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.

DID YOU KNOW?

Haematopoietic Stem Cells (HSCs) can potentially be used to treat over 80 diseases1. 

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.

Blood Cancers

Blood Cancers

Leukaemia
  • 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
Myeloproliferative Neoplasms
  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (Myelofibrosis)
  • Essential Thrombocythemia
  • Polycythemia Vera
Myelodysplastic Syndromes
  • Refractory Anaemia
  • Refractory Anaemia with Excess Blasts
  • Refractory Anaemia with Excess Blasts in Transformation
  • Refractory Anaemia with Ringed Sideroblasts (sideroblastic Anaemia)
  • Chronic Myelomonocytic Leukaemia
Bone Marrow Cancers
  • Multiple Myeloma
  • Plasma Cell Leukaemia
  • Waldenstrom’s Macroglobulinemia
Non Malignant Blood Disorders

Non Malignant Blood Disorders

Anaemias (Deficiencies or Malformations of Red Cells)
  • Aplastic Anaemia
  • Congenital Dyserythropoietic Anaemia
  • Fanconi’s Anaemia
  • Paroxysmal Nocturnal Hemoglobinuria
Inherited Red Cell Abnormalities
  • Pure Red Cell Aplasia
  • Sickle Cell Anaemia
  • Beta Thalassemia Major / Cooley’s Anaemia
  • Diamond Blackfan Anaemia
Inherited Platelet Abnormalities
  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Glanzmann’s Thrombasthenia
Immune Disorders

Immune Disorders

Severe Combined Immune Deficiencies (SCID)
  • Omenn Syndrome
  • SCID with Adenosine Deaminase Deficiency (ADA SCID)
  • X-linked SCID
  • SCID with absence of T & B Cells
  • SCID with absence of T cells, Normal B Cells
Inherited Disorders of the Immune System & Other Organs
  • Cartilage Hair Hypoplasia
  • Erythropoietic Porphyria
  • Hermansky-Pudlak Syndrome
  • Pearson’s Syndrome
  • Shwachman-Diamond Syndrome
  • Systemic Mastocytosis
Phagocyte Disorders
  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Neutrophil Actin Deficiency
  • Reticular Dysgenesis
Neutropenias
  • Kostmann Syndrome (Infantile Genetic Agranulocytosis)
  • Myelokathexis
Other Inherited Immune System Disorders
  • Bare Lymphocyte Syndrome
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Hemophagocytic Lymphohistiocytosis
  • IKK Gamma Deficiency4,6 (NEMO deficiency)
  • IPEX Syndrome3,5
  • Leukocyte Adhesion Deficiency
  • Wiskott-Aldrich Syndrome
  • Lymphoproliferative Disorders
  • X-linked Lymphoproliferative Disease (Duncan’s Syndrome)
  • Ataxia-Telangiectasia
Metabolic Disorders

Metabolic Disorders

Leukodystrophy Disorders
  • Adrenoleukodystrophy
  • Krabbe Disease (Globoid Cell Leukodystrophy)
  • Metachromatic Leukodystrophy
  • Pelizaeus-Merzbacher Disease
Lysosomal Storage Diseases
  • Niemann-Pick Disease
  • Sandhoff Disease
  • Wolman Disease
Mucopolysaccharidosis (MPS) Storage Diseases
  • Hunter Syndrome
  • Hurler Syndrome
  • Maroteaux-Lamy Syndrome
  • Mucolipidosis II (I-cell Disease)
  • Morquio Syndrome
  • Sanfilippo Syndrome
  • Scheie Syndrome
  • Sly Syndrome (Beta-glucuronidase Deficiency)
Solid Tumors

Solid Tumors

Tumors
  • Hodgkin Lymphoma
  • Neuroblastoma
  • Non-Hodgkin Lymphoma (Burkitt’s Lymphoma)
  • Retinoblastoma
  • Medulloblastoma
Other Metabolic Disorders

Other Metabolic Disorders

Other Metabolic Disorders
  • Lesch–Nyhan Syndrome
  • Osteopetrosis

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 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 December 5, 2022.
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.
6Tono 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.

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
Auto-Immune Disorders
  • 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
Genetic or Metabolic Disorder
Genetic and/or Metabolic Disorder
  • 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
Cardiovascular
Cardiovascular
  • 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
Orthopedic
Orthopedic
  • Ankylosing Spondylitis
  • Cartilage Injury
  • Cleft Palate Repair
  • Non-Union Fractures
  • Osteoarthritis
  • Osteochondral Lesion
  • Spinal Fusion Surgery
Diabetic
Diabetic
  • Diabetes, Type 1 (Auto-Immune)
  • Diabetes, Type 2
  • Diabetic Foot Ulcer
  • Diabetic Peripheral Neuropathy
Neurologic Disorder
Neurologic Disorder
  • 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
Others
  • 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

*The clinical trials listed above may use cell lines other than Haematopoietic Stem Cells, such as Mesenchymal Stem Cells.

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. 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. 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. 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. 4784, Version J, December 2022 (reference from QR 8.1-8-5-i)