Stem cell therapy and Fanconi anaemia

60% of patients with Fanconi anaemia are born with at least one physical abnormality[1]

80 – 90% of Fanconi anaemia cases are due to a genetic mutation[1]

Approximately 10% of people with Fanconi anaemia have leukaemia[2]

Without stem cell therapy, the average lifespan of Fanconi anaemia patients is 20 – 30 years[2]

80% – 90%
of Fanconi anaemia cases
are caused by genetic mutation

What is Fanconi anaemia?

Fanconi anaemia is a rare inherited form of anaemia that affects bone marrow, organs and tissue due to a decrease in all types of cells. It is the most common cause of inherited aplastic anaemia and can lead to serious health problems such leukaemia – a form of blood cancer.[3]

Because the Fanconi anaemia gene defect affects all cells, children with this disorder also have a higher risk of birth defects, hearing loss, growth problems and heart problems. Without stem cell therapy, patients will require regular blood transfusions, bone marrow exams and hormone therapy medication to regulate their condition.

Stem cell therapy research

The first cord blood stem cell transplant for Fanconi anaemia was in 1988. Since then, cord blood transplants have made significant advancements in treating FA and improving survival rate.

There are currently nine clinical trials investigating cord blood to treat Fanconi anaemia.[4] These trials demonstrate that allogenic haematopoietic stem cells found in cord blood can replenish the deficit of healthy cells in FA patients.[5]

Our stem cell releases for anaemia

To date, Future Health Biobank has released a cord blood stem cell sample for the successful treatment of Fanconi anaemia. The patient was a young boy who received an infusion of cord blood from his sibling and is now living a healthy, normal life. You can read more about Laith’s story here.

Many parents and healthcare professionals approach us seeking information for children that are diagnosed with anaemia. Our guidance for therapypage provides useful links to the latest developments in stem cell therapies.

References

1. https://rarediseases.org/rare-diseases/fanconi-anemia/

2. https://www.disabled-world.com/disability/types/fanconi-anemia.php

3. https://www.seattlecca.org/diseases/fanconi-anemia/fanconi-anemia-facts

4. https://clinicaltrials.gov/ct2/results?term=cord+blood+fanconi+anaemia&Search=Search

5. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3129570/

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