What is the Next Era of Treatment for MPNs?

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Topics include: Treatments and Understanding

MPN expert Dr. Brady Stein explains recent research that highlights the positive impact of JAK inhibitors on spleen size, symptoms and survival and the potential for deeper effects, including evidence of bone marrow stability and improvement over time. Dr. Stein explores the next wave of promising JAK inhibitors and how logical and novel combination treatments could lead to an improved response in patients. He feels positive about progress made in a relatively short period of time and believes the most important goal is to dramatically alter the natural history of the disease to see a complete remission in people living with MPNs.

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So we're going to learn also presented this evening that perhaps some patients who can tolerate this drug for long periods of time we may see even deeper effects, and that is either stability or some improvement in their degree of bone marrow scarring.  So this is another important finding that we presented at this meeting. 

We're also going to see updates on the next wave of JAK inhibitor therapies.  So one and the most mature of the next wave, fedratinib, we'll hear about the results from the mature clinical trial in which patients received fedratinib or a placebo therapy.  Now, there will be important results presented, but this drug has been withdrawn due to an adverse effect, so we won't see the development of this agent. But we have to be positive because there are many other JAK inhibitors on the way, novel agents that we're going to learn about at this meeting.  And one might improve the hemoglobin, might help patients liberate from their dependence upon transfusions.  We heard that presentation last night.  This is very exciting. 

We're going to hear about another JAK inhibitor, which may not have so many side effects in the way of declines in blood counts.  We're going to learn about even more novel JAK inhibitors that appear to specifically target the JAK2 V617F mutation, which is important because many of these agents target both the normal JAK2 and the abnormal, mutated JAK2.  So we'll hear an updated presentation on that agent. 

Now, we're looking for unique effects, deeper effects. And with each agent, there are some of these unique effects.  And so as I mentioned perhaps a suggestion that ruxolitinib does more than address symptoms and the spleen is that, as I stated earlier, the bone marrow scarring may stabilize or improve over time.  This is a really important finding both for physicians as we communicate expectations and observe the disease and certainly for patients.  Some of the other drugs again may improve the blood counts, or we may see less of a decline.  

So there's certainly more that these drugs can do, but the most important goal is to re-create the experience that we've seen with imatinib (Gleevec) and the other tyrosine kinase inhibitors in CML, that is, dramatically alter the natural history of the disease, see what we call a complete remission, which means very little to no evidence of the disease when looking comprehensively.  So this is the next step. 

So we believe that the JAK inhibitor is the foundation, the foundation to improve symptoms and splenomegaly and perhaps some of the unique effects, but we need to look to other drugs to add to it. And we need to look for logical and novel partners that would work together nicely with the JAK inhibitor to get a deeper response, and there are so many other aspects of the disease, there are so many other contributors that can be targeted or addressed with novel strategies. So this is the next era for the treatment of myelofibrosis. 

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

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Page last updated on September 4, 2014