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General Updates | The repurposing of drugs for the treatment of sickle cell disease, with a focus on luspatercept
Thiago Trovati Maciel, PhD, Research Director, Institut Imagine, Paris, France, provides insight into the repurposing of drugs for the treatment of sickle cell disease (SCD), focusing on luspatercept, which has shown promising results in preclinical studies and may potentially address unmet needs in patients with this disease. Dr Maciel highlights the overlap in pathophysiology between beta-thalassemia and SCD, particularly related to ineffective erythropoiesis, which underpins the rationale for repurposing this agent. This interview took place virtually.
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Transcript
The main problem today with sickle cell disease is that we have a lot of research ongoing, but very few drugs that are available for the patients. And we have gene therapy that is now being used, but not as we would like to, because the curative treatments for the sickle cell disease patients are still based on gene therapy and also bone marrow transplant and very few patients can use these treatments today...
The main problem today with sickle cell disease is that we have a lot of research ongoing, but very few drugs that are available for the patients. And we have gene therapy that is now being used, but not as we would like to, because the curative treatments for the sickle cell disease patients are still based on gene therapy and also bone marrow transplant and very few patients can use these treatments today. So I think the main goal that we have to start to put in place is to repurpose the drugs that are already available for other diseases and use these drugs to treat patients with sickle cell disease, especially the patients that have severe complications that are not completely treated with the main treatment, hydroxyurea and blood transfusion.
So if we look first at luspatercept, we worked together with Celgene and BMS years ago to develop this treatment for beta-thalassemia patients. So today it is a treatment that is very positive for beta-thalassemia patients. And what we can see is that beta-thalassemia patients and sickle cell patients, they have some similarities in the physiopathology, especially in ineffective erythropoiesis. So we ask ourselves the question, can we also use luspatercept to treat sickle cell patients? So we started to do preclinical work and the results that we obtained that were presented at ASCAT this year are very promising. So I think that maybe in the future with discussions with the clinical partners, the industrial partners, we can also place luspatercept for the sickle patients and that can be another therapy in place for these patients to address all the unmet needs that they have today.
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