January 24, 2024

Unlocking the Potential

Cord Blood Stem Cell Therapy and the Spectrum of Treatable Diseases

Dr. Lana du Plessis

Over the past three decades, the field of cord blood stem cell therapy has witnessed remarkable progress, with more than 40,000 successful transplants worldwide. These transplants have emerged as transformative treatments for various medical conditions, particularly blood disorders, inherited metabolic disorders, and immunodeficiency syndromes. As the therapeutic landscape continues to expand, cord blood stem cells have found applications in over 80 approved therapies, offering hope for patients facing a diverse range of diseases.

Treatable Diseases and Therapies

Cord blood stem cell transplants have proven effective in treating a multitude of diseases, ranging from blood cancers to inherited metabolic disorders and bone marrow disorders. The following is a glimpse into the scope of treatable diseases and approved therapies:

Blood Cancers:

Acute Lymphoblastic Leukaemia (ALL)
Acute Myeloid Leukaemia (AML)
Chronic Myeloid Leukaemia (CML)
Myelodysplastic Syndrome (MDS)
Multiple Myeloma
Hodgkin’s Lymphoma
Non-Hodgkin’s Lymphoma

Inherited Metabolic Disorders:

Hurler Disease (MPS type IH)
Osteopetrosis
Adrenoleukodystrophy
Krabbe Disease

Bone Marrow Disorders:

Aplastic Anaemia
Unspecified Fanconi Anaemia

Other Diseases:

Blood Disorders
Bone Marrow Failure Syndrome
Immunodeficiencies
Neuroblastoma
Solid Tumours

For a comprehensive list of current treatable diseases and therapies, interested readers can refer to the Parents’ Guide to Cord Blood.

Ongoing Clinical Trials

The frontier of cord blood stem cell therapy extends beyond approved therapies, with ongoing clinical trials exploring the potential applications of these cells in various conditions. Some of the areas currently under investigation include:

Acquired hearing loss
Alzheimer’s disease
Acute Ischemic Stroke
Autism Spectrum Disorders
Amyotropic Lateral Sclerosis
Bronchopulmonary dysplasia
Cartilage repair
Critical limb ischemia
Cerebral Palsy
Congenital Diaphragmatic Hernia
Congenital Heart Diseases
Childhood Hearing Loss
Corneal Epithelial Wounds
Ulcerative Colitis / Inflammatory Bowel Disease
Duchenne Muscular Dystrophy
Diabetic Foot Ulcers
Diabetes Mellitus (Type I & Type II)
Encephalopathy (neonatal)
Epidermolysis Bullosa
Fertility
Global development delay
Graft versus host diseases
Glaucoma
Hypoplastic left heart syndrome
HIV
Hydrocephalus
Ischemic Stroke (pre/peri-natal)
Intraventricular haemorrhage
Infant Lung Disease
Idiopathic Dilated Cardiomyopathy
In-Utero Brain Injury / Stroke
Liver Cirrhosis
Neurodegenerative Disorders
Preterm Neonatal Complications
Parkinson’s Disease
Rheumatoid Arthritis
Severe Hypoxic-ischemic Encephalopathy
Systemic Lupus Erythematosus
Spinal Cord Injury
Skin-Wound / Burns
Sweat Gland Diseases / Regeneration

References

Cairo MS, Rocha V, Gluckman E, et al. Alternative allogeneic donor sources for transplantation for childhood diseases: unrelated cord blood and haploidentical family donora. Biol Blood Marrow Transplant. 2008; 14:44-53.
Cairo MS, Tarek N, Lee DA, et al. Cellular engineering and therapy in combination with cord blood allografting in pediatric recipients. Bone Marrow Transplant. 2016;51:27-33
Gluckman E, Ruggeri A, Rocha V, et al. for Eurocord, Netcord, World Marrow Donor Association and National Marrow Donor Program. Family-directed umbilical cord blood banking. Haematolgica. 2001; 96: 1700-1707.
Mazonson P, Kane M, Colberg K, et al. Prevalence of medical conditions potentially amenable to cellular therapy among families privately storing umbilical cord blood. Matern Child Health J. 2016 (online).
https://clinicaltrials.gov

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