What Does the Future Hold for MPNs? Experts Weigh In

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Topics include: Treatment and Understanding

At ASH 2016, MPN experts from across the nation share their perspective on what the future holds for MPNs, in both the short term and long term.   Dr. John Mascarenhas from Mount Sinai School of Medicine in New York City, Dr. Catriona Jamieson of UC San Diego Health Moores Cancer Center, and Dr. Stephen T. Oh of Washington University School of Medicine provide an optimistic outlook on MPN research and treatment.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

Andrew Schorr:

Where do you think we'll be in one year or five years—whatever window you want to take? Because we're hoping we're going to live a long time with any of these conditions. So, Stephen, I'll start with you. Where are we headed, do you think? Give us a timeframe and where you think we'll get to. 

Dr. Oh:

Well, I think, in the short term—meaning the next one to two years—I think it's likely and I hope that we'll gain clarity as to the role of some of these different JAK inhibitors in this field. We're getting there. I think, on the one hand, there's the temptation to say, well, they're all the same, but it's clear that there are distinctions between the different drugs in this class, and there could be different niches and reasons to use one or another. But it's an interesting time in that ruxolitinib (Jakafi)—being approved now more than five years ago—still remains the only JAK inhibitor that's approved for treatment for patients of myeloproliferative diseases 

So I think that's the short term what I'm looking for. In the longer term, I think we've talked a lot about combination therapies. But I think, more broadly, is to get to a point where we can confidently say that, with whatever treatment we're using, we will be able to do more in terms of not just spleen responses, not just symptom responses—see actual colonial reductions and be really, truly modifying the underlying disease.

Andrew Schorr:                  

Catriona, what about your time window and where you think—where you hope—we'll be?

Dr. Jamieson:      

I think things have happened remarkably quickly and I think, again, it's because patients have been involved in asking those tough questions that we can't avoid in clinic so it makes the clinic visits a little bit longer, but that's part of the science. So if we partner in that, I think it's really important.

But I think the good thing is, with some of these studies that we're all doing—John, Stephen and I—is that we're learning that some people don't progress. Some people just sit there. They're fine on ruxolitinib. Why is that? So, in science and medicine, we tend to be like Debbie Downer on “Saturday Night Live”—everything's bad. Just think of the worst-case scenario. But there [are] some very good case scenarios where people aren't progressing, so we need to study the good aspects of people's genomic makeup and their immune system and say why is their clone being kept in check? Because that's pretty interesting, and I think we need to delve more deeply into that. 

The other thing is some of the extrinsic signaling factors that drive the progression of MPNs to AML come because of external factors whether it be changes in the microbiome or even viral infections. So I think chronic inflammation, if curtailed or prevented, could actually prevent progression, and that's based on understanding the radar for viruses in our system and for inflammation which is called ADAR-1.

So keep that on your radar, because that's the editing system for our cells, and it's one of the pathways that the stem cells hijack to make them truly malignant. We have ways of blocking that—JAK-2, interestingly, can dial down activity on that—so I think that staying as healthy as you can, getting a flu vaccine, staying clear of bacterial pathogens that engage pylori, really, really important, staying physically active. Those are the seemingly low-brow things, but they really matter. So I think the healthier you can stay, the better and I think that there is an effect, as John was alluding to, with the immune system that we need to understand how to corral the immune system, either the person's own immune system or bringing in some extra help from an immune support.

Andrew Schorr:                  

Okay. But you're hopeful? 

Dr. Jamieson:      

I'm very hopeful.CD47 is another one—so, as cells start to get malignance, they start to upregulate the "don't eat me signal," so the garbage trucks of the immune system can't eat them. 

There's an antibody to that now. There's an antibody to another stem cell factory called RORA-1. So it's just knowing when and where to use them and then boom. And that's the piece that we're all grappling with right now, but that's the partnership—that's where Patient Power matters.

Andrew Schorr:                  

Well, thank you. John, so where we headed in a year, five years?

Dr. Mascarenhas:               

Like Catriona, I think I'm optimistic as well. I have to be optimistic because it's what brings me to work every day. And I think that the scientific understanding that occurs today and is reported in journals today is really what feeds the pipeline and changes the way we approach the clinical trials of tomorrow. So I do really think that the trial—if we were to have this round table in five years, I think the discussion—I would like to think it would be totally different—in terms of the agents being discussed, the pathways being focused on, and that we'd probably look back and say, oh, we didn't even realize what laid ahead. 

Because that's really what's exciting is, within a year or two, the understanding could triple and then the ability to target something that was never even thought of today would be present tomorrow. So I think there's a lot of hope, and I think that it's only going to be accomplished by patients engaging with their physicians and contributing to tissue banks so that researchers can better understand the underpinnings of the disease, and then investigators can better employ therapies to benefit patients.

Andrew Schorr:                  

Hmmm. I thank all of you for being here—Stephen, from St. Louis, and Catriona, from San Diego, and John, from New York—and your colleagues around the country for your devotion. And, just speaking maybe for the patient community, just Godspeed, and we will partner with you. And I think the key message, for us in the community, is consider seeing a specialist—highly consider—seeing a specialist at least for a consultation.

Consider donating your blood for their research, spit in the tube if that helps, and help them get us where we need to be in getting precision medicine. But it's hopeful—there's no big, big announcement, I don't think. I'm not hearing this year—we heard about CALR another year and different genes being discovered—no big headline but I think continued progress in partnership with us. So thank you so much for joining us. And I'm Andrew Schorr, as I like to say, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

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