How Venetoclax Combination is Changing MDS/CMML Treatment | Alex Bataller, MD

Hello, my name is Alex Batallar. I work at the leukemia department at MD Anderson Cancer Center in Houston, Texas. And I'm going to talk about our recent study with oral decitabines of azuridine plus benetoclax for high-risk MDS and chronic myelomonasetic leukemia. So we know that these diseases are diseases with poor response rates and the overall survival is not that good. So we need newer treatments to really improve the outcomes. Technically all myelodysplastic syndromes usually are treated with what we call hypometallating agents that are azotitidine and acitabine. Those are IV or subcutaneous and they work in some cases, but the majority of time they don't produce a lot of responses and also they need patients to go into the facilities, get infusions, get subcutaneous injections. So it's kind of messy. Then newer formulations are coming into the market and actually oral decitabines of azuridine is an FDA approved therapy for high-risk MDS and CNN. So on top of that, we wanted to see if like by the addition of a novel drug called benetoclax, which is a BCL2 inhibitor, we would be able to improve the outcomes with only the oral decitabine. So that's why we presented this study. This study was a phase one to study that basically included 69 patients and we treated them with the combination of oral decitabine, cell azuridine, which is a pill, it's five days and then benetoclax, which is also a pill, 14 days. So no injections, no need to come to get like an infusion in the hospital. That's more comfortable for our patients. Now what we saw is that this combination is very effective for treating high risk MDS and CMML. 91% of patients achieve what we call complete remission either with count recovery or without count recovery. But basically what it means is that the blast percentage in the bone marrow went from like more than 5% to less than 5%, which is what we want to achieve in high risk MDS and CMML. And fortunately, a lot of these patients could go after achieving response to transplant, the allogenic stem cell transplantation or bone marrow transplant, which is what we aim candidate because this is the only like curative therapy for high risk MDS. So 55% of patients could go into transplant, which is like a really encouraging number. And among them, after transplant, a lot of patients did very well. So this combination is promising. Of course, we need to put that in context. We need to do like more clinical trials comparing what we have now. This combination, see if it's really adding anything to that. The previous therapies, see what are the side effects, et cetera. But at least it's some encouraging news for patients with high risk MDS and CMML, which definitely we need novel therapies to get these diseases out of their body. Thank you very much. If our videos have helped you in any way and you're able to, please consider making a donation to help us continue this important work. Your gift will go three times as far when we reach $500,000 by the end of the year. Every contribution, big or small, makes a difference. And we're deeply grateful for your support.