Stem cell therapy and beta thalassemia major

Beta thalassemia 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 thalassemia is common among people of Mediterranean, Middle Eastern, Asian, Southeast Asian and Caribbean backgrounds[3]

Parents who both carry the beta thalassemia gene have a 1 in 4 chance of having a child with beta thalassemia major[3]

An estimated
300K
people in the UK
carry the BTM gene

What is beta thalassemia major?

Beta thalassemia 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: thalassemia major and thalassemia intermedia, thalassemia 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 thalassemia major, patients must have regular blood transfusions to correct anaemia symtpmos 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 thalassemia major. Once infused, these healthy cord blood stem cells divide and replicate in the patient’s bloodstream to replenish red blood cells and sustainable 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 thalassemia, and the method is now recognized as a safe and effective form of treatment in children.[5]

Our stem cell releases for autism

To date, Future Health Biobank has released one cord blood stem cell samples to treat beta thalassemia 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.

References

1. https://www.uclh.nhs.uk/OurServices/ServiceA-Z/Cancer/CBD/THAL/Pages/Home.aspx

2. https://kidshealth.org/en/parents/beta-thalassemia.html

3. http://www.cooleysanemia.org/updates/THALFACTSHEET.pdf

4. https://www.ncbi.nlm.nih.gov/books/NBK1426/

5. http://www.bloodjournal.org/content/101/6/2137

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