Diseases Treated with Stem Cells and their Potential Applications
What are the types of diseases and conditions that can be treated with Cord Blood Stem Cells and their potential applications?
Stem cells are at the forefront of one of the most fascinating and revolutionary areas of medicine today. Doctors recognise that stem cells have the potential to help treat numerous diseases by generating healthy new cells and tissue.
As a parent, you want to protect your family. At your baby’s birth, you have the unique opportunity to safeguard the health of the ones you love by storing his/her precious cord blood stem cells.
Stem cells in your baby’s cord blood have the potential to be used in the treatment of many diseases today. Stem cells could be used to treat haematopoietic and genetic disorders. In a cord blood transplant, stem cells are infused into a patient’s bloodstream where they go to work - healing and repairing damaged cells and tissue. Upon successful engraftment of the stem cells, the patient’s blood and immune system are regenerated.
There are a wide range of diseases that are treatable with stem cells derived from cord blood and other sources of similar type of stem cells (Haematopoietic Stem Cell), like bone marrow and peripheral blood, including stem cell disorders, acute and chronic forms of leukaemia, myeloproliferative disorders, and many more.
In addition to the host of conditions that can now be treated, it is the potential of stem cell treatments that holds the most excitement as research continues to uncover new possibilities. The potential and efficacy of treating diseases with stem cells are real.
Diseases Treated with Stem Cells
The following is a list of some of the diseases that have been treated with Haematopoietic Stem Cell.
DID YOU KNOW?
Umbilical cord blood stem cells can be used to treat more than 801 diseases.Blood Cancers
- Acute Biphenotypic Leukaemia
- Acute Lymphoblastic Leukaemia
- Acute Myelogenous Leukaemia
- Acute Undifferentiated Leukaemia
- Chronic Myelogenous Leukaemia
- Chronic Lymphocytic Leukaemia
- Juvenile Myelomonocytic Leukaemia
- Juvenile Chronic Myelogenous Leukaemia
- Acute Myelofibrosis
- Agnogenic Myeloid Metaplasia (Myelofibrosis)
- Essential Thrombocythemia
- Polycythemia Vera
- Refractory Anaemia
- Refractory Anaemia with Excess Blasts
- Refractory Anaemia with Excess Blasts in Transformation
- Refractory Anaemia with Ringed Sideroblasts (Sideroblastic Anaemia)
- Chronic Myelomonocytic Leukaemia
- Multiple Myeloma
- Plasma Cell Leukaemia
- Waldenstrom’s Macroglobulinemia
Solid Tumors
- Hodgkin Lymphoma
- Neuroblastoma
- Non Hodgkin Lymphoma (Burkitt’s Lymphoma)
- Retinoblastoma
- Medulloblastoma
Non Malignant Blood Disorders
- Aplastic Anaemia
- Congenital Dyserythropoietic Anaemia
- Fanconi’s Anaemia
- Paroxysmal Nocturnal Hemoglobinuria
- Pure Red Cell Aplasia
- Sickle Cell Anaemia
- Beta Thalassemia Major/Cooley’s Anaemia
- Diamond Blackfan Anaemia
- Amegakaryocytosis / Congenital Thrombocytopenia
- Glanzmann’s Thrombasthenia
Immune Disorders
- Omenn Syndrome
- 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
- Kostmann Syndrome (Infantile Genetic Agranulocytosis)
- Myelokathexis
- Chediak Higashi Syndrome
- Chronic Granulomatous Disease
- Neutrophil Actin Deficiency
- Reticular Dysgenesis
- Cartilage Hair Hypoplasia
- Erythropoietic Porphyria
- Hermansky Pudlak Syndrome
- Pearson’s Syndrome
- Shwachman Diamond Syndrome
- Systemic Mastocytosis
- Bare Lymphocyte Syndrome
- Common Variable Immunodeficiency
- DiGeorge Syndrome
- Hemophagocytic Lymphohistiocytosis
- IKK Gamma Deficiency3 (NEMO Dificiency)
- IPEX Syndrome4
- Leukocyte Adhesion Deficiency
- Wiskott Aldrich Syndrome
- Lymphoproliferative Disorders
- X linked Lymphoproliferative Disease (Duncan’s Syndrome)
- Ataxia-Telangiectasia
Metabolic Disorders
- Adrenoleukodystrophy
- Krabbe Disease (Globoid Cell Leukodystrophy)
- Metachromatic leukodystrophy
- Pelizaeus-Merzbacher Disease
- Niemann Pick Disease
- Sandhoff Disease
- Wolman Disease
- Hunter Syndrome
- Hurler Syndrome
- Maroteaux Lamy Syndrome
- Mucolipidosis II (I-cell Disease)
- Morquio Syndrome
- Sanfilippo Syndrome
- Scheie Syndrome
- Sly Syndrome (beta glucuronidase deficiency)
Other Metabolic Disorders
- Lesch–Nyhan Syndrome
- Osteopetrosis
Storing cord blood does not guarantee that the cells will provide a cure or be applicable in every situation. Use will be ultimately determined by the treating physician.
References:
- 1 Diseases treated page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases. Accessed April 23, 2021.
- 2 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.
- 3 Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) Syndrome. Stanford Children’s Health website. https://www.stanfordchildrens.org/en/service/stem-cell-transplantation/conditions/ipex-syndrome. Accessed April 5, 2021.
- 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 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.
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 Transplantation. 2007;39(12):801-804. doi:10.1038/sj.bmt.1705658.
Clinical Trials
With advancements in stem cell research, the list of ongoing clinical trials; one of the key driving forces behind medical
breakthroughs continue to grow, representing hope for families with conditions that currently have no known cure.
Depending on the disease, stem cell* treatments that are currently undergoing clinical trials may slow the progression of the
disease, unlock new medical options or help effect a cure. Patients with “incurable” diseases, such as AIDS and certain types of cancer, may want to pursue participation in clinical research trials as an alternative option.
Below are some of the diseases1 that are currently undergoing clinical trials. For the full list, please visit clinicaltrials.gov
- Alopecia Areata
- Amyotrophic Lateral Sclerosis (ALS)
- Crohn’s Disease
- Eczema (Atopic Dermatitis)
- Graft-versus-Host Disease (GvHD)
- Lupus
- Multiple Sclerosis
- Psoriasis
- Rheumatoid Arthritis
- Systemic Sclerosis
- Ulcerative Colitis
- Ankylosing Spondylitis
- Cartilage Injury
- Cleft Palate Repair
- Non-Union Fractures
- Osteoarthritis
- Osteochondral Lesion
- Spinal Fusion Surgery
- Acute Myocardial Infarction (Heart Attack)
- Cardiomyopathy
- Critical Limb Ischemia (CLI)
- Heart Failure
- Hypoplastic Left Heart Syndrome2
- Ischemic Heart Disease3
- Peripheral Arterial Disease (PAD)
- Surgery for Congenital Heart Defects
- Alzheimer’s Disease
- Autism
- Cerebral Palsy
- Encephalopathy
- Global Developmental Delay
- Hearing Loss (acquired sensorineural)
- Intraventricular Hemorrhage
- Parkinson’s Disease
- Spinal Cord Injury
- Stroke
- Traumatic Brain Injury
- Diabetes, Type 1 (Auto-Immune)
- Diabetes, Type 2
- Diabetic Foot Ulcer
- Diabetic Peripheral Neuropathy
- Acute Respiratory Distress Syndrome (ARDS)
- Bronchopulmonary Dysplasia (BPD) (lung disorder due to premature birth)
- COVID-195
- Erectile Dysfunction
- Eye Diseases
- Fistula
- HIV
- Kidney Failure
- Liver Cirrhosis
- Liver Failure
- Ovarian Cancer6
- Peyronie’s Disease
- Premature Ovarian Failure
- Testicular Tumour7
- Uterine Scars
- Wounds
- Aging Frailty
- Duchenne Muscular Dystrophy
- Epidermolysis Bullosa
- Gaucher’s Disease4
- Hereditary Ataxia
- Lysosomal Storage Diseases
- Metabolic Syndrome
- Severe Combined Immunodeficiency
- Spinal muscular atrophy
- Tay-Sachs Disease
*Stem cells mentioned here comprises of other cell lines such as Mesenchymal stem cells. The clinical trials listed above may be using other lines of stem cells, and not only haematopoietic stem cells.
References:
- 1 Therapies in Clinical Trials Page. Parent’s Guide to Cord Blood Foundation website. https://parentsguidecordblood.org/en/diseases#trial. Accessed June 14, 2021.
- 2 ClinicalTrials.org registry… NCT01883076, NCT03431480, NCT03779711. Last accessed 5 April, 2021.
- 3 Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Ischemic Cardiomyopathy. ClinicalTrials.gov website. https://clinicaltrials.gov/ct2/show/NCT01946048?term=cord%2Bblood&cond=Ischemic%2BHeart%2BDisease&draw=2&rank=2. Accessed April 5, 2021.
- 4 ClinicalTrials.org registry… NCT00176904, NCT04528355. Last accessed 5 April, 2021.
- 5 Mesenchymal Stem Cells and COVID-19: Cure, Prevention, and Vaccination. Hindawi.com website. https://www.hindawi.com/journals/sci/2021/6666370/. Accessed June 14, 2021.
- 6 Cady C., McAsey M., Li J. Progress towards a Stem Cell Based Therapy for Ovarian Cancer – Proceeding of the STEMSO Conference. https://www.longdom.org/open-access/progress-towards-astem-cell-based-therapy-for-ovarian-cancer.pdf. Accessed June 2, 2021.
- 7 High-Dose Chemo & Stem Cell Transplant for Testicular Cancer. American Cancer Society website. https://www.cancer.org/cancer/testicular-cancer/treating/high-dose-chemo-stem-cell.html. Published May 17, 2018. Accessed June 6, 2021.
DCR No. 4383, Version I, QR 8.1-8-5-i. September 2021