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ESH Erythro 2025 | Repurposing drugs in sickle cell disease: lessons learned from hydroxyurea

In this video, Sara El Hoss, PhD, Institut Imagine, Paris, France, shares insights into the potential value of repurposing existing drugs for sickle cell disease (SCD), highlighting hydroxyurea as a prime example. Dr El Hoss explains that repurposing drugs can accelerate the development of new treatments. This interview took place at the 4th ESH Translational Research Conference on Pathophysiology and Clinical Advances in Sickle Cell Disease and Thalassemia in Dublin, Ireland.

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Transcript

This is a commentary that was published in the British Journal of Haematology, and it’s a commentary that I wrote with my student… and in this commentary we were aiming to really look back into the literature and to see what drugs have been repurposed for sickle cell disease. And I think that one major drug that we need to mention is hydroxyurea, because hydroxyurea was a drug that was used in other pathologies, and scientists saw that it could increase fetal hemoglobin levels...

This is a commentary that was published in the British Journal of Haematology, and it’s a commentary that I wrote with my student… and in this commentary we were aiming to really look back into the literature and to see what drugs have been repurposed for sickle cell disease. And I think that one major drug that we need to mention is hydroxyurea, because hydroxyurea was a drug that was used in other pathologies, and scientists saw that it could increase fetal hemoglobin levels. And so it made sense for it to be used in sickle cell disease. And now it is one of the main treatments given to sickle cell patients. And so in this commentary, our aim was to highlight the fact that, of course, we need to look for novel treatments, but sometimes it is worth it to go back and to look at other available treatments that are used for other pathologies and see if we can repurpose them, because this really makes things go faster, because when you’re repurposing a drug, you don’t have to go back and get approval for the drug to be on the market, because it is usually on the market, but then you just do studies to prove that it is required for another – that it could be efficient for another disease. So this was the aim of this commentary.

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