Diseases Treated with Umbilical Cord Blood Stem Cells and its Potential Applications

Stem cells in the blood of your baby’s umbilical cord have the potential to be used in the treatment of many diseases today. Stem cells could be used to treat hematopoietic and genetic disorders. In a stem cell transplant, stem cells are infused into a patient’s bloodstream where they go to work - healing and repairing damaged cells and tissue. With the successful engraftment of the stem cells, the patient’s blood and immune system are regenerated.

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 (Hematopoietic Stem Cell), like bone marrow and peripheral blood. Stem cell therapies continue to change and evolve quickly.

There are a wide range of diseases that are treatable with Hematopoietic Stem Cells (HSCs) from umbilical cord blood, bone marrow, and peripheral blood such as stem cell disorders, acute and chronic forms of leukemia, myeloproliferative disorders, and many more. Stem cell therapies continue to change and evolve quickly.

Umbilical Cord Blood Stem Cells

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

Blood Cancers


  1. Acute Biphenotypic Leukaemia
  2. Acute Lymphoblastic Leukaemia
  3. Acute Myelogenous Leukaemia
  4. Acute Undifferentiated Leukaemia
  5. Chronic Myelogenous Leukaemia
  6. Chronic Lymphocytic Leukaemia
  7. Juvenile Myelomonocytic Leukaemia
  8. Juvenile Chronic Myelogenous Leukaemia

Myelodysplastic Syndromes

  1. Refractory Anaemia
  2. Refractory Anaemia with Excess Blasts
  3. Refractory Anaemia with Excess Blasts in Transformation
  4. Refractory Anaemia with Ringed Sideroblasts (Sideroblastic Anaemia)
  5. Chronic Myelomonocytic Leukaemia

Myeloproliferative Neoplasms

  1. Acute Myelofibrosis
  2. Agnogenic Myeloid Metaplasia
  3. Essential Thrombocythemia
  4. Polycythemia Vera

Other Blood Cancers

  1. Multiple Myeloma
  2. Plasma Cell Leukaemia
  3. Waldenstrom’s Macroglobulinemia
  4. Histiocytic Neoplasms

Solid Tumors


  1. Hodgkin Lymphoma
  2. Langerhans' Cell Histiocytosis
  3. Neuroblastoma
  4. Non Hodgkin Lymphoma (Burkitt’s Lymphoma)
  5. Retinoblastoma
  6. Medulloblastoma
  7. Wilms Tumor

Non Malignant Blood Disorders

Anaemias (Deficiences or malformations of red cells)

  1. Aplastic Anaemia
  2. Congenital Dyserythropoietic Anaemia
  3. Fanconi’s Anaemia
  4. Paroxysmal Nocturnal Hemoglobinuria

Hereditary Bone Marrow Failure Syndromes

  1. Diamond Blackfan Syndrome
  2. Dyskeratosis Congenita
  3. Pearson’s Syndrome
  4. Shwachman Diamond Syndrome

Inherited Red Cell Abnormalities

  1. Pure Red Cell Aplasia
  2. Sickle Cell Anaemia
  3. Beta Thalassemia Major/Cooley’s Anaemia

Inherited Platelet Abnormalities

  1. Congenital Amegakaryocytosis Thrombocytopenia
  2. Glanzmann’s Thrombasthenia

Immune Disorders

Severe Combined Immune Deficiency (SCID)

  1. Bare Lymphocyte Syndrome
  2. Omenn Syndrome
  3. Reticular Dysgenesis
  4. Neutrophil Actin Deficiency
  5. SCID with Adenosine Deaminase Deficiency (ADA SCID)
  6. SCID which is X linked
  7. SCID with absence of T & B Cells
  8. SCID with absence of T Cells, Normal B Cells


  1. Kostmann Syndrome (Infantile Genetic Agranulocytosis)
  2. Myelokathexis

Phagocyte Disorders

  1. Chediak Higashi Syndrome
  2. Chronic Granulomatous Disease

Inherited Disorders of the Immune System & other Organs

  1. Cartilage Hair Hypoplasia
  2. Gunther’s Disease (Congenital Erythropoietic Protoporphyria)
  3. Systemic Mastocytosis

Other Inherited Immune System Disorders

  1. Common Variable Immunodeficiency
  2. DiGeorge Syndrome
  3. Evans Syndrome
  4. Hemophagocytic Lymphohistiocytosis
  5. IKK Gamma Deficiency (NEMO Deficiency)
  6. IPEX Syndrome
  7. Leukocyte Adhesion Deficiency
  8. Wiskott Aldrich Syndrome
  9. X linked Lymphoproliferative Disease (Duncan’s Syndrome)
  10. X linked Hyper IgM Syndrom
  11. Ataxia-Telangiectasia

Metabolic Disorders

Leukodystrophy Disorders

  1. Adrenoleukodystrophy
  2. Krabbe Disease (Globoid Cell Leukodystrophy)
  3. Metachromatic leukodystrophy
  4. Pelizaeus-Merzbacher Disease

Lysosomal Storage Diseases

  1. Alpha Mannosidosis
  2. Gaucher’s Disease
  3. Niemann Pick Disease
  4. Sandhoff Disease
  5. Wolman Disease

Mucopolysaccharidosis (MPS) Storage Diseases

  1. Hunter Syndrome
  2. Hurler Syndrome
  3. Maroteaux Lamy Syndrome
  4. Mucolipidosis II (I-cell Disease)
  5. Morquio Syndrome
  6. Sanfilippo Syndrome
  7. Scheie Syndrome
  8. Sly Syndrome (beta glucuronidase deficiency)

Other Metabolic Disorders

Other Metabolic Disorders

  1. Lesch–Nyhan Syndrome
  2. Osteopetrosis
  3. Hermansky-Pudlak Syndrome

The successful application of umbilical cord blood stem cells is dependent on the condition of each individual patient. Banking umbilical cord blood does not guarantee that the cells will provide a cure or be applicable in every situation. The eventual use of the umbilical cord blood can only be determined by the treating physician. Though some of the conditions listed here may be treated with the patient’s own umbilical cord blood (autologous cord blood), it may not be suitable to treat genetic diseases. In such cases, a matching umbilical cord blood from a sibling may be used. However, there is no guarantee that the umbilical cord blood will be a match for every family member or will provide a cure for every condition. Please consult your treating physician for further advice.

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.


  • 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 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 represent an avenue for receiving promising new therapies that would not otherwise be available. Patients with difficult to treat or currently "incurable" diseases, such as AIDS or certain types of cancer, may want to pursue participation in clinical research trials if standard therapies are not effective. 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 hematopoietic stem cells.

Umbilical Cord Lining Stem Cells

Today, more than 600 clinical trials1 are on-going worldwide to uncover the potential of Mesenchymal Stem Cells (MSCs). Thus far, encouraging results have been published; stem cell treatments have been proven safe and capable of repairing damage caused by stroke and heart disease. MSCs have also been used in combination with Hematopoietic Stem Cells (HSCs) as a dual therapy to promote faster engraftment of HSCs and to reduce immune system complications2.

Although there is evidence that MSCs can be changed to certain types of Epithelial Cells, the cells cannot be changed into Epithelial Stem Cells (EpSCs). The difference between Epithelial stem cells and Epithelial cells is that Epithelial stem cells can differentiate into all different epithelial cell types such as skin, cornea, lining of the gut, etc. on demand. Whereas, (non-stem) Epithelial cells have already reached terminal differentiation so the cells cannot be further changed into different Epithelial cell types when needed. Thus, MSCs and EpSCs cannot be replaced by each other.

1 ClinicalTrials.gov. Accessed on 13 August 2017. (http://www.clinicaltrials.gov/ct2/results?term=mesenchymal+stem+cells)
2 Battiwalla M, Hematti P. 2010. Cytotherapy. 1 January 2010.

Umbilical Cord Lining

Potential Therapeutic Application using MSCs and EpSCs

Mesenchymal Stem Cells (MSCs)

Tissue Repair

  1. Stroke
  2. Heart Failure
  3. Alzheimer’s Disease
  4. Parkinson’s Disease
  5. Spinal Cord Injury
  6. Orthopaedic indications (bone, cartilage, tendon repair)
  7. Liver Failure
  8. Autism

Immune Modulation

  1. HIV
  2. Type 1 diabetes
  3. Graft versus host disease (GvHD)

HSCs engraftment support

  1. Shorten time of engraftment
  2. Reduce immune system complications

MSCs Research

Worldwide researches on diseases treated with MSCs by various Universities or Research Institutions

DiseasesName of universities or research Institions
StrokeUniversity Hospital, Grenobe, France
Spinal Cord InjuryCairo University, Egypt
Chinese University of Hong Kong
Multiple SclerosisSpain-Carlos Health Institute
University of Cambridge, U.K.
Cleveland Clinic, U.S.
Amylotrophic Lateral SclerosisHadassah Medical Organization, Israel
Parkinson's DiseaseJaslok Hospital and Research Centre, India
Multiple System AtrophyYonsei University, South Korea
Liver DiseaseCytori Therapeutics, U.S.
Diabetic Foot UlcerThird Military Medical University, China
Washington DC Veterans Medical Centre, U.S.
Ruhr-University Bochum, Germany
DiabetesFuZhou General Hospital, China
Uppsala University, Sweden