Stem cell therapy and beta thalassaemia major

Beta thalassaemia major (BTM) affects around 1,000 people in England[1]
An estimated 300,000 people in the UK are carriers of this BTM[1]
Without stem cell therapy, BTM patients require blood transfusions every 2-4 weeks[2]
Beta thalassaemia is common among people of Mediterranean, Middle Eastern, Asian, Southeast Asian and Caribbean backgrounds[3]
Parents who both carry the beta thalassaemia gene have a 1 in 4 chance of having a child with beta thalassaemia major[3]

An estimated
people in the UK
carry the BTM gene

What is beta thalassaemia major?

Beta thalassaemia is an inherited blood disorder that reduces the production of haemoglobin. Haemoglobin is the iron-containing protein in red blood cells, and low levels of it result in a lack of oxygen in various part of the body. Of the two types: thalassaemia major and thalassaemia intermedia, thalassaemia major is more severe, giving a higher risk of developing abnormal blood clots.

People with this disorder also suffer from anaemia (lack of red blood cells), causing anaemia symptoms such as pale skin, fatigue, delayed growth and other health complications. These symptoms often appear within a child’s first two years, where they can develop life-threatening anaemia.

To manage beta thalassaemia major, patients must have regular blood transfusions to correct anaemia symptoms and increase absorption of iron. This method carries its own complications, as a build up of infused iron can cause liver, heart and hormone problems.[1]

Stem cell therapy research

Stem cell therapy using the cord blood stem cells of an HLA-matched donor (preferably a sibling) is the only known cure for beta thalassaemia major. Once infused, these healthy cord blood stem cells divide and replicate in the patient’s bloodstream to replenish red blood cells and sustain iron levels. To add to the benefits of this treatment method, cord blood transplants carry a low risk of the patient developing graft-v-host disease, unlike a bone marrow transplant.[4]

There have been many clinical trials into the related and un-related cord blood transplants for beta thalassaemia, and the method is now recognized as a safe and effective form of treatment in children.[5]

Our stem cell releases for beta thalassaemia major

To date, Future Health Biobank has released one cord blood stem cell sample to treat beta thalassaemia major. The sample was a successful HLA match from the patient’s younger sibling, and greatly improved the patient’s condition following transplant.

Many parents and healthcare professionals approach us seeking information for children that are diagnosed with a genetic blood disorder. Our guidance for therapy page provides useful links to the latest developments in stem cell therapies.

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