma Deficiency 4,5 (NEMO Dificiency) X IPEX Syndrome 6,7 X Leukocyte Adhesion Deficiency X Wiskott Aldrich Syndrome X Lymphoproliferative Disorders X X linked Lymphoproliferative Disease (Duncan’s Syndrome) X Ataxia-Telangiectasia X Metabolic Disorders Leukodystrophy Disorders STANDARD THERAPIES Allogeneic Autologous Adrenoleukodystrophy (ALD) X Krabbe Disease (Globoid Cell Leukodystrophy) X Metachromatic Leukodystrophy X Pelizaeus-Merzbacher Disease X Lysosomal Storage Diseases STANDARD THERAPIES Allogeneic Autologous Niemann Pick Disease X Sandhoff Disease X Wolman Disease X Mucopolysaccharidosis (MPS) Storage Diseases STANDARD THERAPIES Allogeneic Autologous Hunter Syndrome X Hurler Syndrome X Maroteaux Lamy Syndrome X Mucolipidosis II (I-cell Disease) X Morquio Syndrome X Sanfilippo Syndrome X Scheie Syndrome X Sly Syndrome (beta-glucuronidase deficiency) X Other Metabolic Disorders STANDARD THERAPIES Allogeneic Autologous Lesch–Nyhan Syndrome X Osteopetrosis X Solid Tumors Tumors STANDARD THERAPIES Allogeneic Autologous Hodgkin's Lymphoma X X Neuroblastoma X Non Hodgkin's Lymphoma (Burkitt’s Lymphoma) X Retinoblastoma X Medulloblastoma X Storing cord blood does not guarantee that the cells will provide a cure or be useful in all situations. The treating physician will ultimately decide if cord blood can be used. References: 1 Diseases treated page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases. Accessed December 5, 2022. 2 B-cell and T-cell acute lymphoblastic leukemia page. Mayo Clinic Laboratories website. https://news.mayocliniclabs.com/hematology/lymphoma/b-cell-and-t-cell-acute-lymphoblastic-leukemia/#:~:text=Acute%20lymphoblastic%20leukemia%20accounts%20for,2%E2%80%935%20years%20of%20age. Accessed June 28, 2023. 3 Bizzetto R, Bonfim C, Rocha V, et al. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica. 2011; 96(1):134-141. 4 Picard C, J-L Casanova, Puel A, Infectious diseases in patients with IRAK-4, MyD88, NEMO, or IκBα Deficiency. Clin. Microbiol. Rev. 2011; 24(3):490-497. 5 Tono C, Takahashi Y, Terui K, et al. Correction of immunodeficiency associated with NEMO mutation by umbilical cord blood transplantation using a reduced-intensity conditioning regimen. Bone Marrow Transplant. 2007;39(12):801-804. 6 Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) Syndrome page. Stanford Children’s Health website. https://www.stanfordchildrens.org/en/service/stem-cell-transplantation/conditions/ipex-syndrome. Accessed December 5, 2022. 7 Rao A, Kamani N, Filipovich A, et al. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007; 109(1):383-385. Clinical trials Below are some of the diseases1 that are currently undergoing clinical trials using blood-forming cells or cord blood. As researchers continue to push the boundaries of stem cells for some of the most common life-threatening diseases, saving your baby's cord blood now will give your child access to her own stem cells once these therapies are available. For the complete and most updated list, please visit clinicaltrials.gov. Auto-Immune Disorders STANDARD THERAPIES Allogeneic Autologous Alopecia Areata X Amyotrophic Lateral Sclerosis (ALS) X Crohn’s Disease X Eczema (Atopic Dermatitis) X Graft-versus-Host Disease (GvHD) X Lupus X Multiple Sclerosis X Psoriasis X Rheumatoid Arthritis X Systemic Sclerosis X Ulcerative Colitis X Genetic and/or Metabolic Disorder STANDARD THERAPIES Allogeneic Autologous Aging Frailty X Duchenne Muscular Dystrophy X Epidermolysis Bullosa X Gaucher’s Disease4 X Hereditary Ataxia X Lysosomal Storage Diseases X Metabolic Syndrome X Severe Combined Immunodeficiency X Spinal muscular atrophy X Tay-Sachs Disease X Cardiovascular STANDARD THERAPIES Allogeneic Autologous Acute Myocardial Infarction (Heart Attack) X Cardiomyopathy X Critical Limb Ischemia (CLI) X Heart Failure X Hypoplastic Left Heart Syndrome2 X Ischemic Heart Disease3 X Peripheral Arterial Disease (PAD) X Surgery for Congenital Heart Defects X Orthopedic STANDARD THERAPIES Allogeneic Autologous Ankylosing Spondylitis X Cartilage Injury X Cleft Palate Repair X Non-Union Fractures X Osteoarthritis X Osteochondral Lesion X Spinal Fusion Surgery X Diabetic STANDARD THERAPIES Allogeneic Autologous Diabetes, Type 1 (Auto-Immune) X X Diabetes, Type 2 X X Diabetic Foot Ulcer X Diabetic Peripheral Neuropathy X Neurologic Disorder STANDARD THERAPIES Allogeneic Autologous Alzheimer’s Disease X Autism X X Cerebral Palsy X X Encephalopathy X Global Developmental Delay X X Hearing Loss (acquired sensorineural) X Intraventricular Hemorrhage X X Parkinson’s Disease X Spinal Cord Injury X Stroke X X Traumatic Brain Injury X X Others STANDARD THERAPIES Allogeneic Autologous Acute Respiratory Distress Syndrome (ARDS) X Bronchopulmonary Dysplasia (BPD) (lung disorder due to premature birth) X COVID-195 X X Erectile Dysfunction X Eye Diseases X Fistula X HIV X Kidney Failure X Liver Cirrhosis X Liver Failure X Ovarian Cancer6 X Peyronie’s Disease X Premature Ovarian Failure X Testicular Tumour7 X Uterine Scars X Wounds X *The clinical trials listed above may use cell lines other than Haematopoietic Stem Cells, such as Mesenchymal Stem Cells. A majority of autologous infusions/regenerative applications listed above were performed under clinical trial. References: 1 Therapies in clinical trials page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases#trial. Accessed December 5, 2022. 2 ClinicalTrials.org registry… NCT01883076, NCT03431480, NCT03779711. Last accessed December 5, 2022. 3 Umbilical cord derived mesenchymal stem cell therapy in ischemic cardiomyopathy page. ClinicalTrials.gov website. https://clinicaltrials.gov/ct2/show/NCT01946048. Accessed December 5, 2022. 4 ClinicalTrials.org registry… NCT00176904, NCT04528355. Last accessed December 5, 2022. 5 Mesenchymal stem cells and COVID-19: Cure, prevention, and vaccination page. Hindawi.com website. https://www.hindawi.com/journals/sci/2021/6666370/. Accessed December 5, 2022. 6 Cady C., McAsey M., Li J. Progress towards a stem cell based therapy for ovarian cancer – Proceeding of the STEMSO Conference. https://www.cellr4.org/article/780. Accessed December 5, 2022. 7 High-dose chemo & stem cell transplant for testicular cancer page. American Cancer Society website. https://www.cancer.org/cancer/testicular-cancer/treating/high-dose-chemo-stem-cell.html. Published May 17, 2018. Accessed December 5, 2022. .DCR No. 4990, Version K, September 2023 (reference from QR 8.1-8-5-J)
