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MPN Workshop of the Carolinas 2025 | Translating CHIP research into preventive or therapeutic interventions

In this video, David Steensma, MD, Ajax Therapeutics, New York, NY, comments on the challenges with translating clonal hematopoiesis of indeterminate potential (CHIP) research into preventive or therapeutic interventions. Dr Steensma notes that current research has primarily been descriptive and emphasizes that any interventions developed for this precursor condition would need to have a highly tolerable safety profile. He highlights that ongoing trials focus on individuals with clonal cytopenia of undetermined significance (CCUS), and that findings from these studies could potentially be moved into the CHIP setting in the future. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.

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Transcript

One of the challenges with CHIP and CH more broadly is that so far it’s been primarily descriptive. There’s been a lot of associations with different disease states and with clinical outcomes, but there are as yet nothing interventional that can be done for it. And that’s hopefully an area in the future that can be explored. One of the challenges is that because clonal hematopoiesis is so common, if there is an intervention, it would have to be something that has a very, very benign safety profile...

One of the challenges with CHIP and CH more broadly is that so far it’s been primarily descriptive. There’s been a lot of associations with different disease states and with clinical outcomes, but there are as yet nothing interventional that can be done for it. And that’s hopefully an area in the future that can be explored. One of the challenges is that because clonal hematopoiesis is so common, if there is an intervention, it would have to be something that has a very, very benign safety profile. You know, something that has certainly no more of an adverse event profile than the statins for cardiovascular disease, for instance. And there are some early explorations, for instance, into inhibition of the NLRP3 inflammasome around clonal hematopoiesis in patients with established cardiovascular disease, there are some ongoing trials. But more commonly, more of the trials are focusing on patients who have clonal hematopoiesis and established cytopenias, CCUS, it’s called. And they don’t quite have overt MDS yet, but they do have a risk for progression similar to lower-risk MDS, and they sometimes run into problems with symptoms or complications of their cytopenias. So there was a vitamin C trial, for instance, in CCUS, which looked interesting. There’s some trials of specific inhibitors like IDH inhibitors in IDH-mutant CCUS and some other very interesting studies being developed as well. So, you know, we’ll see how this all shakes out, but we have to walk before we run. So CCUS is probably the test lab for things that ultimately may move into clonal hematopoiesis without cytopenias.

 

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Disclosures

Employment: Ajax Therapeutics.