Thalassemia is an inherited blood disorder that cannot be treated with medication. But stem cell therapy may be able to help. Know if stem cell therapy will work for the disease.

Written by Editorial Team | Updated : May 18, 2022 10:56 AM IST

Haemoglobin is the oxygen-carrying protein in the blood. In the absence or reduced amount of haemoglobin, the red cells do not survive for a long period and there is a diminished oxygen supply to the tissues causing a variety of symptoms. Thalassemia is an inherited blood disorder in which there is less haemoglobin than normal. There are two parts to the haemoglobin molecule alpha and beta. The disease is named based on the subunit that is not being made by the body alpha or beta-thalassemia.

Thalassemia runs in families; however, not everyone who has a dysfunctional gene for haemoglobin will have the symptoms of the disease. Those with moderate-severe disease experience symptoms such as breathlessness, weakness etc. (symptoms of anaemia), all associated with low levels of oxygen, from childhood. However, those with the mild disease might incidentally be diagnosed with thalassemia, mostly during investigations for other conditions or for anaemia.

Unfortunately, thalassemia cannot be prevented. Patients with the severe disease receive frequent blood transfusions, in an attempt to replenish a pool of healthy red blood cells. However, this procedure is associated with several issues such as iron overload that can deposit in different organs over time, immune system reactions, and risk of blood-related infections such as hepatitis, HIV etc. Folic acid is also prescribed to patients, which can help in the development of red blood cells.

Considering the issues with the conventional treatment of thalassemia, stem cell therapy can be an attractive choice for the condition. Allogeneic hematopoietic stem cell (blood stem cells) transplantation has been considered the only curative treatment for thalassemia. This treatment works by replenishing a healthy pool of blood-forming stem cells (master cells of the body) in the bone marrow consider the bone marrow as a factory of stem cells and specialized blood and tissue forming cells. However, considering that the source of stem cells is from a donor (usually a first-degree relative) because a patient's own blood-forming stem cells would be affected by the genetic mutation there are chances of certain side effects. One way to manage these issues is by the use of mesenchymal stem cells, which are known to have immune system regulating properties. By this, the rejection-related issues with hematopoietic stem cells as well as reduced immunity-related side effects can be handled efficiently. Considering that thalassemia is a disease that affects blood cells, mesenchymal cells (tissue forming cells) from the patient's own body are not affected.

Through this combined approach, a patient can achieve long-term remission from the symptoms of thalassemia without side effects. "Stem cell therapy for thalassemia is not a new treatment, only the approach by using a combination of stem cells to achieve holistic outcomes is novel.

(The article is contributed by Dr Pradeep Mahajan, Regenerative Medicine Researcher, StemRx Bioscience Solutions Pvt. Ltd., Navi Mumbai/Mumbai)

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How Does Stem Cell Transplant Work In Thalassemia? Know Everything | TheHealthSite.com - TheHealthSite

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