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Expert Perspective: Advancing MPN Treatment Through Education and Clinical Trials

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Published on June 13, 2016

What is the latest research in myeloproliferative neoplasms (MPNs)? Patient Power founder and host, Andrew Schorr, sits down with clinical researcher and medical oncologist, MPN expert Dr. John Mascarenhas of Mount Sinai School of Medicine. Dr. Mascarenhas shares his expert—and hopeful—opinion on the latest novel therapies and how he expects them to change the course of MPNs, including remission and cure.

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Transcript | Expert Perspective: Advancing MPN Treatment Through Education and Clinical Trials

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:

Hello and welcome to Patient Power. I'm Andrew Schorr. So it is a great delight for me to meet leading researchers in the field of the MPNs for all of us in the community. We want to know that science is moving forward. Someone in that category is joining us now, Dr. John Mascarenhas from Mount Sinai in New York. Thank you for being with us.

Dr. Mascarenhas:               

Pleasure to be here.

Andrew Schorr:                  

Okay, so tell us—give us a window into what you're researching now and what benefits it could have for us patients with MPNs? 

 

Dr. Mascarenhas:               

So I'm primarily a clinical investigator with myeloid malignancies with a real focus in myeloproliferative neoplasms. So my interest really is in trying to bridge the translation from the laboratory to the clinic to help patients with novel therapies that would hopefully improve multiple aspects of their life, potentially change the course of the disease, even things like remission and cure that we like to talk about.

So things that I'm interested in or I think have prospect are drugs that range from interferon for the treatment of essential thrombocythemia and polycythemia vera to newer tyrosine-kinase inhibitors. There are a number of drugs that Dr. Heaney has talked about like telomerase inhibitors, anti-fibrotic drugs, histone deacetylase inhibitors, and then recently or increasingly the combinations of therapies, putting different drugs with different mechanisms of action together to try to improve upon the effect of each single drug. Hopefully, without overlapping toxicities to get synergistic effect. Of course, all of this is what we're doing right now. I think a lot of the hope is that the research that's coming out of the laboratories today will fuel newer approaches tomorrow that we may not even appreciate at this moment that will be even better than what we're doing today. 

I think that's what really drives myself and I think Dr. Heaney to keep coming in and doing this every day. I mean, it's an exciting period with a lot of insight from the laboratory that may not have existed to the same degree a decade ago. Even within the last decade of doing this, there has been tremendous growth and it's really paid off. I think it will pay off more—we will learn more that will ultimately funnel down to helping the patient as we understand the disease mechanisms better. 

Andrew Schorr:                  

Giving that you're doing all this work, is the strategy for today's patient may be getting currently approved therapies or maybe being an existing clinical trials, because you're working to get even further ahead. Should the strategy be help me do as well as I can as a bridge to what's next? So the therapies I may have made peter out, may have certain toxicities, but it needs to get to when what comes out of your lab will be available to me.

Dr. Mascarenhas:               

Yes, I think so. I think right now the only, for example, in myelofibrosis the only FDA-approved therapy is ruxolitinib (Jakafi), which is a fantastic drug and has clearly changed the treatment paradigm from myelofibrosis. It's an excellent drug from proving certain aspects of the disease, mainly splenomegaly or symptom burden. But there is still a need, an unmet need to try to change the disease course or affect the disease at its core in order to modify the natural history of the disease. So in some sense, ruxolitinib is a great drug for many patients as their first therapy. 

But we're still trying to figure out what second-line therapies or other therapies can be added to improve upon that response. So one could look at ruxolitinib as a bridge, but for some patients ruxolitinib has been and maybe the definitive therapy for that given individual. So it's not always a bridge. It can be a bridge to bone marrow transplant. It could be a bridge to another clinical trial, so it could be a bridge in some senses.

Andrew Schorr:                  

But you're delighted that you have for some patients now something that's working.

Dr. Mascarenhas:               

Absolutely. I mean, ruxolitinib is a drug that has without doubt changed the lives of many patients, but it's also set the tone or the bar maybe for where we need to go and what we need to think about as we develop newer therapies that can add upon that. Perhaps in combination or in the second-line setting that would perhaps—I am careful to say, but perhaps put patients into remission, stop the disease, progression, things that remain a worry to patients and caregivers.

Andrew Schorr:                  

Okay. Now, science sometimes is two steps forward, one step back.

Dr. Mascarenhas:               

Often.

Andrew Schorr:                  

So we had a drug that was very promising that at least right now as you record this, pacritinib, the trials were stopped, okay, for concerns, safety concerns. We'll see where that goesbut yet with that drug and other drugs that you've been working on to try to advance care. So tell us about that process. You're trying to answer scientific questions, and it's not always a straight line.

Dr. Mascarenhas:               

It's not. I mean, unfortunately, there have been a number of drugs I can think of over the last five years that have looked very promising early on that sometimes at the very last or later stages… 

Andrew Schorr:                  

Fedratinib was one.

Dr. Mascarenhas:               

Fedratinib—that was very disappointing. I don't look at it as a total loss, because we still learned a lot. We learn a lot about the mechanisms of these drugs, how they interface in the patient and how we can improve upon them. So there's still a benefit from these drugs that go forward like this, even if they don't go the extra mile and become FDA approved. Pacritinib, I think, is still an interesting drug. I'm perhaps one of the few people that do not think the story is over for that drug. I still think it has a niche. There is a subpopulation of patients with myelofibrosis that is clearly an unmet need of patients with very advanced disease, typically with a very low platelet count that may not be eligible for ruxolitinib, and that's really an urgent unmet need. These patients are suffering. They need therapy, and pacritinib fills that spot. It has to be balanced with the potential for toxicity that needs to be better elucidated and understood. But I still think that there is room to bring that drug back into development and hopefully improve upon its toxicity profile or optimize the toxicity profile; therefore, allowing this subset of patients some benefit that they've been excluded from to date.

Andrew Schorr:                  

And that's what we've seen in other areas of oncology, for instance, in CML. We saw a drug where there were concerns about toxicity but figuring out who it's right for and then the risk benefit ratio, and that may be the same in MPNs.

Dr. Mascarenhas:               

Yeah.

Andrew Schorr:                  

So what you understand of where the research is going and what you're doing to help patients, are you hopeful for those of us living with MPNs? 

Dr. Mascarenhas:               

Very hopeful. Very hopeful because I have to be. It's my personality. It's what drives me to work every day, even though I walk. And I'm very hopeful, because I really do believe in what we do. I believe in the dedication and the interest of the multitude of people in the laboratory that have an interest in developing an understanding of the scientific aspect of the diseases that will ultimately translate to better therapies. I believe that pharmaceutical companies are also interested in the same goal.

Their motivations may be sometimes different, but at the end of the day I think that there's a concerted effort by multiple different types of people from different backgrounds to really bring better therapies that will really help patients, and I think that's exciting. I'm fortunate enough to be doing this in a time where the science has matured to a point where these things are becoming a reality, and we're testing them in the clinic, and it's really gratifying. It's very exciting. I wouldn’t want to do anything differently.

Andrew Schorr:                  

Well, I'm delighted to hear that. One last thing is what can we in the community and as individual patients do to help you?

Dr. Mascarenhas:               

That's a great question. So this is a question that I often—sometimes patients ask me, and I often wonder too. I think the real answer is participate in clinical trials. That's not always an attractive answer. Some patients have I think very valid concerns about trials. I think some patients don’t really understand what trials are. I think that one obstacle to success sometimes is the mystique or the misunderstanding maybe of what clinical trial participation is. There is often this attitude of yes, we need clinical trials, but it's for someone else.

I think that's sometimes unfortunate. I think just engaging the patient community so that there can be an understanding on both sides—the physician, the pharmaceutical company and the patient community about how to make this process more attractive for everyone involved, how to optimize it, because we can take all of these findings in the lab, translate them into oral and intravenous therapies. But if the patients are not onboard and don't trust the system or don't know about it, then it's really not going to help. So I think it's really a joint effort between all parties involved.

Andrew Schorr:                  

Okay, well we're partnering with you and other researchers and clinicians to do this, and we want to thank you for all you do.

Dr. Mascarenhas:               

My pleasure.

Andrew Schorr:                  

Thanks and let's keep getting those answers for better therapies for all of us. Andrew Schorr with Dr. John Mascarenhas from Mount Sinai, as we push forward in MPN to do better for all of us. Remember knowledge can be the best medicine of all.

Okay, Dr. John Mascarenhas, thank you so much for being with us and for your perspective from Mount Sinai. Pushing ahead in MPNs to do better for all of us. 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 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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