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MPN Experts Discuss Clinical Trials and Transplants

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Published on June 19, 2020

What is a randomized double-blind clinical trial? Why should MPN patients consider a stem cell transplant? Are researchers still "in the game" despite COVID-19?

In this final segment from our recent Answers Now program, host Andrew Schorr gets the answers from MPN experts Dr. Naveen Pemmaraju from the University of Texas MD Anderson Cancer Center and Dr. Jeanne Palmer from the Mayo Clinic Arizona. MPN patient advocate Phil Arner also shares why he chose to undergo a stem cell transplant after participating in a clinical trial. Watch now to learn more!

This is Part 4 of a 4 part series. Watch Part 1 MPN Patient Grateful for Research and Clinical Trials, Part 2 Is MPN Research Still Moving Forward Despite COVID-19?, and Part 3 New Drugs Advance MPN Treatment Options.

If you are a Myelofibrosis patient interested in participating in a clinical trial, use our online tool to start your search. For other MPNs visit the MPN Research Foundation to find open trials.

This program is sponsored by Sierra Oncology. This organization has no editorial control. It is produced by Patient Power in partnership with the MPN Research Foundation. Patient Power is solely responsible for the program content.


Transcript | MPN Experts Discuss Clinical Trials and Transplants

Andrew Schorr:
Greetings from southern California. I'm Andrew Schorr. Welcome to this Answers Now program for those of us living with an MPN, a myeloproliferative neoplasm. And I've been living with myelofibrosis for about nine years, so I'm vitally interested. Joining us we'll have a number of experts. There's one of them that just popped on, Naveen Pemmaraju from MD Anderson Cancer Center in Houston, Texas. Dr. Jeanne Palmer joins us from the Mayo Clinic in Scottsdale, Arizona. And also joining us is, way up from Canada, Ottawa, Canada, Phil Arner, who like me, has been treated for myelofibrosis.

Okay. Our topic and we want to thank the MPN Research Foundation for partnering with us, and Sierra Oncology for being an educational supporter of this program. Our topic is the future of MPN research after COVID-19. But I want to talk about during COVID-19 because we don't have an end date on this right now, and so we're worried. So we're going to start with Phil. But first of all Naveen, thank you for being with us once again.

Dr. Pemmaraju:
Thank you, Andrew and Patient Power, for having me.

Andrew Schorr:
Okay. As a physician-researcher and Jeanne Palmer thank you for being with us too.

Dr. Palmer:
Yes, thank you for having me.

Andrew Schorr:
Dr. Palmer, basic question. People hear this term randomized, double-blind study. Does that mean you're going to get a placebo? You're not going to get the potentially good stuff?

Dr. Palmer:
The answer to that is no. People always say, "Are you going to give me a sugar pill? Am I not going to get the treatment?" They've changed it. Randomized clinical trials, if they are randomized to a placebo, it's not that you will not get treatment, it's just you may not get the drug specifically being studied. And to give a good example of it, let's say you want to find out whether adding something to a JAK inhibitor helps. So what would happen you'd get the JAK inhibitor and then on top of that you'd either the study drug, or you may get a sugar pill that looks like the study drug. And the randomized part is that you don't get to pick which arm, and people just go onto the other, to the other, just back and forth.

The blinded part is that you're given a pill either way. Whether the pill has the drug or whether the pill doesn't is the blinded part. But that being said, you're still getting treated. This person will still get Jakafi. So they will still get some type of treatment. We don't run the risk of randomizing, giving somebody a sugar pill so that they don't get any treatment for their disease. As observation, it's an appropriate alternative.

Andrew Schorr:
Phil, with your success of your transplant, it's a big deal to have a transplant. And one of my friends who hasn't needed a transplant, she sees it as the nuclear option. I don't know if Dr. Palmer being a transplant-er would say that. But you don't go into it lightly. How did you view it, knowing that not everybody comes through it, or comes through it as well as you?

Phil Arner:
Going into it you have to look at the pros and cons. And if I was feeling well, and was not experiencing any difficulty I would probably not do the transplant. But when your back's against the wall, and the numbers are looking bad, it's degrading, it's time to pull the plug. And that's what I had to do. I was in the middle of a clinic, and the doctors were talking transplant, and I wasn't leaning that way, and by the end of the clinic, I made my decision to do it. It's done. And that's what started the process. I was not ready for it until things started to go bad after the momelotinib had stopped working basically.

So it's not a decision that you go into lightly. And it takes a lot of consideration. And everybody is going to be different. So it's not an easy decision.

Andrew Schorr:
Well, we're delighted that you've done so well, and that the trial and those five years gave you good years and then put you in a stronger position to have the transplant. And probably the transplant technology improved. Right, Dr. Palmer? You keep refining transplant technology, so during those five years, you got more refined, while he was doing well on his study drug. Okay.

Well, I want to thank you. I want to get a final comment from our doctors. And Phil, we wish you all the best. Dr. Pemmaraju, first to you. What do you want to say to our MPN audience about us having some confidence that you're going to stay at it? They're going to be challenges, viruses, deflections, demands on resources, but you're still in the game for us.

Dr. Pemmaraju:
Well, that's so important. Yeah, first of all, I agree 100 percent. I think two main themes that I would say for our patients is, the number one area is the digitization of information. Both acquisition and creation of content. Luckily that was already crystallized before this current pandemic. And by that, I mean not only simple Internet and Google, but also all the other things that we're using. This kind of platform, so Zoom, WebEx technology, email, social media, all of these things coming together.

There are many, many stories of investigators and folks that are able to conduct full 80-hour weeks from their house, from their base of operations. So I do want to assure you that whether you're here on campus, or elsewhere, that people are working hard during this pandemic. There's a second aspect to what you bring up, which is innovation. The thing I keep thinking about, Dr. Palmer mentioned that you did too, is sure, at the beginning of any scary time, a hurricane in your area, a pandemic affecting the whole world, war, anything, as human beings we must stop and address those important issues.

Once those issues are started to address, and we put them into our day to day thinking, a lot of innovations are coming because of COVID, around it, and so on, and so forth. Dr. Palmer mentioned one, the telemedicine itself. But I'd mention one or two other ones, which is the ability to connect with researchers all around the world has become paramount now. This EHA going virtual, ASCO did it, AACR did it successfully, Andrew. We saw two to three times more people engage at AACR than before. And so people in remote parts of the world, unable to travel, we're actually getting more scientific engagement than before the pandemic. So think about that.

And then the second aspect is, we're going to rethink from here on out how we can bring in other stakeholders that previously were excluded or felt that they were excluded into research. So put it on Palmer's good list of things that innovation. Necessity is the mother of invention. Back to you, Andrew.

Andrew Schorr:
Okay. Back to Dr. Palmer just to wrap up. Can you give us any more on your good list so we can go out feeling a little better in a tough time?

Dr. Palmer:
Well, I think a couple of really good things. It's been mentioned multiple times the telemedicine has really changed things. I mean, we look at Arizona, we have a big catchment area where people have to drive across a big, hot, long desert. And so I've been able to do video consults. Another good thing about that, sometimes people come for a clinical trial, and they're not ready. They need to be on this drug for a certain amount of time. They need to have this many phlebotomies, whatever it may be. By doing that virtual consult, you're really able to set the stage so when the person comes, they can hit the ground running. So I think the virtual aspect of this.

And then I think the companies understanding that people are scared to travel and scared to go places are really going to I think work better with Lab Corp, and Sonora Quest. Well, we have Sonora Quest, I think it's Quest Lab, whatever that is. They're going to work more with these so that we have opportunities to take care of patients remotely so that they don't have to be under such financial duress to be coming to places for clinical trials. So I'm really hoping that that's one of the very positive things that come out of this.

Andrew Schorr:
Okay. So Jeanne Palmer, as a researcher you're still right in the game.

Dr. Palmer:
Right in the game, and looking forward to keeping my list going. Being ready to move forward. Absolutely.

Andrew Schorr:
Well as I like to say to people like you, you are our angels, the clinicians. And Phil you too. There's data about momelotinib as a potential new option for us that we wouldn't have unless you'd been in the trial. So thank you and that you benefited so much. So I want to thank our experts for joining us, and an expert patient included. Naveen Pemmaraju from MD Anderson in Houston, all the best to you, and keep at it. Dr. Jeanne Palmer joining us, thank you so much from the Mayo Clinic in Scottsdale Arizona. Phil Arner in Ottawa, Canada. Thank you Phil. Long life please. Enjoy those children and grandchildren. I want to thank the MPN Research Foundation for being our partner in this series, and Sierra Oncology for being a supporter of today's program.

I'll let you all go as I just wrap up. If you all have questions, keep sending them into and we have this ongoing series of Answers Now programs specifically for people with all types of MPNs.

What do you want to know? We'll get the experts, and we'll bring them to you. If you think there's somebody you know should be featured on a program, please suggest that, okay? I want to thank all of you for joining us today. And as I like to say as I wrap up our program, 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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