ISAL 2025 | Targeting mitochondrial metabolism in hematopoietic stem cells for the treatment of acute leukemias

So there’s actually quite a few things we try with stem cells, if I’m honest. So of course, in patients, we try to mobilize stem cells. There are treatments for that. We try to make them differentiate in certain lineages when there is cytopenia. So these are kind of mostly cytokine-driven treatments that are quite successful. When it comes to enhancing function of stem cells in vivo or ex vivo, there is not so much work being done outside of the cord blood expansion field. When it comes to mitochondrial treatments, of course, they are a really booming business. So not per se in the field of hematopoietic stem cell therapies, but in the context of treatment-resistant leukemias. So hopefully this work can kind of bridge that gap a little bit. The mitoquinol itself is freely available and also used in numerous clinical trials. These are mostly focused on neurodegenerative disorders or cardiac or skeletal muscle disorders. Not a huge amount of those trials are in hematopoiesis yet. This seems very promising and relatively safe. There are, of course, other mitochondrial drugs trying to target specifically complex one, for instance, where you really have to think more about the balance between toxicity and beneficial effect, where that has shown to be quite problematic. So of course, every cell in your body has mitochondria. Targeting them in one way is really promising because you can have transient effects if it’s non-genetic targeting and possibly you can make more personalized treatments this way. But if you’re truly targeting, you know, one of the electron transport chain complexes, you really have to think about toxicity. But the beauty of these mitochondrial-targeted antioxidants is that they don’t seem to be toxic.

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