Diseases Treated with Stem Cells and its Potential Applications
What are the types of diseases and conditions that can be treated with Cord Blood Stem Cells and its 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 cord blood and other sources of similar type of stem cells (Haematopoietic Stem Cell), like bone marrow and peripheral blood. Stem cell therapies continue to change and evolve quickly.
DID YOU KNOW?
Haematopoietic stem cells or HSCs have been used to treat over 801 diseases. Cord blood is a rich source of HSCs.Blood Cancers
- Acute Biphenotypic Leukaemia
- Acute Myelogenous Leukaemia
- Acute Undifferentiated Leukaemia
- Chronic Myelogenous Leukaemia
- Chronic Lymphocytic Leukaemia
- Juvenile Myelomonocytic Leukaemia
- Juvenile Chronic Myelogenous Leukaemia
- B-cell Acute Lymphoblastic Leukaemia
- T-cell Acute Lymphoblastic Leukaemia
- Acute Myelofibrosis
- Agnogenic Myeloid Metaplasia
- 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
- Histiocytic Neoplasms
Solid Tumors
- Hodgkin Lymphoma
- Langerhans' Cell Histiocytosis
- Neuroblastoma
- Non Hodgkin Lymphoma (Burkitt’s Lymphoma)
- Retinoblastoma
- Medulloblastoma
- Wilms Tumor
Non Malignant Blood Disorders
- Aplastic Anaemia
- Congenital Dyserythropoietic Anaemia
- Fanconi’s Anaemia
- Paroxysmal Nocturnal Hemoglobinuria
- Diamond Blackfan Syndrome
- Dyskeratosis Congenita
- Pearson’s Syndrome
- Shwachman Diamond Syndrome
- Pure Red Cell Aplasia
- Sickle Cell Anaemia
- Beta Thalassemia Major/Cooley’s Anaemia
- Congenital Amegakaryocytosis Thrombocytopenia
- Glanzmann’s Thrombasthenia
Immune Disorders
- Bare Lymphocyte Syndrome
- Omenn Syndrome
- Reticular Dysgenesis
- Neutrophil Actin Deficiency
- 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
- Cartilage Hair Hypoplasia
- Gunther’s Disease (Congenital Erythropoietic Protoporphyria)
- Systemic Mastocytosis
- Common Variable Immunodeficiency
- DiGeorge Syndrome
- Evans Syndrome
- Hemophagocytic Lymphohistiocytosis
- IKK Gamma Deficiency (NEMO Deficiency)
- IPEX Syndrome
- Leukocyte Adhesion Deficiency
- Wiskott Aldrich Syndrome
- X linked Lymphoproliferative Disease (Duncan’s Syndrome)
- X linked Hyper IgM Syndrom
- Ataxia-Telangiectasia
Metabolic Disorders
- Adrenoleukodystrophy
- Krabbe Disease (Globoid Cell Leukodystrophy)
- Metachromatic leukodystrophy
- Pelizaeus-Merzbacher Disease
- Alpha Mannosidosis
- Gaucher’s 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
- Hermansky-Pudlak Syndrome
Banking 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. - 6 B-cell and T-cell acute lymphoblastic leukemia. 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 August 28, 2023.
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 are an alternative avenue for receiving new and promising therapies that would otherwise be unavailable. Patients with difficult-to-treat or ‘incurable’ diseases, such as HIV or certain types of cancer, may choose to participate in clinical research trials should standard therapies prove to be ineffective. Clinical research trials are sometimes lifesaving.
For the latest information, please visit www.clinicaltrials.gov
- Alzheimer’s Disease
- Amyotrophic Lateral Sclerosis
- Autism
- Brain Tumour
- Cardiomyopathy
- Cartilage repair
- Cerebral palsy
- Cleft Palate Repair (Alveolar)
- Compartment Syndrome (Battlefield Trauma)
- Critical Limb Ischemia
- Crohn's disease
- Diabetes Type 1
- Epidermolysis Bullosa
- Ewing Sarcoma
- Graft versus Host Disease (GvHD)
- Hearing Loss (acquired sensorineural)
- HIV
- Huntington’s Disease
- Hypoplastic Left Heart Syndrome
- Hypoxic Ischemic Encephalopathy (HIE)
- Ischemic Heart Disease
- Ischemic Stroke
- Kidney plus stem cell transplant
- Liver cirrhosis
- Lupus
- Multiple Sclerosis
- Myocardial Infarction
- Open cardiac surgery for congenital heart diseases
- Ovarian Cancer (Link to clinical trials)
- Parkinson’s Disease
- Rhabdomyosarcoma
- Rheumatoid Arthritis
- Scleroderma
- Spinal cord injury
- Testicular Tumour
- Tissue Engineered Vascular Grafts for cardiac defects
- Traumatic Brain Injury
*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:
- 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: QR 8.1-7-16, ver A
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