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Improving Treatment Strategies for MPNs

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Published on February 10, 2014

Dr. Brady Stein, an MPN expert from Northwestern Memorial Hospital, discusses developing clinical trial research and what it means for patients. Dr. Stein explains the importance of maximizing the potential of existing therapies by continuing to modify the dose to offset side effects and increase positive effects. Dr. Stein further explains how combination therapy clinical trials are the "wave of the future" and why patients should feel hopeful.



Northwestern Medicine


Northwestern Medicine

Transcript | Improving Treatment Strategies for MPNs

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. 

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.  

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.  

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

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.

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