Educated Patient® MPN Summit Focus on Clinical Trials Panel: May 7, 2022

This panel was moderated by Dr. Jamile Shammo, and featured Dr. Angela Fleishman and Kapila Viges.

Shammo: We'll begin with a question to Dr. Fleishman, what is the latest basic research on progression from PV to myelofibrosis and to acute leukemia? Are there any clinical trials in progress or planned?

Fleishman: So that's actually a very good question in terms of basic science, to further our understanding of why PV patients progressed, and myelofibrosis and then also to an acute leukemia, I think the sort of the leading area of research is in mutations, we do know that particular mutations will be associated with an increased risk of developing an acute leukemia. So I could say that that probably is where the focus of research is at the present time.

The second part of the question, I'm assuming, what they're getting at is, are their clinical trials, which are addressing that key question in PV as to, you know, what therapies can I take that will, if I'm in PV will prevent my progression of myelofibrosis and prevent my progression to an acute leukemia? I think that's a clear area of need. Because there's specific end points and clinical trials, which are not necessarily those very important clinical end points are my personal view. I think that in very early stages of PV or ET, things that patients can do for themselves, through reduction of inflammation, through lifestyle choices, I think, have a big impact. And that's where I'd like my research to go in the long term. But those sorts of studies would require a large number of people and a large timeframe because PV patients, this is a slow, very slow progression. So I'm sorry, I'm not really answering specifically the question. But it's the progression from PV to myelofibrosis in acute leukemia is a key area, which is understudied. And we really do need clinical trials specifically addressing

Shammo: I totally agree in that PV, in particular, pose a particular difficulty in that they are chronic illnesses. And if you want to address certain key issues, such as the one that's posing the question as to how do you actually address progression, while you need to, as you pointed out that a Fleishman, you have to follow those patients for a long period of time, and I'm not aware of any clinical trials that monitor those patients long enough. And of course, there's new evidence, new data that come up, every day that you come and you see new evidence, they've come up with, like, for example, the ion data that we've learned from you, and obviously, others, eating back a decade ago, on the importance of ion and hip siding, and things of that nature. So that also plays into our understanding to the disease. So and then we haven't even touched upon the mutation and what it means to mutation profile in those illnesses and how they play into disease progression. So it's actually more of a reason to sort of have registries and follow patients and their progression to understand what that means for each disease entity. But I couldn't agree more with the piece on inflammation, and how we combat that. And we all believe that that probably is the way to reduce the progression is just that we don't have solid evidence to say that this is actually what is going to be reducing the rate of progression because we need many years of follow up. So hopefully that answered that I can answer.

The next question, which is, what was the name of the doctor who's doing the Mediterranean diet and that is actually Dr. Fleishman.

Fleishman: So just for the caveat in terms of the clinical trial takes a while to organize It's an open, we've completed two pilot studies, we're in the process of developing a much larger study in a diet, it's not currently open, we honestly we need to find funding for a large study, and get all the approvals. So we don't necessarily have a diet study open right this minute. But I just wanted to tell the audience that is in development. And, but if you are interested, please email me. And then I can keep you on the list for when we do actually open and we could contact you and the large city will be an online study, because we realize that, you know, in order to reach people around the United States that really does need to be online, rather than requiring people to actually come in person.

Shammo: And then Kapila, can you address the next question, and it is discussing this issue about cost? So can you talk about the cost of clinical trials? And how was that addressed?

Viges: Sure. No, It's a wonderful question. And, and it can be a little difficult to navigate. But again, getting as much information upfront will be helpful. So for the most part, there are core elements of a clinical trial, that typically the sponsor does cover. So for example, the cost of the drug itself, the cost of any tests or screens are studies that have to be part that are part of the study itself that are required, oftentimes, those costs will be covered by the sponsor, or the institution where the study is taking place. Sometimes, there will also be some additional stipends for things like travel or accommodations that can but those can sometimes be few and far between. And so you do want to understand what impact that might have for you before you go into this into the study and ask all of those questions of the specific site director or your point of contact and there to understand what are all those pieces? Certainly, we know, you know, if there is travel or time away, there's an impact perhaps on your work-life balance a little bit. And so there may be some indirect costs to that. So really understanding for each specific trial, what it's going to under what it's going to take is important. But there are some core elements of it that are covered by this by the sponsor.

Shammo: Thank you. And then Dr. Fleishman, I liked very much what you had said about phlebotomy. And we all understand that it's a cornerstone of treating patients who have PV to reduce the amount of you to below 45%. But the point about hematocrit not being below 45%, at all times is very well taken. And it sort of makes me think when I take care of my patients that there are certain times were when they are not protected. They're only treated by phlebotomy. How do you address this? And does that make you want to put them on cytoreduction? If they require frequent phlebotomy is?

Fleishman: I mean, I guess I don't have a great answer for that. From my perspective, I guess some patients and then the physician can sort of get an idea about their required cycle of how long it takes them their medica to sort of trend back up after a phlebotomy. So in some patients, you can sort of anticipate what their trajectory will be. And then, you know, scheduled DVCS and, you know, appointments of the blood donor center accordingly, I guess that's been my approach that sort of what what's the patient's style in terms of how does their how quickly does a Connecticut come up after each phlebotomy? Because that's sort of an unscientific approach that I just sort of guessed, anticipate when the Hermetica is going to come up. Or more frequent CVC. I mean, another issue is, a lot of times patients won't be able to tell how, I mean, some people won't, but some people will be very attuned to their body and know exactly when their hematocrit is coming up based on their symptoms. So, that's another approach that a lot of patients won't really know. But I don't have a good answer for you, other than sleeping on their phone commander crits. Frequently and knowing each person how, how quickly it doesn't matter if it comes up after phlebotomy.

Shammo: And then one last question for Kapila before we wrap up. What about accepting Canadians in trials?

Viges: It partly also depends on the overall site and each individual trial itself. So unfortunately, it's a great question, but I don't have a great answer for it. It is possible it has happened. But again, it is very trial specific.

Shammo: And it may have to do with insurance, as well. And yeah, exactly that may have to be governed by that. So that always needs to be done by insurance. All right, thank you very much for participating and for providing your expertise on this particular topic. Why don't we have final thoughts from each one of you starting with Dr. Fleishman and then I'll turn it over to Kapila.

Fleishman: From my perspective, I think this is a very exciting time for polycythemia vera and myeloproliferative neoplasms. Because we're really learning much more about the biology. And before getting, in order to really figure out clever ways to address clinically addressed the problem. We need to know the basic science and I think we're getting the basic science, and then I'm excited that very soon afterwards, then there'll be just the explosion of, of new ways to I mean, ideally, I mean, it's been wonderful to cure polycythemia vera. We're not quite there yet. But I think, you know, that would be a great goal.

Viges: Yeah. So to echo what Dr. Fleishman said, it is a really exciting time, there is quite a bit in development. Yet there is more to be learned and more to discover and understand. So a good time to be very optimistic. And just leave you with the participation in a clinical trial actually, is empowering both for your own treatment for understanding your own options and advancing your own care. But really is part of, I'll say, the greater good and advancing the overall research paradigms, because successful trials can make those treatments available to more and more patients so you can feel empowered both ways that not only you're doing good for yourself, but you're doing good for the overall disease area and population as well. So thank you.

Raw transcriptions have been lightly edited for clarity.

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