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EHA 2026 | The future of precision medicine in MDS: genomic profiling and venetoclax-based strategies
Alex Bataller, MD, PhD, The University of Texas MD Anderson Cancer Center, Houston, TX, discusses the future of precision medicine in myelodysplastic syndromes (MDS), emphasizing the need for a patient-centered approach that accounts for the disease’s heterogeneity and complex biology. Dr Bataller also reviews the VERONA (NCT04401748) trial of hypomethylating agents (HMAs) plus venetoclax, noting that the study did not meet its primary endpoint, and future research should focus on identifying MDS subgroups most likely to benefit from this combination. This interview took place at the 31st Congress of the European Hematology Association (EHA) in Stockholm, Sweden.
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Transcript
Yes, so I do believe that we are going towards an era in which we are not going to treat all MDS the same. We know that MDS is very heterogeneous, so there are patients with different mutations. Also, epigenetically, it’s a very complex disease. So we are treating all these patients the same, and this in this era makes no sense, so I believe that we are going into an era in which treatments, also prognostication, is going to be patient-centered, we’re going to be centered on the patient, so we’re going to decide which clinical trial, what is the prognosis, etc...
Yes, so I do believe that we are going towards an era in which we are not going to treat all MDS the same. We know that MDS is very heterogeneous, so there are patients with different mutations. Also, epigenetically, it’s a very complex disease. So we are treating all these patients the same, and this in this era makes no sense, so I believe that we are going into an era in which treatments, also prognostication, is going to be patient-centered, we’re going to be centered on the patient, so we’re going to decide which clinical trial, what is the prognosis, etc., based on the biological characteristics of that disease. In AML, HMA venetoclax is the standard of care, as it has been demonstrated that it’s better than HMA alone. But in MDS, although early clinical trials were showing promising results, the VERONA clinical trial really didn’t meet its primary outcome, which was overall survival. So the main question now is to understand why VERONA failed and likely identify which subgroups of patients, maybe genomically defined, maybe because of clinical characteristics, there should be some patients that may benefit from the addition of venetoclax. Maybe not all patients with myelodysplastic syndrome, but some of them may benefit. So we need to identify and understand who these patients are to treat them appropriately. Likely, I think that novel treatment combinations are going to come into the MDS field that actually will provide some insight in terms of which treatment maybe added to HMA, maybe not added to HMA, could impact the response rate and also survival in MDS patients. So likely, novel treatments will need to be explored to see if they are useful in the field of MDS.
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