Expert Perspective: An Optimistic Outlook on the Future of MPN Therapy

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Topics include: Treatments

What new therapies are in development for MPNs? What does the future hold? MPN specialist Dr. Stephen Oh from the Washington University School of Medicine shares his perspective on the latest advances in MPN treatment. He comments on older therapies like Hydrea and new agents in development such as telomerase inhibitors; and he discusses his optimism for the future of MPN therapy.

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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. 

Susan Leclair:

Welcome to the MPN meeting in Chicago, Illinois.  We have with us today Dr. Stephen Oh.  He's going to speak to us a little bit about the treatment aspects and research that's involved with dealing with myeloproliferative neoplasms.  Dr. Oh. 

Dr. Oh:

Thank you.  Thanks for having me here today.  So just so introduce myself, my name is Stephen Oh.  I'm assistant professor at Washington University in St. Louis, and my clinical practice and research focus is on myeloproliferative neoplasms.

Susan Leclair:

Dr. Oh, I'm curious.  There have been so many changes in medicine in the last, say, five to 10 years.  Why do we always fall back on hydroxyurea (Hydrea®), which has been around for a very long time?  Isn't there something better, nicer, faster, easier? 

Dr. Oh:

Right.  So that's an excellent question.  In fact, I was in clinic yesterday and a patient asked me, you know, with hydroxyurea, isn't this a really old medication? And, as you said, shouldn't there be something newer and better?  And I think that is actually a complicated question. 

An old medication could perhaps be outdated, but actually in many cases it could still be quite effective. And for many patients with different myeloproliferative diseases, hydroxyurea is quite effective and well tolerated.  With that said, there are newer medications available. 

Interferon, which is not necessarily a newer medication, but pegylated forms of that, that's seen a bit of a resurgence in the past several years, and can be effective for many patients.  And more recently, as many people know, inhibit??targeted inhibitors or JAK2 are now available, both FDA approved in terms of ruxolitinib (Jakafi®) and several that are in clinical trials. 

So it's in some ways a very exciting time right now because there are newer drugs available, again, some that are approved and some that are in clinical trials, but also a challenging time in that we have now several different choices, and the question is which to use and when.  

Susan Leclair:

You're involved in this research.  What are you working on now, if it's not secret, and—and what excites you the most about this? 

Dr. Oh:

Yeah.  I think it's, you know, it's really—a really very exciting time for us in terms of research and for the patients I think.  If you go back, you know, it's basically been about 10 years since the discovery of the JAK2 mutation in the MPNs, and at the time really we had very few treatment options available. 

And now so many more, again some approved and some in clinical trials, and so I think it's very exciting in terms of still looking at the different JAK inhibitors. 

We're still trying to figure out which perhaps would be the best agents and in what setting for the different MPNs, and a number of different combination therapies are being examined in clinical trials, so ruxolitinib or other JAK inhibitors with other agents and agents that have completely different other mechanisms of action. 

So, one example, imetelstat, which is a telomerase inhibitor, has received a fair amount of attention recently because with small numbers of patient, some patients with myelofibrosis have actually experienced complete remissions of their disease. 

Susan Leclair:

I know I think a lot of people think, well, gee, it was in 1999, I think, that imatinib mesylate changed the world, for one of the myeloproliferatives in a sense, chronic myelogenous leukemia.  Do you see one of those coming any time soon, within 10 years, sometime?  

Dr. Oh:

Yeah, it's an interesting question because I think based on the success of imatinib (Gleevec®) in CML, when the JAK2 mutation was discovered in 2005 I think many people at least hoped that we would have a similar story in the JAK2-associated MPNs, and that has clearly not been the case despite, you know, these drugs do provide significant benefit particularly in terms of symptom benefit. 

But I think it really underscores that these diseases are very complex, and having the kind of success that has been seen with imatinib is certainly not here now.  Whether we will see that in the next, say, five to 10 years I think is very difficult to say, but I'm very optimistic that we're going to get closer to that with the therapies that are being developed. 

Susan Leclair:

Thank you, Dr. Oh.  That was very informative, and I think it's an exciting time in the development of medications and treatments for these diseases.  Thank you all for participating in this presentation, and we hope to see you again on patientpower.info. 

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 July 14, 2015