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
| 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 |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Acute Myelofibrosis | X | |
| Agnogenic Myeloid Metaplasia (Myelofibrosis) | X | |
| Essential Thrombocythemia | X | |
| Polycythemia Vera | X |
| 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 |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Multiple Myeloma | X | X |
| Plasma Cell Leukaemia | X | X |
| Waldenstrom’s Macroglobulinemia | X | X |
Non Malignant Blood Disorders
| STANDARD THERAPIES | Allogeneic | Autologous |
| Aplastic Anaemia | X | |
| Congenital Dyserythropoietic Anaemia | X | |
| Fanconi Anaemia 3 | X | |
| Paroxysmal Nocturnal Hemoglobinuria | X |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Pure Red Cell Aplasia | X | |
| Sickle Cell Anaemia | X | |
| Beta Thalassemia Major / Cooley’s Anaemia | X | |
| Diamond Blackfan Anaemia | X |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Amegakaryocytosis / Congenital Thrombocytopenia | X | |
| Glanzmann’s Thrombasthenia | X |
Immune Disorders
| 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 |
| 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 |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Chediak Higashi Syndrome | X | |
| Chronic Granulomatous Disease | X | |
| Neutrophil Actin Deficiency | X | |
| Reticular Dysgenesis | X |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Kostmann Syndrome (Infantile Genetic Agranulocytosis) | X | |
| Myelokathexis | X |
| 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
| STANDARD THERAPIES | Allogeneic | Autologous |
| Adrenoleukodystrophy (ALD) | X | |
| Krabbe Disease (Globoid Cell Leukodystrophy) | X | |
| Metachromatic Leukodystrophy | X | |
| Pelizaeus-Merzbacher Disease | X |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Niemann Pick Disease | X | |
| Sandhoff Disease | X | |
| Wolman Disease | X |
| 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 |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Lesch–Nyhan Syndrome | X | |
| Osteopetrosis | X |
Solid 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.
| 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 |
| 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 |
| 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 |
| 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 |
| STANDARD THERAPIES | Allogeneic | Autologous |
| Diabetes, Type 1 (Auto-Immune) | X | X |
| Diabetes, Type 2 | X | X |
| Diabetic Foot Ulcer | X | |
| Diabetic Peripheral Neuropathy | X |
| 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 |
| 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)