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The Sickle Cell Disease Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
ASH 2024 | The potential for disease modification in SCD using pyruvate kinase activators
At present, therapeutic options are limited for patients with sickle cell disease (SCD). Santosh Saraf, MD, University of Illinois Hospital, Chicago, IL, comments on the potential value of pyruvate kinase activators in SCD, highlighting their ability to modify the disease by reducing sickling, hemolysis, anemia, and the incidence of vaso-occlusive episodes. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Transcript (AI-generated)
So the PKR activators are another really exciting group of therapies for sickle cell disease. So we have gene therapy, which is more of a curative or transformative therapy. But for those that want to have something more for disease modification, it’s something they can take orally at home, the PKR activators seem to be a really promising class of agents. They seem to have a double-edged effect where they help improve red cell health and then they also help reduce sickling...
So the PKR activators are another really exciting group of therapies for sickle cell disease. So we have gene therapy, which is more of a curative or transformative therapy. But for those that want to have something more for disease modification, it’s something they can take orally at home, the PKR activators seem to be a really promising class of agents. They seem to have a double-edged effect where they help improve red cell health and then they also help reduce sickling. And preliminary data is showing that they help improve the hemolysis and anemia, and they may also reduce vaso-occlusive episodes. And so it may have two different kinds of benefits. And there’s exciting data being presented at ASH this year, again, on etavopivat. Mitapivat has had exciting data. So I think these are going to be part of the new drug therapies that we have for sickle cell disease that I’m excited for.
This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.
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