Preventing Progression in MPNs: Researcher Perspectives From ASH 2017

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Topics include: Treatments

What treatment options are available to thwart progression in MPNs? Noted MPN experts, Dr. Angela Fleischman from UC Irvine Health, and Dr. John Crispino from Robert H. Lurie Comprehensive Cancer Center of Northwestern University, share their insights on clinical data released at the American Society of Hematology (ASH) 2017 annual meeting related to disease-modifying agents and treatment strategies that may impact MPN progression. Watch now to find out the latest.

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Andrew Schorr:

Well, first of all, there are some people with some of those conditions (ET) who may not be getting treatment really. They may be taking aspirin. So they say, “Well, we don’t know if we’re gonna progress to this sort of more serious condition, but what medicines are there for us?” 

So we mentioned about interferon. Angela, do you want to mention about that? There was some research about interferon and what role that plays. 

Dr. Fleischman: 

Yes, and I think interferon is a very special agent in MPN. We really don’t know exactly why it’s so useful in MPN. But, in my opinion, interferons have the best evidence as a disease-modifying agent; meaning have an actual impact on the disease course. So I was really excited to see the data for ropeginterferon, because interferon alpha can be a little difficult for some patients to tolerate, given its side effect profile. So if we could have a drug that has the benefits of interferon, but not the side effects, that would be really, really great.

It looks like ropeginterferon can fit that bill in terms of it seems to be quite well tolerated and does have the same effects as interferon, like Pegasys, the pegylated interferon alpha. And I think you brought up a very good point, is that a lot of clinical trials really focus on myelofibrosis patients who have failed other treatments, but there’s less enthusiasm or fewer clinical trials looking at very early staged patients and trying to block the progression.

In my mind, I feel like it would be a lot more beneficial to start treatment early, so as one will block progression, rather than waiting until somebody progresses and then trying to fix it. It just seems, to me, a lot easier to fix a problem that’s very big—a small problem than a big problem. 

And I think that if I were to sort of want something in terms of where I think that the efforts in MPN should go, it would be to really focus on the early stages of disease and work on ways to prevent progression. 

Andrew Schorr:

Dr. Crispino, I know you’re a medical advisor to the MPN Research Foundation, so talk about what she was just saying related to helping a broader array of MPN patients and earlier as well.

Dr. Crispino:       

Absolutely. That’s a very important aspect of the disease, this progression from PV or ET to myelofibrosis or even to acute leukemia. And we don’t understand the mechanisms by which people transform. I would also just say that some of these therapies that are investigational are being tested in myelofibrosis, because the side effect profile would contraindicate taking this drug for 10, 20 years. 

So whatwe’d like to do is uncover the pathways that drive progression and then develop drugs that would prevent that, and hopefully drugs that will have less toxicity. So the MPN Foundation has initiated a program called the Progression Project, which is to study a number of patients, could be up to 1,000 or more, to look at blood samples from patients over time and also follow clinical progression as a way to study biologically what happens to these cells as people age or people progress with this disease.

Hopefully we’ll find something. Maybe we’ll find a gene that gets mutated during the transition, or maybe it gets mutated early in transition, and then we might be able to develop drugs that would stop that gene from getting mutated or stop the malignant effects of that mutation. And so we’re gonna be looking at the patient community to help join this effort and sign up to be part of this Progression Project. 

Andrew Schorr:

Well, I’m all for that. I think most of us watching, we understand now as patients that we’re in partnership with you. 

We’re in partnership with Dr. Fleischman and your colleagues around the world. Often you need blood and tissue samples and things like that so that you can try your concepts on and see what may be working. I would urge everybody to be plugged in to what the MPN Research Foundation is doing, the registry, and putting together these different initiatives because we keep waiting for a new drug to be approved or a late state clinical trial that maybe is available near us, but you all are in the lab trying to get us to that point, and we need to be your partner in that. 

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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Page last updated on March 22, 2018