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MPN Workshop of the Carolinas 2025 | The potential challenge in integrating imetelstat into the MPN treatment landscape

In this video, John Mascarenhas, MD, Icahn School of Medicine at Mount Sinai, New York, NY, highlights that the key challenge in integrating telomerase inhibitors, specifically imetelstat, into the treatment landscape of myeloproliferative neoplasms (MPNs) may be the potential for cytopenia, particularly in patients with advanced cytopenic disease. However, Dr Mascarenhas notes that initial data from the combination of imetelstat and ruxolitinib do not indicate a significant risk of cytopenias. Utilizing the drug earlier in the disease course may also potentially reduce the risk for this complication. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.

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Transcript

So I think probably the biggest challenge is the potential for cytopenia. So patients who have very advanced cytopenic disease may be challenging to give this drug, because there is some on-target cytopenias that can occur, not every patient. It would be interesting to see the data mature, but the initial data we’re seeing with combination ruxolitinib does not seem to have significant cytopenias, which is somewhat surprising...

So I think probably the biggest challenge is the potential for cytopenia. So patients who have very advanced cytopenic disease may be challenging to give this drug, because there is some on-target cytopenias that can occur, not every patient. It would be interesting to see the data mature, but the initial data we’re seeing with combination ruxolitinib does not seem to have significant cytopenias, which is somewhat surprising. So as I said in the previous question, right now the Phase III focus is a single agent in the relapsed/refractory setting with survival as an endpoint. But if you were to ask me where would this drug fit maybe the best, it might be with our earlier disease, suboptimal responders. And again, if the data pans out, maybe cytopenias won’t be such a vulnerability.

 

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Disclosures

Paid steering clinical trial committee member for Geron.