MPN Workshop of the Carolinas 2025 | The unique challenges that underlie the lack of therapeutic advances in CMML to date
Eric Padron, MD, Moffitt Cancer Center, Tampa, FL, comments on the challenges of developing therapies for chronic myelomonocytic leukemia (CMML), a rare hematological malignancy. Dr Padron highlights that CMML has had a non-linear classification history and was previously considered a subset of myelodysplastic syndrome (MDS), resulting in limited data on the efficacy of approved therapies specifically in the CMML population. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.
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Transcript
Yeah, so CMML is a rare cancer and rare cancers in general are challenging to develop therapies. I always say by the National Cancer Institute definition, CMML for sure is a rare cancer. But if you look at all cancers, 30% of all cancers would be defined as a rare cancer, or 30% of patients who have cancer have a rare cancer. And so, you know, I think what is unique to CMML, though, is not only is it rare, but it’s gone through a non-linear classification history where it used to be called another disease, a subset of another disease, namely myelodysplastic syndrome, then was within the last 15 years decided it was its own disease...
Yeah, so CMML is a rare cancer and rare cancers in general are challenging to develop therapies. I always say by the National Cancer Institute definition, CMML for sure is a rare cancer. But if you look at all cancers, 30% of all cancers would be defined as a rare cancer, or 30% of patients who have cancer have a rare cancer. And so, you know, I think what is unique to CMML, though, is not only is it rare, but it’s gone through a non-linear classification history where it used to be called another disease, a subset of another disease, namely myelodysplastic syndrome, then was within the last 15 years decided it was its own disease. And I think that’s provided a lot of challenges in that many of the clinical trials include CMML, but are really MDS studies. And there are approved therapies here in the United States that are approved for CMML, but the study was never intended to or powered to determine whether that CMML group was particularly responsive relative to the broader MDS population. And so, you know, rare cancer, that has unique challenges, but a rare cancer in which we’ve jumped around how we call it is even harder.
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