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Hematopoietic Stem Cells: What Diseases Can these Stem Cells Treat?

Hematopoietic Stem Cells

Hematopoietic stem cells (HSCs) are defined as stem cells that have a preference for becoming cells of the blood and immune system, such as white blood cells, red blood cells, and platelets. Found in the peripheral blood and bone marrow, hematopoietic stem cells are also present in plentiful supply within the umbilical cord blood of newborn babies.

For the past thirty years, cord blood has been used within transplant medicine, including for the treatment of leukemia and other blood diseases. For most conditions in which a bone marrow or peripheral blood stem cell transplant is an option, a cord blood transplant is a potential alternative.

Hematopoietic Stem Cells

In this article:

Hematopoietic Stem Cell Transplant as Treatment

Hematopoietic stem cells (HSCs) are the stem cells that repopulate the blood and immune system within humans, via a process known as haematopoiesis. For this reason, hematopoietic stem cell transplantation, better known as HSCT, can be a promising treatment approach for a wide range of conditions.

Human Cord Blood Cells

The use of human cord blood cells dates back as early as 1974, when it was first proposed that stem cell and progenitor cells were present in human cord blood.  By 1983, the use of cord blood as an alternative to bone marrow had been proposed. Five years later in 1988, the first successful cord blood transplant to restore a patient’s blood and immune system cells took place in France.

In addition to a long history of use within transplant medicine, human cord blood cells are playing a growing role within regenerative medicine. Cord blood stem cells have been induced to develop into neural cells, suggesting that they may represent a potential treatment for neurological conditions, such as Alzheimer’s, Parkinson’s, spinal cord injury, dementia, and related conditions.

Human cord blood cells can also develop into blood vessels, making them promising for the repair of tissues following stroke, coronary heart disease, rheumatic heart disease, congestive heart failure, and congenital heart conditions.

Common Diseases Treatable with Cord Blood Transplant

It is also interesting to consider the common disease categories treatable with cord blood transplant, as shown in the table below.

Common Disease Categories
1. Cancers
  • leukemias
  • high-risk solid tumors
  • lymphomas
2. Blood Disorders
  • Thalassemia
  • Anemias
  • Sickle Cell Disease
3. Immune Disorders
  • Severe Combined Immunodeficiency Disease
  • Hystiocytic Disease
  • Wiskott-Aldrich Syndrome
4. Metabolic Diseases
  • Krabbe Disease
  • Hurler Syndrome
  • Leukodystrophies

Allogeneic and Autologous Transplants

There are more than 80 medical conditions for which transplantation of hematopoietic stem cells (including cord blood transplant) is a standard treatment option. Most of these therapies require allogeneic transplants, where the patient must use a genetically-matched cord blood donor. The only exceptions to this are patients who are transplanted for solid tumors or acquired anemias. In these situations, the patient may receive an autologous transplant.

Comprehensive lists of conditions treatable with hematopoietic stem cells are available here and here.

Diseases Treatable with Hematopoietic Stem Cells in the Future

In addition, there is a range of disease categories for which cord blood transplant could represent a viable treatment method in the future. For these conditions, there are still unknown criteria that need to be determined before the cord blood stem cell transplant can become commonplace, such as patient criteria for optimal treatment effectiveness, optimum stem cell quantity for use in transplant, and preferred method of stem cell delivery into the patient, as shown below.

1. Brain Disorders
  • Alzheimer’s
  • Dementia
  • Huntington’s Disease
  • Cerebral Palsy
  • Down’s Syndrome
2. Cartilage Growth & Joint Rejuvenation
  • Traumatic or Surgical Damage
  • Rheumatoid Arthritis
3. Diabetes
  • Type I diabetes
  • Type II diabetes
  • Gestational diabetes
4. Heart Disease
  • Coronary Heart Disease (CHD)
  • Heart Attack
  • Ischemic Heart Disease
  • Rheumatic Heart Disease
  • Pulmonary Heart Disease
5. Liver Disease
  • Alcoholic Liver Disease
  • Nonalcoholic Fatty Liver Disease
  • Hepatocellular Carcinoma
  • Reye’s syndrome
  • Wilson’s Disease
  • Other Hepatic and Biliary Disorders
6. Multiple Sclerosis
  • Relapsing-Remitting Multiple Sclerosis (RRMS)
  • Secondary Progressive Multiple Sclerosis (SPMS)
  • Primary Progressive Multiple Sclerosis (PPMS)
  • Malignant Multiple Sclerosis
  • Chronic Progressive Multiple Sclerosis
7. Muscular Dystrophy (MD)
  • Duchenne MD
  • Becker MD
  • Congenital MD
  • Emery-Dreifuss MD
  • Facioscapulohumeral MD
  • Limb-Girdle MD
  • Myotonic MD
  • Oculopharyngeal MD
8. Skin and Tissue Regeneration
  • Burn Damage
  • Trauma Damage
  • Epidermolysis Bullosa
9. Spinal Cord Degeneration or Trauma
  • Spinal Cord Trauma
  • Paralysis
  • Spinal Stenosis
  • Paraplegia
10. Stroke
  • Ischemic Stroke
  • Intracerebral Stroke
  • Subarachnoid Hemorrhage

Download this infographic below to reference it later.

What do you think of the future of hematopoietic stem cell transplant? Share your thoughts in the comments below.

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