Published on July 29, 2020
When Should MPN Patients Join a Clinical Trial?
Why should MPN patients consider a clinical trial? How are clinical trials involving JAK inhibitors improving MPN care?
In this segment from a recent MPN Answers Now program, host Andrew Schorr talks to MPN Patient Marilyn Lidor about her clinical trial experience and how her husband Dov supported her through it. They are joined by MPN expert Dr. Christopher Hillis from Juravinski Cancer Centre in Ontario, Canada who shares why trials are so important.
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 and BagIt Cancer. Patient Power is solely responsible for the program content.
Transcript | Would I Be A Good Candidate For An MPN Clinical Trial?
Greetings and welcome to this MPN Answers Now program. I'm Andrew Schorr. Today, in Northern California, Belmont, which is on the peninsula south of San Francisco, we are going to be joined by some wonderful guests. I want to thank the MPN Research Foundation and Bag It Cancer for working with us on this program and also Sierra Oncology for sponsoring it, although Sierra has no editorial control. We're going to be talking about clinical trials related to MPNs. We've got some great guests. Let me tell you, we're going to go around North America here. Let's go over to Toronto, and you see Marilyn and Dov Lidor. I've had the privilege of meeting them in person when they were on vacation in Miami Beach and I was down there too, but they are in Toronto. Marilyn's a patient and then Dov is a wonderful care partner and very active in advocacy in Canada.
Then let's skip over, also in Ontario, to Hamilton, Ontario, Dr. Chris Hillis is a researcher and clinician in MPNs at McMaster University. And then let's go to Lake Tahoe, California, not that far from where I am in the northern part of California right now, and that's Dr. Barbara Klencke, and she is the medical director for Sierra Oncology.
Let's start with Marilyn and Dov. Marilyn, first of all, it's great to see you and Dov again. ET, essential thrombocythemia, going back to 2006, find out you have myelofibrosis in 2014, and eventually decide, starting nine months ago, to be on a clinical trial, which you're on in Toronto with a combination combining ruxolitinib (Jakafi) with another drug. First of all, how are you doing? How are you doing, Marilyn?
I'm doing great. I'm feeling great, big difference since I'm on the trial.
And the big difference was to try to shrink your spleen and do better with your platelets, right?
Okay. And I should mention that Dov is very involved in advocacy across Canada. You two made the decision together, I imagine, to be in a clinical trial. One does not do that lightly. First of all, Marilyn, why'd you do it? Why'd you decide to be in a trial?
Well, originally, the only thing for me was Jakafi, that was the only thing that was available, and Jakafi really only address the symptoms of the disease. When Dr. Gupta presented this trial, it was a combination of Jakafi and a new drug called CPI-0610, and the CPI drug has the potential to modify the underlying disease. I figured I couldn't go wrong by joining the trial.
See if you could have a one-two punch, and we're going to talk to Dr. Hillis about research in combination therapy and where that's headed. I'm glad you're doing well. Dov, just for you as an advocate and a care partner, often it's a family decision for somebody to be in a clinical trial. You have to sign a lot of papers. Sometimes you've got to be a lawyer to read some of this. They've been trying to make that better, and I've been in two trials myself, and I'm not in an MPN trial now, but I would certainly consider it. What was your thinking about Marilyn being in a trial?
Well, I think as a care partner, I have to be totally supportive of whatever she wants to do, and the second thing is that I feel that I need to know as much as she does about her condition and about her treatment. We're in it together, so there was never a question of what or if, it was just total support and have as much knowledge as she does, so we are both on the same level in dealing with this issue.
Okay. I love you guys. Thank you for doing this together. Okay. One other thing before we move on with our other guests too. We're doing this program during the coronavirus pandemic. Fortunately, for you in Canada, you've had more control of it than we have here in the States, and so there are many patients though worldwide, who say, "I really want to be close to home. I don't want to run over to the clinic. I don't want to have a lot of blood tests. What can I do in other ways?" In this environment, how have you felt about being in a clinical trial? Marilyn, how about you?
Well, I still go down to the hospital every six weeks, but there's hardly anybody there really, so I really have no concerns, and everybody who I do meet is masked and takes a lot of precautions, so I'm really very comfortable. And then, in between the six weeks, I go to a local lab near me which is also…hardly anybody there, so I'm very comfortable.
Okay. Dov, for you, obviously, worry about your wife's safety, but what would you say, let's say, there in Canada, the patients are still living with the cancer, hopefully, the pandemic, we'll put in the rearview mirror at some point, but you'll still have the MPN. What would you say to people, from your point of view, of considering clinical trials so that we can partner with people like Dr. Hillis or Dr. Klencke to have more options?
I think clinical trials may not be for everyone, but the first point, I think, is to trust your doctor. If you don't trust your doctor, then don't even go there, but we do trust Dr. Gupta 100% and we took his advice. We considered it though, carefully, and we know that clinical trials is the only vehicle really to move forward in resolving this issue and today, more than ever, it holds a lot of promise. We really couldn't go wrong with this.
Right, right. Listen, as I said, I'm not on a clinical trial, but I don't know what's next for the medicines I'm taking. I don't know how long they'll last or whether, if something were added, that would give it a bigger bang, so, yeah, I agree with you. First of all, I want to say to both of you, thank you for being a family participating in a clinical trial and, Marilyn, we're all delighted that you're doing better. You're our hero, along with the researchers, so thank you for doing that. Let's go to Dr. Chris Hillis, down the road in Hamilton, right? We're skipping around Canada for a minute. Dr. Hillis, so there used to not be much research in MPNs, but it seems like the pace is quickening. Would you agree with that?
Oh, definitely. I think we've seen a very nice increase in the number of clinical trial options for patients, both for drug treatments, but also for non-drug interventions. Thank you, Marilyn and Dov. I think you just did the best ad for participation in clinical trials I've ever heard of. I may play that in my clinic waiting room. But, certainly, we've had a big leap forward with some of the novel agents as a platform for doing clinical trials, but also for increasing attention on myeloproliferative neoplasms. MPNs are very rare, and so folks who are living with MPNs and were diagnosed 20 or 30 years ago really didn't have a lot of options for treatments beyond aspirin and phlebotomy, and we've doing phlebotomy in medicine for hundreds of years, and so certainly, I think, after a few hundred years, it's time that we started testing some new therapies, and we really are at an exciting time.
Okay. Let's talk about where we are, and we're going to learn more about additional JAK inhibitors. She's been on one. I was on that one earlier, ruxolitinib, Jakafi or Jakavi, it's called in some countries. I'm on a different one now, fedratinib (Inrebic), which been on hold and then it came off and it got approved, trade name Inrebic. And then there are others like momelotinib and also pacritinib and maybe some others for all I know. That's one approach, and these are not necessarily alike. They have differences, right? JAK inhibitors have different ... it's not the same thing, right? They can be different, correct?
Yeah. Whether you want to call them and brothers and sisters or cousins, I think we could all agree, that you can be from the same family, but all be quite different. Certainly, if you met my family, you would agree that we can all be very different but be from the same place. And so JAK inhibitors have changed the treatment of MPNs for the better, there's no doubt about that. I think Marilyn put it really well about what our first and foremost goal of treatment with the JAK inhibitors are, which is to live better, and we would certainly hope you'd not only live better, but you would live longer. And for those of you who followed the clinical development story, there's some controversy around that as well with some other aspects of MPN care.
But we know they are great drugs, but not sufficient, and so I think combination therapies like what Marilyn is on might be the way of the future, but we don't know what the right combination is, and so entering a trial, either after ruxolitinib to say, "What's the next best step for me?", or if ruxolitinib is not working for you is one type of trial or if you're at the beginning of your MPN journey, thinking about a combination trial just so that we can learn more about what's next or what can make the JAK inhibitors better.
Okay. And just about JAK inhibitors, so there are ones in trials, so in other words, it may be that, let's say, if a JAK inhibitor ends up being the foundation of MPN care, you will learn the nuances of different ones, both through trials or, as they're approved for greater use, for what's right for what patient, right? Which brother or sister is your best match?
Exactly. And MPNs are very complex diseases, and we talk about having the JAK mutation or the calreticulin (CALR) mutation or MPL or one of the driver mutations or perhaps other mutations, and every MPN patient is very unique, so to think there's a one-size-fits-all strategy is not correct. Even if you have two myelofibrosis patients, both with the JAK mutation, for instance, one could have a great response to this ruxolitinib and the other one could have no response. And we, through clinical trials, need to learn who's best with what treatment and very kind people, like Marilyn and Dov, participating in clinical trials are how we get these answers.