ASCAT 2023 | The current landscape of thalassemia treatment
Subarna Chakravorty, MBBS, MRCPCH, FRCPath, PhD, King’s College Hospital NHS Foundation Trust, London, UK, gives an update on advances in thalassemia treatment. Whilst blood transfusion and iron chelation serve as well-established treatment options, there are still further steps to be taken to provide patients with the best possible quality of life. Dr Chakravorty discusses the alternative treatment options being considered for treatment of the disease, with gene therapy showing great promise. 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)
So in the thalassemia space, of course gene therapy is the big thing again. I think there is more understanding now and I think clinicians are certainly talking about quality of life issues with thalassemia patients, although you might argue that with regular transfusion and iron chelation, perhaps the quality of life for patients is reasonable. But actually there’s lots more to thalassemia as an adult patient than just coming to hospital for transfusions, there are things like pain and fatigue and bone loss and so on, which also will need to be continued to be addressed...
So in the thalassemia space, of course gene therapy is the big thing again. I think there is more understanding now and I think clinicians are certainly talking about quality of life issues with thalassemia patients, although you might argue that with regular transfusion and iron chelation, perhaps the quality of life for patients is reasonable. But actually there’s lots more to thalassemia as an adult patient than just coming to hospital for transfusions, there are things like pain and fatigue and bone loss and so on, which also will need to be continued to be addressed. So gene therapy obviously will provide a fantastic opportunity for cure without the need for having an allogeneic bone marrow donor. So as long as it can be delivered in a, you know, relatively safe and relatively cost effective way, that will be a real game changer.
There are some other investigational products that are being considered, but not that many. Right now there is nothing new approved in the UK space but there is luspatercept which is used in Europe but unfortunately that reduces your transfusion burden but doesn’t completely negate it. So it will reduce your transfusion burden to a certain extent.
And the other thing that I would really be interested in seeing, but I don’t really hear that much about it is other types of iron chelation. So there are new drugs that are being developed but I’m hoping that we get to hear more about them. Right now we only have three different iron chelation treatments, of which one has to be given either through subcutaneous or intravenous route, which has been around for over 50 years. We’ve not really moved on from that. And there are two other oral drugs. Not everyone tolerates all these drugs easily, they’re difficult to take. So we really want a solution for that and it will also make a huge difference, but there’s none which are really very promising right now.
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