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Advancing MPN Treatment Through Clinical Trials

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Published on July 10, 2014

At a roundtable discussion from the 2014 American Society of Clinical Oncology (ASCO) meeting, Dr. Ross Levine from Memorial Sloan Kettering Cancer Center and Dr. Olatoyosi Odenike from University of Chicago Medical Center joined Dr. Srdan Verstovsek from MD Anderson Cancer Center to review the latest advancement in the treatment of MPNs.  The experts examine how the development of ruxolitinib (Jakafi®) has influenced enrollment in clinical trials. They also discuss the promises of combination therapy and how DNA tests will affect treatment moving forward. 

Sponsored by Incyte Corporation.              


Transcript | Advancing MPN Treatment Through Clinical Trials

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. Odenike:

I mean, I think it’s very exciting that we have these many trials going on. This was a disease, myelofibrosis in particular, that was an orphan disease, I mean, not much attention was being paid to it.

So it is wonderful that we are in an era, as Ross mentioned, where we can actually test so many different agents as single agents in combination and in parallel. And it would be very nice to see whether the next couple of years bring, in terms of, you know, which ones seem to be the most promising.

Dr. Levine:

I think the other really important lessons so far are twofold. One is that patients are enrolling in these trials at a very high frequency just because they’re getting benefit from ruxolitinib (Jakafi). They’re excited about that, but they want more. And I think we’re responding as doctors, as companies, as clinical trialists, as laboratory scientists, to that need.

So I think you’ll see this clamoring for combinations to challenge all of us to give patients new opportunities and I think that that, to me, is the most exciting thing moving forward is that the patients are really voting with their feet, if you will, and they’re joining the trials, they’re looking for more. The other really important question—and I think is something that maybe you both have thought about as much as I have—is going to be, when we do combinations, how do we do them?

Do we only take patients who never started any drug, and then we start two drugs together? Or can we take a lot of these patients that are on ruxolitinib, and getting benefit, and actually ramp in other agents? And I think one of the things I’m paying attention to in these trials is how they’re done, because that has nuts-and-bolts realities for my patients.

Because starting them on two new drugs and giving them two pills and expecting me to deal with the side effects is very different than all right, you started the first drug a year ago. It’s benefiting you, and now we’re going to add the second drug. And so, I think that’s another lesson as we get more experience at these meetings and in these presentations that we’ll learn more.

Dr. Verstovsek:

I think this is very important point, not just for the patients to understand what is going on, but for physicians to learn from their own experience. We have started some of the studies early on with the medications together, not accounting for a possibility of being too optimistic about two medications possibly working together, and then the result was even less than what we would expect.

So now, we have modified our approach, at least at MD Anderson, to be more cautious, understand the combinations better, what would be benefit, what would be combination toxicity, and that we choose and pick the combinations. And the patients are fully informed what is done and why.  

Dr. Levine:

So I think the other challenge we need to figure out is that all my patients ask me whether they have polycythemia vera myelofibrosis leukemia. When are DNA tests going to be useful in my care? When I have a patient with acute leukemia, when I have a patient with myelodysplasia, I already use gene tests to decide who should get aggressive treatment, who should go on a trial, who should actually be watched.

And it’s a bit of a challenge so far because in myeloproliferative neoplasms—myelofibrosis, PV and ET, we haven’t gotten there. And I think one of the questions we have to begin to ask ourselves is, what’s it going to take for mutations to hit prime time in these diseases? The answer is we’re starting to see it.

I think Alessandro Vannucchi has got very exciting data presenting at the meeting and at earlier meetings, suggesting we can be in to develop prognostic schema. I think that’s the first step. We need to make sure these are robust in many different countries and many different cohorts in all of our hands with many different therapies and approaches that does the aggressive or non-aggressive nature of the therapy affect the prognosis?

That’s the first part. The second is we need access to tests. We need to make sure that these tests are available everywhere, not just University of Chicago, or MD Anderson, or Sloan Kettering, but are they available in a doctor’s office in suburban Illinois? Are they available in central New Jersey? Are they available in north Texas before the patient comes to see us?

And I think we’re starting to see some of those exciting results, and I think that that’s a fundamental question that, at least that I’d like to see the field and hopefully contribute to, move forward, and I just don’t think we’re as far along as I would like to see us be.

Dr. Verstovsek:

So to, just to summarize where we are right now, from the 2005, when the discovery of the JAK2 V617F mutation was made, this is the one that we know all about, but now we have so many other mutations that we can potentially test for, some of them are important for disease progression, perhaps.

Some other have, are all that we know understand very well, we can possibly test for them in some settings, but not in everyday setting.

And the combination or just the presence of one or the other may have importance for the overall outcome of the patients, perhaps not in terms of the response to therapy, but for prognostication in terms of the survival and perhaps we’ll change, in the near future, our view on when to, for example, intervene with a bone marrow transplant in the early stage patients, who is doing well but has bad mutations.

And we know, maybe about 25, 50 mutations present, and we can test for them in some setting at this point. I’m not ready for prime time.

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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