ASH 2025 | Lessons learned from recently failed Phase III clinical trials in HR-MDS

Sure, yeah. So this is a paper that actually Amer Zeidan and myself from Yale have published. And the thought of this paper was trying to reflect on what happened in the world of higher risk MDS. We had so many randomized phase three trials with a lot of promise in early phase clinical trials. But unfortunately, this did not translate into a survival benefit. And we tried to look back at what are maybe common themes in those trials that we might be able to improve in the future. And a couple of things that I think we found was that, number one, MDS is a very heterogeneous disease itself. And the trial that maybe just includes all of those patients quite blindly might not be successful, and that we should rather look at biological subsets of MDS. We also talked about TP53-mutated MDS and CMML, two subsets that are frequently included in higher-risk MDS trials, but they do quite differently. And we should probably focus on developing dedicated drugs for TP53 mutated MDS and also for CMML. Another part we talked about was that we should apply the new response criteria that I already mentioned based on the studies that we present here at ASH. And the last point is that we really want to encourage the pharmaceutical companies, but also our larger scientific community to make data from those trials available and particularly patient-specific data or patient individual data. So we can look back and learn from those trials and see what subsets could potentially benefit in the trials of the future.

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