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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 Haematopoietic Stem Cell.

DID YOU KNOW?

Haematopoietic stem cells or HSCs have been used to treat over 801diseases. Cord blood is a rich source of HSCs.

Blood Cancers

Leukaemia
  • Acute Biphenotypic Leukaemia
  • Acute Lymphoblastic Leukaemia
  • Acute Myelogenous Leukaemia
  • Acute Undifferentiated Leukaemia
  • Chronic Lymphocytic Leukaemia
  • Juvenile Chronic Myelogenous Leukaemia
  • Acute Myelofibrosis
Myeloproliferative Neoplasms
  • Agnogenic Myeloid Metaplasia
  • Essential Thrombocythemia
  • Polycythemia Vera
  • Chronic Myelogenous Leukaemia
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
  • Juvenile Myelomonocytic Leukaemia
Other Blood Cancers
  • Multiple Myeloma
  • Plasma Cell Leukaemia
  • Waldenstrom’s Macroglobulinemia

Solid Tumors

Tumors
  • Hodgkin Lymphoma
  • Neuroblastoma
  • Non Hodgkin Lymphoma (Burkitt’s Lymphoma)
  • Retinoblastoma
  • Medulloblastoma

Non Malignant Blood Disorders

Anaemias (Deficiences or malformations of red cells)
  • Aplastic Anaemia
  • Congenital Dyserythropoietic Anaemia
  • Fanconi’s Anaemia
  • Paroxysmal Nocturnal Hemoglobinuria
Hereditary Bone Marrow Failure Syndromes
  • Diamond Blackfan Syndrome
  • Dyskeratosis Congenita2
  • Pearson’s Syndrome
  • Shwachman Diamond Syndrome
Inherited Red Cell Abnormalities
  • Pure Red Cell Aplasia
  • Sickle Cell Anaemia
  • Beta Thalassemia Major/Cooley’s Anaemia
Inherited Platelet Abnormalities
  • Congenital Amegakaryocytosis Thrombocytopenia
  • Glanzmann’s Thrombasthenia

Immune Disorders

Severe Combined Immune Deficiency (SCID)
  • 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
Neutropenias
  • Kostmann Syndrome (Infantile Genetic Agranulocytosis)
  • Myelokathexis
Phagocyte Disorders
  • Chediak Higashi Syndrome
  • Chronic Granulomatous Disease
Inherited Disorders of the Immune System & other Organs
  • Cartilage Hair Hypoplasia
  • Erythropoietic Porphyria
  • Systemic Mastocytosis
Other Inherited Immune System Disorders
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Hemophagocytic Lymphohistiocytosis
  • IKK Gamma Deficiency3 (NEMO Dificiency)
  • IPEX Syndrome4
  • Leukocyte Adhesion Deficiency
  • Wiskott Aldrich Syndrome
  • X linked Lymphoproliferative Disease (Duncan’s Syndrome)
  • Ataxia-Telangiectasia

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)

Other Metabolic Disorders

Other Metabolic Disorders
  • Lesch–Nyhan Syndrome
  • Osteopetrosis
  • Hermansky-Pudlak Syndrome

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 potential use of stem cells continues to grow. Clinical trials are one of the key driving forces behind medical breakthroughs, and they represent 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 or unlock new medical options that are currently unavailable.

There are also diseases where stem cell* treatments may help effect a cure, but further research is needed to determine the best candidate patients for stem cell therapy, optimum stem cell dosage and method of cell delivery, etc.

Patients with difficult to treat or currently “incurable” diseases, such as AIDS and certain types of cancer, may want to pursue participation in clinical research trials if standard therapies are not effective.

Below are some of the diseases that are currently undergoing clinical trials. For the full list, please visit clinicaltrials.gov

  • Acute Myocardial Infarction (Heart Attack)
  • Acute Respiratory Distress Syndrome (ARDS)
  • Aging Frailty
  • Alopecia Areata
  • Alpha-Mannosidosis
  • Alzheimer’s Disease
  • Amyotrophic Lateral Sclerosis
  • Ankylosing Spondylitis
  • Autism
  • BronchoPulmonary Dysplasia (BPD) (lung disorder due to premature birth)
  • Cardiomyopathy
  • Cartilage injury
  • Cerebral palsy
  • Cleft Palate Repair
  • Critical Limb Ischemia
  • Crohn's disease
  • Diabetes, Type 1
  • Diabetes, Type 2
  • Diabetic Foot Ulcer
  • Diabetic Peripheral Neuropathy
  • Duchenne Muscular Dystrophy
  • Eczema (Atopic Dermatitis)
  • Encephalopathy
  • Epidermolysis Bullosa
  • Erectile Dysfunction
  • Ewing Sarcoma
  • Eye Diseases
  • Fistula
  • Gaucher's Disease
  • Global Developmental Delay
  • Graft versus Host Disease (GvHD)
  • Hearing Loss (acquired sensorineural)
  • Heart Failure
  • Hereditary Ataxia
  • HIV
  • Huntington’s Disease
  • Hypoplastic Left Heart Syndrome
  • Intraventricular Hemorrhage
  • Ischemic Heart Disease
  • Liver cirrhosis
  • Liver Failure
  • Lupus
  • Lysosomal Storage Diseases
  • Metabolic Syndrome
  • Multiple Sclerosis
  • Non-Union Fractures
  • Osteoarthritis
  • Osteochondral Lesion
  • Ovarian Cancer
  • Parkinson’s Disease
  • Peripheral Arterial Disease (PAD)
  • Peyronie's Disease
  • Premature Ovarian Failure
  • Psoriasis
  • Rheumatoid Arthritis
  • Severe Combined Immunodeficiency
  • Spinal cord injury
  • Spinal Fusion Surgery
  • Spinal muscular atrophy
  • Stroke
  • Surgery for Congenital Heart Defects
  • Systemic Sclerosis
  • Tay-Sachs Disease
  • Testicular Tumour
  • Traumatic Brain Injury
  • Ulcerative Colitis
  • Uterine Scars
  • Wounds

*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:



DCR No. : 1458 QR 8.1-8-11- d, August 2019
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