What is the goal of treatment (manage symptoms or cure MDS)?

What is the goal of treatment? Is it to manage symptoms or cure MDS? Yeah, so for MDS, the only known curative option is a stem cell transplant. And so that is still our goal for all of our patients who are eligible for that. But we know that MDS typically occurs in older patients. Age is not a limit to the stem cell transplant, but you do accumulate other medical problems as you get older. And some of those heart conditions, kidney conditions may make somebody not the ideal candidate for a transplant. They may have too many side effects from a stem cell transplant. So for patients who aren't eligible for a stem cell transplant, we don't have a curative treatment. But the goal is to prolong survival and make the patient's lives better during that time. So the only, again, we use randomized controlled trials and the only drug that has been proven to be better than, say, just doing nothing supportive care is azacitidine. This is a hypomethylene agent used in MDS and AML. And it is the only drug that we have for frontline treatment of MDS. It is able to put a good number of patients into remission. Say, 60% of patients can go into remission with azacitidine treatment. And so that's encouraging, but we could always do better. So I think the newer studies are looking at adding different drugs to azacitidine to try to improve the remission rates. And also, it did prolong the survival, but we know that it doesn't cure. So can we push that survival further and further? And if patients relapse, what are our second line options? Currently, there's no approved second line options. They may get different forms of therapy. But so that's another area. So combining things with azacitidine in the front line and then looking at post azacitidine relapse treatment options. And it's similar to AML. I'm excited because there are lots of new immunotherapies and other drugs being studied. We haven't had quite the number of breakthroughs as AML, but I think it's coming around the corner. You know, there were a few phase three trials of combining agents with azacitidine that didn't quite meet the mark. But they were encouraging enough that I think ultimately we'll get there in terms of the combinations. And mainly this is for high risk patients with MDS. Again, we have risk stratification for MDS into different risk categories. You can have actually up to five different risk categories. But to keep it simplistic, we typically think of lower risk and higher risk. And so someone with lower risk, they may not need azacitidine treatment. They can be managed with ESA's erythropoietin stimulating agents. If the main symptom is anemia, then this helps boost the hemoglobin and the red blood cell count so that the patients don't have to get transfusions. There's also a new drug approved recently called Luspattercept. This is for patients after the ESA's stop working. A number of patients in clinical trials were shown to respond in terms of anemia to the Luspattercept. And so the goal in the lower risk MDS patients is just to prevent needing transfusions, needing to come into the hospital, try to keep the patients functioning at home. Ultimately, they may progress to a higher risk MDS from lower risk and then they may need the azacitidine treatment. So I think it really depends on the risk of the patient and what their main cytopenia is. So is it mainly anemia? Is it mainly thrombocytopenia or low platelets? Or do they also have neutropenia? So we take all those into consideration when choosing the treatment. The goal in treating myelodysplastic syndromes really depends on the type of myelodysplastic syndromes a person has. And in broad strokes, we divide this into lower risk myelodysplastic syndromes or higher risk myelodysplastic syndromes. With lower risk myelodysplastic syndromes, people typically have more mild blood count abnormalities, may or may not even require transfusions, and may or may not even require therapy. If we do initiate therapy in somebody with lower risk myelodysplastic syndromes, our goals are to minimize or even eliminate transfusions and to improve that person's quality of life. To date, no study has ever shown that a drug intervention in somebody with lower risk myelodysplastic syndromes has improved that person's survival. So we focus on quality of life, minimizing transfusions. For somebody who has higher risk myelodysplastic syndromes, so more profound blood count abnormalities, an excess in cells called blasts in the bone marrow, blasts are immature white blood cells. We say that a person has too many blasts when they have more than 5% blasts in the bone marrow. Once somebody has 20% blasts in the bone marrow, we say that person has acute leukemia. So you can guess where I'm going with this. In somebody with higher risk myelodysplastic syndromes, our goal is to prevent the myelodysplastic syndromes from converting into acute leukemia and to prolong that person's overall survival and always, always, always to try to improve somebody's quality of life.