ASCO 2015: Discussing MPN News With a Roundtable of Experts

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Topics include: Treatment

This coverage from the 2015 annual meeting of the American Society of Clinical Oncology (ASCO) is from our partner, the Patient Empowerment Network. MPN experts, Dr. Ruben Mesa, Dr. Alison Moliterno, Dr. Naveen Pemmaraju and patient advocate Heidi Cascarano gathered to discuss the latest news in myeloproliferative neoplasms (MPNs). The experts spoke about building on the JAK2 inhibitor story, how allogeneic stem cell transplant is showing promise in certain types of myelofibrosis patients, data from trials presented at ASCO, and progress in trials currently in progress, among many other topics. Dr. Mesa and Heidi shared hope for the future and stressed the importance of partnerships between physicians and patients, especially for those with MPNs. As noted by the experts, this is an exciting time in the field of MPNs, with new classes of drugs and combination therapies under study.  

Sponsored by the Patient Empowerment Network though an educational grant from Incyte Corporation.

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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.          

Dr. Mesa:

Hello.  I'm Ruben Mesa, and it's a pleasure to be leading this roundtable as we discuss updates from the American Society of Clinical Oncology, and I'm joined today by a wonderful roundtable of friends and colleagues.  To begin with, Alison Moliterno from Johns Hopkins School of Medicine in Baltimore, Dr. Naveen Pemmaraju from MD Anderson in Houston, and we're very pleased to have Heidi Cascarano, an MPN patient advocate diagnosed with polycythemia vera back in 2008 and subsequently now has myelofibrosis. So we're particularly lucky to have Heidi's experience and expertise with us. 

So as we discuss the American Society of Clinical Oncology, to frame it for people watching this video, this is probably the largest oncology meeting in the world.  There's a little over 30,000 physicians, nurses, scientists, pharmacists all here in Chicago really covering the entire world of cancer, everything from brain cancers, pancreatic cancers, blood diseases and even myeloproliferative neoplasms. 

So maybe to begin with I'll ask Naveen, you've been integrally involved in tracking the MPN news from this meeting.  What things have gotten you excited that you've seen here at ASCO? 

Dr. Pemmaraju:

Well, thank you, Ruben.  I think some of the major developments at ASCO this year for MPN have been very exciting for our patients and for our families with MPN.  Two of the major ones are building on the JAK2 inhibitor story, which is a new JAK2 inhibitor besides the one that's already FDA approved, and that's a drug called pacritinib, which actually you presented in oral presentation yesterday.  And a second exciting development is more maturing data from the perspective of allogeneic stem cell transplant, and that is in patients specifically with myelofibrosis, and that was presented by Dr. Uday Popat from MD Anderson yesterday as well.  I think these two developments are some of the more exciting developments for our patients with MPN. 

Dr. Mesa:

Very exciting.  So, Alison, you've been integrally involved in particular with the genetics of polycythemia vera, the evolving story in polycythemia vera, the therapies for polycythemia vera, Serge Verstovsek is presenting a poster today on the updated data with RESPONSE, the randomized study with ruxolitinib (Jakafi) versus best alternative therapy in people with advanced disease.  How do you feel now that ruxolitinib has been out available for patients?  How has that experience been going? 

Dr. Moliterno:

Well, as we spoke earlier today, this is the 10th anniversary of the JAK2 discovery, the 10th year we've had this knowledge, and it's been wonderful to see this knowledge applied so quickly translationally to therapies.  We can diagnose patients better, and now we have targeted therapy. So this has really been an amazing decade for this—for the MPN.  And even in our own practices now, we are no longer thinking about it or talking about it. We're actually using this knowledge in treating patients. 

For me personally and my patients, targeted therapy has really been a boon.  First of all, it's given a lot of hope, but it's also translated into better quality of life, more tolerable disease and symptoms.  And there's ultimate hope that maybe the disease will really change its natural history now with these targeted therapies. 

Dr. Mesa:

Very exciting.  Very exciting.  Now, Heidi, as these medical meetings occur, there's a lot of information that comes out on the news, you know, whether it's at CNN or things of that nature.  As you hear about different things in medicine from the perspective as it could impact you, how do you learn about things that are occurring through MPNs?  Did you learn them from your doctor or website, patient groups?  How do you find out about this information? 

Heidi Cascarano:

I was very fortunate to discover a lot of support groups online early on, and it's so important for patients to do their own research, and—from a lot of different reputable sources and good hospitals.  So patients actually are always really excited when these conferences are going on, and they're following online on Facebook on these groups I'm on, posting, you know, scientific papers, and it's a really educated group of patients that have MPNs. 

They read the scientific papers, and they share information.  They share information about doctors, so any news coming out of these meetings is really important to the patients.  I mean really, they follow it very closely, the latest research. 

Dr. Mesa:

So, Naveen, you had mentioned before specifically some important information presented by your colleague at MD Anderson, Uday Popat, wonderful guy, a tremendous bone marrow transplanter for MPNs.  We're really lucky to have him in our field.  What were some of the key take-aways from Uday's presentation? 

Dr. Pemmaraju:

Sure.  The background is that in our disease areas, myeloproliferative neoplasms, the only curative option at this point we think of is the allogeneic stem cell transplant.  Unfortunately, the problems with allogeneic transplant, even as it's improved over time is the attendant risks, side effects, toxicities, and so a vast majority of patients may not be eligible for it. 

However, in those patients who are able to tolerate and go for it, Dr. Popat presented on a group of patients followed at MD Anderson, at our institution, with a median follow-up, that means approximately an average follow?up of five years, which is actually a quite a nice follow-up to be able to look at.  This was a group of patients who had to be healthy and fit enough to go for allogeneic transplant, and what he showed in this data set was quite remarkable, that there is a group of patients who benefit, that possibly the intermediate 2 risk, that is the folks who have not the lower risk or the high risk but in-between group of patients, tended to do very well or better with this approach, and I thought that was very exciting. 

Lastly, he also showed that the rate of toxicities that's graft?versus?host disease or GVHD, were well?tolerated in these group of patients, both the acute and the chronic phases.  So I think the summary statement was it's another large institution study that shows that myelofibrosis can be applied in the right patient with myelofibrosis and that they can have a long-term good quality of life and follow-up even after the transplant. 

Dr. Mesa:

It is certainly one of the challenges with the issue of transplant in patients with myelofibrosis in that it seems that likely from his data the best time to consider it is really before the disease is really in its most advanced forms.  I think everyone feels the most comfortable, I'm going to hold on to that option until I'm really sick and really need it, and it seems that doing that might be delaying it a little bit too much. 

I presented yesterday the data from the PERSIST study, interesting study also with this issue of advanced patients.  This was the first randomized study that allowed patients with very advanced disease features of low platelets to receive this JAK inhibitor.  So it was a two?to?one randomization of over 300 patients globally, randomized between pacritinib and best alternative therapy. 

And what we saw is that individuals were able to obtain benefits in splenomegaly and symptoms, but additionally that it was safe to use in patients that had really low blood counts, in particular low platelets.  So very interesting information, may well lead to that drug being available for that group of individuals. 

Additionally, there is a parallel study ongoing that Serge is leading.  All of us are involved with that study, the PERSIST 2, study for individuals that have either been on JAK inhibitors before as well as having very low, low platelet counts. 

Now, Alison, many different trials are ongoing at the moment.  There are many different combinations.  I'm sure patients watching this are mindful that there are many different drugs being discussed, all sorts of combinations with ruxolitinib as a base, new drugs on their own such as imetelstat.  Why don't you just kind of give a snapshot?  You know, are these things, are they ready for prime time, are they still very much in the experimental phase?  Where do you think we stand with kind of this pretty broad pool of MPN trials? 

Dr. Moliterno:

Yeah, that's a great question, and something we all have to attend to in our meetings with patients about, well, what can we deliver locally and globally, right, what's going on for the best treatment?  One thing I think we've learned from the diseases and from these trials is that we always have time to consider what would be best for an individual.  You really have to personalize to individuals.  Are they willing or able to travel to maybe enroll in a trial, or—or not. 

I also have come to realize with patients that an individual patient is going to have these diseases a long time, which is a good thing, they're indolent, but that doesn't preclude them from trying different things at different points of their disease.  I think that's one thing that we both experienced with these long-term patients, that you may participate in a clinical trial and that doesn't shut off other treatments in the future for you. 

It's a very exciting time, and a lot to talk about in individual sessions, right?  Some of these diseases, the new drugs are totally different classes, and again are we looking for a cure for the disease or palliation?  And so a lot to weigh but very exciting times. 

Dr. Mesa:

It's very true.  I think you raised a couple excellent points.  One, the MPN community is incredibly diverse.  It's individuals that are late teenagers to individuals that are in their late 80s or 90s and have incredibly different perspectives, different diseases, different ways that it impacts them, you know, and being mindful of everyone's journey as well as a lot of different individual factors are relevant. 

I sometimes will find that people are incredibly passionate about one specific treatment, and again almost without exception I can think of any of the therapies we've used in which there are some patients who have done really well and others in which it really has been a very poor fit. 

Now, Heidi, physicians spend a lot of time assuming they know what patients are looking for as outcomes in a therapy, sometimes not necessarily with asking the patients first.  I've seen people stand up, you know, and sometimes give a relatively silly perspective that they view their judgment on what is meaningful for patients supersede the patients themselves.  So someone who is afflicted, and obviously you're a tremendous stakeholder in all of this process, what are the things that are meaningful for you?  As you hear about new therapies or what impact they have, what things really resonate with you that you think matter? 

Heidi Cascarano:

Well, I do know a lot of patients that are on the wait-and-watch protocol, so to speak.  I personally am a big advocate of being proactive and fairly aggressive.  I had a pretty aggressive disease myself, I progressed pretty quickly, and I have probably been a thorn in my doctor's side because I have decided, you know, with his help to attack my disease pretty strongly, and it's been going really well. 

And I mean every patient is so different, though, and I realize that, and different treatments don't work, but I think a lot of patients are very excited about the combinations, hoping that they'll reverse the disease process, reverse the fibrosis and the early myelofibrosis.  So that is my goal is reversing that process, and also having really good quality of life.  I mean that's—that's huge so I can keep working and keep enjoying life. 

Dr. Mesa:

Well, great.  I think that's a great perspective, and I think really helps to kind of summarize our goals as a whole.  The process of scientific advance as I chat with patients is a little bit like watching sausage being made, as they said.  You know, there [are] ups and downs, and sometimes for some pulling back the curtain on that process can be a little too much information for folks, but I do think that the partnership between physicians and patients, particularly in these diseases is really key. 

A lot of the advances have really been made by patients being so generous with their time or involvement in trials or allowing their blood to be used for research.  All of these key advances have really come from those sorts of efforts. 

So I'd summarize to say that a lot of exciting things going on in the field of MPNs.  This summer there continues to be more and more information.  Some information shared here at the American Society of Clinical Oncology very soon will be in Europe for the European Hematology Association, another meeting where many advances in MPNs are discussed.  So keep tuned to Patient Power for further updates. 

I very much would like to thank our roundtable members, Alison, Naveen and Heidi, for sharing of their time and talents today.  Thank you.  

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 December 17, 2015