Improving Treatment Strategies for MPNs

Please remember the opinions expressed on Patient Power are not necessarily the views of Northwestern Memorial Hospital, its medical staff 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:

Hello and welcome to Patient Power sponsored by Northwestern Memorial Hospital.  I'm Andrew Schorr on location with Dr. Brady Stein from Northwestern.  We're in New Orleans, Louisiana.  How come?  Because we're at the American Society of Hematology meeting, and among the important conditions they speak about here are the MPNs, myeloproliferative neoplasms.  Anybody who has one has heard that acronym.  So, Dr. Stein, for your patients, what do you want them to know about news here for MPNs, hopefully some upbeat news? 

Dr. Stein:

Okay.  So there's absolutely a lot of upbeat news.  There is a lot of data that will be presented about existing therapies, maximizing the potential of our existing therapy, a JAK inhibitor, ruxolitinib (Jakafi), and so this meeting will be presenting the three?year follow?up from the COMFORT?I study, the original study that led to its approval.  So that will be very exciting.

There are other strategies being presented here, either in poster form and then sometimes in abstract form, oral abstract form, about how we maximize the potential, modifying the dose to offset some of the side effects and maximize some of the positive effects.  

There are many updates on the JAK inhibitors that will follow, so this will be very important to patients as we see the development of newer agents that hopefully can build upon the foundation of the existing standard. 

So one of the newest agents, unfortunately, has been discontinued from clinical trials.  So this is a drug that was showing a lot of benefit for patients in clinical studies, but this has been discontinued. So it's important to know that there are others that will follow and that are being presented at this meeting.  And some of the JAK inhibitors, one might improve the hemoglobin, might help liberate patients from requiring transfusions.  That's very, very important. 

There's another novel JAK inhibitor that might have less of a suppressive effect on the bone marrow so less declines in blood counts while still preserving some of the important clinical effects.  That's very, very important news for patients.  And there are even more novel compounds that are going to be presented at this meeting in an update. 

There are other strategies that are going to be presented as well.  The JAK inhibitor we know the most about, ruxolitinib, targets JAK1 and JAK2, and we know there are many clinically important effects.  We're going to learn this weekend about an inhibitor that targets JAK1 hoping to really relieve symptoms without causing much of a decline in blood counts. 

Other strategies, perhaps one of the more exciting strategies that many are anxious to hear about is a drug called imetelstat.  That will be presented by the Mayo Clinic, and this presentation is this evening, a novel agent with very potentially exciting findings, and we'll see more about that this evening.  So, many, many new novel agents maximizing what we know now or developing new agents.  This is very, very important news for patients.  

Andrew Schorr:

Dr. Stein, the sort of more aggressive end of the MPN spectrum is myelofibrosis.  People have said can the new approved drug, maybe others coming, can they help me live longer?  Do we know about that from the data coming out? 

Dr. Stein:

So we do know.  So there is survival data.  There are data that tell us that patients are living longer on this drug.  We know this is true when you compare this drug to a placebo pill.  We know this is true when you compare this drug to what we call best available therapy, which in a clinical trial means the investigators' decision about what the right drug for that patient might be.  We know this is true when you compare historical controls.  Patients who are of a similar risk with a similar disease have been treated differently, we know that one of our JAK inhibitors has been shown to help patients live longer.  So this is important data for patients for certain. 

Andrew Schorr:

Okay.  Now, people understand that with the JAK inhibitors so far there's better control of symptoms, incredibly better control for many patients.  They say is it dealing with the underlying disease?  That's the hope.  Where do you think we are with that or in combination with others so we can accomplish that? 

Dr. Stein:

So at this point in time, it is helping patients live longer, and I think many believe that it's through some of the indirect benefits.  Patients feel better on the drug. They're becoming more active, they're gaining weight, and they're developing more reserve to fight out somewhat common illnesses that before their myelofibrosis they would have less difficulties in facing.  So the life expectancy, the important benefits are likely indirect.  Patients are feeling better, some of the symptoms I described. 

The drug may not be having what we call a direct effect on the disease per se, so there are at this meeting there will be an update on what happens in bone marrow scarring with long?term exposure to this drug. So even though at present we view this drug as helping with symptoms, we know it absolutely helps with the spleen. We know that patients are living longer, we've all been looking for deeper effects. 

This is perhaps one example because bone marrow scarring over time appears to be stable and in some patients improves.  So perhaps there's some evidence that if you take the drug for a longer period of time, if the patients can tolerate, there are some deeper effects and relief from symptoms and splenomegaly.  And that's what we're looking for. 

Combinations I think are the next step, and this is where all the clinical trials are moving towards, the JAK inhibitors, the foundation to help with symptoms and splenomegaly, an enlarged spleen, and then adding something, either practical or novel, to address the way the disease in a way that a JAK inhibitor may not be addressing.  So this is absolutely the wave of the future.  

Andrew Schorr:

Okay.  Are you feeling positive for patients? 

Dr. Stein:

Oh, I'm absolutely feeling positive for patients, and that's what we talk about.  It's obviously a huge adjustment to learn about a diagnosis of myelofibrosis and to come to terms with the ramifications of such a diagnosis, but what I talk about are first?off we now have a medication that is very likely to help patients feel better.  We now have a foundation or a platform to build upon, and I show them or discuss all of the scientific activity in the clinical trials that are out there to tell us that in one to two years or three years I think the strategy is going to be entirely different for the better.  So there's a lot of hope for patients, for sure. 

Andrew Schorr:

Okay.  Good news, and, of course, you're at a research center where a lot of this happens.  And, of course, this can also help people with more aggressive polycythemia vera, and research goes on as well with essential thrombocytopenia, so the whole MPN area I believe can be changing.  This man is right in the thick of it, and we'll be hearing more from him, of course, during the year. 

On location in New Orleans at the American Society of Hematology, Dr. Brady Stein. 

Dr. Stein:

Thank you very much.  I appreciate it 

Andrew Schorr:

Thank you for all you do.  I'm Andrew Schorr.  Remember, knowledge can be the best medicine of all.  

Please remember the opinions expressed on Patient Power are not necessarily the views of Northwestern Memorial Hospital, its medical staff 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.