The possibility of using hematopoietic stem cells to restore production of normal hemoglobin is being investigated as a potential strategy to reduce the number of blood transfusions for thalassemia patients.
Magdalene Crabbe, Pharma Analyst at GlobalData, a leading data and analytics company, offers her view:
“In clinical trials, stem cells from the bone marrow of thalassemia patients with the normal form of the HBB gene were transfected through a viral vector into their erythrocytes. The majority of patients (68.2%) no longer required monthly blood transfusions.
“Despite the positive trial results, the procedure is expensive and questions remain over the long-term effects of stem cell therapy on people with hematological disorders.
“Most thalassemia patients need regular transfusions to supply them with red blood cells that can carry enough oxygen, which can improve the patient’s prognosis. However this can result in a multitude of complications.
“Continued research efforts are needed to find ways to improve the life expectancy of these patients, and to also give them the opportunity to enjoy energetic activities that require the correct functionality of oxygen homeostasis machinery.”