Reversing Anemia with Stem Cell Treatments

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Reversing Anemia with Stem Cell Treatments

How Stem Cells Treat Anemia

  • All blood cells, including red blood cells, white blood cells and platelets, originate from hematopoietic stem cells in the bone marrow. These master cells have the ability to self-renew as well as develop into specialized blood cells. In many cases of anemia, the bone marrow lacks enough healthy stem cells to produce adequate blood cell counts.
  • Stem cell therapy aims to restore normal blood cell production by introducing new, healthy hematopoietic stem cells. When transplanted into a patient, these stem cells can travel to the bone marrow and begin proliferating. Over time, they differentiate into mature red blood cells, white blood cells and platelets, reconstituting the blood system.
  • Stem cell transplants have been used for decades to treat blood cancers like leukemia. More recently, research has shown they can also effectively treat other hematologic disorders like aplastic anemia and sickle cell disease. In fact, stem cell therapy is currently the only established curative treatment for severe aplastic anemia.

Treating Aplastic Anemia

  • Aplastic anemia is a rare, life-threatening condition where the bone marrow stops producing enough new blood cells. This leads to extremely low counts of all major blood components that require frequent blood transfusions. The only definitive cure is a bone marrow transplant to restore normal blood cell production.
  • Stem cells from umbilical cord blood represent an exciting new transplantation source. Because these immature cells are less developed immunologically, cord blood transplants have a lower incidence of rejection and complications compared to adult marrow. Recent studies estimate 60-70% of patients who receive a cord blood transplant for aplastic anemia are cured and disease-free after 5 years.

Reversing Inherited Anemias

  • Stem cell therapy also holds promise for correcting inborn genetic defects that cause some rare types of inherited anemia. In disorders like Fanconi anemia, Diamond-Blackfan anemia and sickle cell disease, genetic mutations affect the normal development of red blood cells.
  • An allogeneic stem cell transplant from a healthy matched donor offers the only way to permanently correct the underlying genetic defect. When transplanted, the normal stem cells take over blood cell production, replacing diseased cells. Today over 90% of children with Fanconi anemia can be cured with a stem cell transplant from a matched sibling donor. Ongoing research also continues to improve transplant success for sickle cell disease.

Treating Acquired Aplastic Anemia

  • While inherited conditions are due to genetic defects from birth, acquired aplastic anemias develop later in life. The aplastic anemia itself is not inherited, but rather arises from damage to the bone marrow.
  • Research now indicates that stem cell therapy without a full transplant may help many acquired aplastic anemia patients. In this approach, patients receive an infusion of allogeneic mesenchymal stem cells. While too few in number to replace the entire blood system, these mesenchymal stem cells still tend to stimulate recovery in the patient’s own bone marrow.
  • In clinical experience, a majority of acquired aplastic anemia patients experience significant improvement in blood counts and reduced transfusion needs following mesenchymal stem cell therapy. For older or weaker patients unable to withstand a transplant, mesenchymal stem cell infusions may provide a restorative new treatment option.

Optimizing Stem Cell Therapy Success

Ongoing research aims to optimize stem cell therapy protocols and success rates for all types of anemia. Careful donor matching, stem cell processing, and treatment monitoring can help ensure patients have the greatest chance of achieving disease remission or a complete cure.


For patients suffering from severe anemia, stem cell therapy represents a revolutionary regenerative medicine that can restore their vitality and quality of life. Instead of lifelong transfusions and medications, they now have access to treatments that can potentially reverse or permanently cure their condition. As research continues to advance, stem cell transplants and infusions offer renewed hope for those with both inherited and acquired forms of anemia.