MPN Researcher Shares Optimism for Future Treatment

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Topics include: Emerging Research

Patient Power founder Andrew Schorr is joined by expert Dr. Jason Gotlib, from the Stanford University School of Medicine, to discuss progress being made to improve treatment durability and efficacy for myeloproliferative neoplasm (MPN) patients. Dr. Gotlib gives an overview of the ever-evolving MPN treatment landscape, potential new drug approvals and a hopeful look at combination therapies in clinical trials. Watch now to learn his expert knowledge.

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Transcript

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.

Andrew Schorr:

Dr. Gotlib, how do you feel about progress in MPN generally?  We've had ruxolitinib (Jakafi) for some PV patients and for MF patients. We've had other agents for PV and some uses for ET.  Generally, how do you feel about progress in your field?  

Dr. Gotlib:

Hi Andrew. I'm actually very excited about the progress in our field.  You've highlighted the fact that ruxolitinib, or Jakafi, has been now around for some eight years in myelofibrosis.  It's been around for some five years ago in PV.  We have a potential for two new JAK inhibitors to gain regulatory approval. We will see.  Those drugs are fedratinib (Inrebic) and pacritinib, so I'm hopeful that in the one to two years that may bear fruit.  

I'm also very hopeful that there will be combination therapies that will also show themselves to be effective and well tolerated, so a lot of these combination therapies, ruxolitinib with another drug, with alternative mechanism of action, we're looking at those combinations now in a Phase I and Phase II setting, and we'll see whether they show efficacy and of course whether they're well tolerated. So these are antifibrotics.  These are drugs that try to increase cell death of malignant cells in PV and myelofibrosis, for example.

So we're always looking toward combinations.  We're looking at drugs with new mechanisms of action, and I think that over the next few years we're going to gain a lot of insights from the clinical trials but also going back to the lab to see where we can look at new signaling pathway, new targets, and then bring them to the bedside.   

Andrew Schorr:

So for me and some of my peers who are living with myelofibrosis for a number of years now, but we worry whether the medicine we're taking, like for me, ruxolitinib, that may poop out on us, I guess is one way to say.  Do you feel there's hope that there will be other agents that can help us?  

Dr. Gotlib:

Yeah.  I mean, that's a good point.  Nature has a stunning way to show how cunning it is in terms of showing resistance.  You know, not—there is really no drug that I'm aware of with perhaps rare exceptions that show its benefit for necessarily decades.  So I think that with that in mind we also have a lot of investigators that are trying to come up with ways to improve durability, improve efficacy, try to bypass mechanisms of resistance.   

So, you know, 10 years ago ruxolitinib was not available.  Patients had poor quality of life.  There's some sense that ruxolitinib may also improve survival with five?year follow?up.  We are doing better, but we can do a lot better moving forward.  I'm very hopeful.   

Andrew Schorr:

Okay. Well, if you're hopeful, then, Jason Gotlib, I am hopeful.  Thank you so much for being with us on Patient Power.  

Dr. Gotlib:

You're very welcome.  Thanks for having me.  

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 August 20, 2019