ASCAT 2023 | Currently approved therapies for the treatment of SCD
Miguel Abboud, MD, American University of Beirut Medical Center, Beirut, Lebanon, outlines the four currently approved medications and therapies for sickle cell disease (SCD), as well as the therapies which are being developed and refined to improve treatment of patients with the disease. This interview was recorded at the 18th Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) 2023, held in London, UK.
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Transcript (edited for clarity)
These are very exciting days for sickle cell. You know, we have gone in my lifetime from having no major disease modifying therapies for sickle cell disease, then Hydroxyurea was approved and that took quite a while, and now we have four approved medications for sickle cell disease. P-selectin inhibitors, an inhibitor of sickling, L-glutamine (which improves the redox potential within the cells), and gene therapy approaches that are aimed at increasing fetal hemoglobin or providing an anti-sickling hemoglobin to the cells as well...
These are very exciting days for sickle cell. You know, we have gone in my lifetime from having no major disease modifying therapies for sickle cell disease, then Hydroxyurea was approved and that took quite a while, and now we have four approved medications for sickle cell disease. P-selectin inhibitors, an inhibitor of sickling, L-glutamine (which improves the redox potential within the cells), and gene therapy approaches that are aimed at increasing fetal hemoglobin or providing an anti-sickling hemoglobin to the cells as well. So this is in addition to the refining of transfusion therapy and the development of alternative donor bone marrow transplants, that will allow the expansion of bone marrow transplantation for patients with sickle cell disease.
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