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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 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.

Umbilical Cord Blood Stem Cells

Umbilical cord blood stem cells can be use to treat more than 113 diseases.


  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myelogenous Leukemia (AML)
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia
  • Chronic Lymphocytic Leukemia (CLL)
  • Chronic Myelogenous Leukemia (CML)
  • Chronic Myelomonocytic Leukemia
  • Juvenile Chronic Myelogenous Leukemia (JCML)
  • Juvenile Myelomonocytic Leukemia (JMML)
  • Refractory Anemia (RA)
  • Refractory Anemia with Ringed Sideroblasts (RARS)
  • Refractory Anemia with Excess Blasts (RAEB)
  • Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
  • Chronic Myelomonocytic Leukemia (CMML)

Other Disorders of Blood Cell Proliferation

  • Congenital Dyserythropoietic Anemia
  • Dyskeratosis Congenita
  • Fanconi's Anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • Shwachman-Diamond Syndrome
  • Severe Aplastic Anemia, Unspecified
  • Sideroblastic Anemia
  • Aplastic Anemia
  • Thalassemia
  • Beta Thalassemia Major/ Cooley's Anemia
  • Diamond-Blackfan anemia
  • Pure Red Cell Aplasia
  • Sickle Cell Disease
  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Glanzmann Thrombasthenia
  • 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
  • Omenn Syndrome
  • Ataxia Telangiectasia
  • Bare Lymphocyte Syndrome
  • Common Variable Immune Deficiency
  • Congenital Immune Deficiency
  • DiGeorge Syndrome
  • Griscelli Syndrome
  • Leukocyte Adhesion Deficiency
  • Lymphocyte Adhesion Disease
  • Lymphoproliferative Disorders (LPD)
  • Nezelof's Syndrome
  • Systemic Lupus Erythematosus (SLE)
  • X-linked Hyper-IgM Syndrome
  • X-linked Immune Dysregulation Polyendocrine Enteropathy
  • X-linked Lymphoproliferative Disorder (Epstein-Barr Virus Suspectibility)
  • Wiskott-Aldrich Syndrome
  • Kostmann Syndrome
  • Myelokathexis
  • Congenital Neutropenia
  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (Myelofibrosis)
  • Polycythemia Vera
  • Essential Thrombocythemia
  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Neutrophil Actin Deficiency
  • Reticular Dysgenesis
  • Multiple Myeloma
  • Plasma Cell Leukemia
  • Waldenstrom's Macroglobulinemia
  • Reticular Dysgenesis
  • Familial Erythrophagocytic Lymphistiocytosis
  • Hemophagocytic Syndrome
  • Hemophagocytic Lymphohistiocytosis
  • Histiocytosis
  • Langerhans Cell Histiocytosis

Transplants for Inherited Metabolic Disorders

  • Mucopolysaccharidoses (MPS)
  • Hurler's Syndrome (MPS-IH)
  • Hurler-Scheie Disease (MPS-IS)
  • Hunter's Syndrome (MPS-II)
  • Sanfilippo Syndrome (MPS-III)
  • Morquio Syndrome (MPS-IV)
  • Maroteaux-Lamy Syndrome (MPS-VI)
  • Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)
  • Mucolipidosis II (I-cell Disease)
  • Adrenoleukodystrophy (ALD) / Adrenomyeloneuropathy (AMN)
  • Krabbe Disease (Globoid Cell Leukodystrophy)
  • Metachromatic Leukodystrophy
  • Pelizaeus-Merzbacher Disease
  • Gaucher Disease
  • Niemann-Pick Disease
  • Sandhoff Disease
  • Tay-Sachs Disease
  • Wolman Disease
  • Alpha Mannosidosis
  • Amyloidosis
  • Aspartylglucosaminuria
  • Austin's Disease (Multiple Sulfatase Deficiency)
  • Fucosidosis
  • Gangliosidosis
  • Infantile Ceroid Lipofucoscinosis
  • Neiman-Pick Disease
  • Sialidosis
  • Lesch-Nyhan Syndrome
  • Osteopetrosis

Solid Tumors not Originating in the Blood or Immune System


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.

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.

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  • 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

The above list of diseases is a compilation from the above websites and other sources such as medical literatures and journals. Kindly approach us at (852) 3980 2888 or (853) 6881 0781 or This email address is being protected from spambots. You need JavaScript enabled to view it. to request for a particular medical journal / literature.

*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.

DCR No. 3321, October 2017