Dr. Deininger:
Hello, I'm Mike Deininger. I'm the director of the Versiti Blood Research Institute and a professor of medicine at the Medical College of Wisconsin.
Graphic: What current clinical trials are there for SM patients?
Dr. Deininger:
In terms of interventional trials, I think this is what we really talk about. Let me break that up into three categories. So, one is patients with SM but not ASM. So the non-aggressive form. They suffer mostly from symptoms, but their survival, length of life in other words, will not typically be compromised. So there are trials underway to see whether those patients might benefit from drugs such as avapritinib (Ayvakit).
Bear in mind, all these inhibitors or these new drugs will have their own side effect profile. So, one always has to balance the benefits of these new agents versus their potential risks. And so these trials are still underway to determine where exactly these new drugs fit in the treatment of – we call that indolent SM or ISM.
The second category is, again, ASM. And there are two, I think, avenues that the clinical trials are moving forward. One is based on what I just said. That sometimes, the mast cell component is much easier to control than the non-mast cell component. And so, in those patients, we are looking at clinical trials that combine avapritinib with other agents that are hopefully then controlling the non-mast cell component. And there is one study of this kind, in the planning stages, that I may have opened at a couple of centers already.
And then secondly, there is another molecule that is very similar to avapritinib in terms of its activity. And there is a clinical trial, at least one clinical trial on the way, developing this new agent. It's made by a company called Cogent. And so these trials are moving forward as we speak.
Graphic: Which patients with SM should consider enrolling in a trial?
Dr. Deininger:
Every patient who has aggressive systemic mastocytosis should really seek counsel at one of the centers that have experience with the disease. The Mastocytosis Society will have information available. There is, out of Europe, the European Competence Network on Mastocytosis that has a process in place that approves centers of excellence for mast cell disease. So I would highly encourage every ASM patient with newly-diagnosed mastocytosis to seek advice in one of these centers. And then, inquire about clinical trial availability and to see whether they would qualify.
Now, why is that so important? ASM, the aggressive systemic mastocytosis, is a rare disease. And so, if you want to learn and make progress, we need to make sure that as many patients as possible go on clinical trials so that we can create the knowledge and then benefit the next generation of patients. So, in other words, we all stand on the shoulders of those patients who already volunteer their time to participate in these trials. I cannot overemphasize that, especially for a rare disease.
Graphic: Should patients with SM consider enrolling in a clinical trial?
Dr. Deininger:
Absolutely. Because if you look at the last few years, with midostaurin (Rydapt) coming along as, really, the first therapy that got an approval for ASM altogether. There was nothing before that. So that did establish a standard of care, and it would not have happened if we hadn't spent decades on figuring out what is driving the disease. And with avapritinib, a second agent is available that is even more potent and more specific, to target the specific genetic lesion. And I have no reason to believe why that progress shouldn't continue. Yeah, so I think good things are happening for patients with ASM.
