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When to Consider an MPN Clinical Trial: Is It Right for Everyone?

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Published on August 19, 2014

When is it appropriate for an MPN patient to consider a clinical trial? Dr. Brady Stein from Northwestern Medicine discusses how the latest studies of interferon are affecting MPN treatment and describes the pros and cons of participating in a clinical trial.  Dr. Alison Moliterno from Johns Hopkins Medicine then weighs in on patients that may be candidates for a trial.

Sponsored by Incyte Corporation. Produced in association with the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.


Transcript | When to Consider an MPN Clinical Trial: Is It Right for Everyone?

Please remember the opinions expressed on Patient Power are not necessarily the views of Northwestern Medicine, its medical staff 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.

Jeff Folloder:

Let’s talk a little bit about that research, that learning; clinical trials. Clinical trials have kind of a warm spot in my heart right now, because I participated in a clinical trial with MD Anderson a couple of years ago. And the clinical trial not only got me to complete remission, it got me to this fancy term called “minimal residual disease” status, which evidently, is something of a big deal.

If you took my blood right now, ran a few tests on it, my cancer’s not there. Now my cancer doctors know it’s going to come back, but I participated in a clinical trial because my doctor told me that in his opinion the clinical trial offered me the best chance at complete relief with the minimum amount of side effects.

Now that doesn’t happen for everyone, but let’s talk about when it’s appropriate for an MPN patient to consider a clinical trial.  

Dr. Stein:

Remind everyone because I think it’s a very, if I might also talk a little bit about the experience with Interferon, and I think it brings up the importance of clinical trials as well. So maybe it’s a natural segue, hopefully it’s a natural segue. The reports are definitely out there. There are, this is what intrigues us about Interferon, there are reports of making the blood look like quite normal, there are reports, as Alison had stated about decreasing the molecular burden and, in some cases, JAK2 is gone. The really interesting part as well is that there are reports out there of patients who have discontinued therapy and for years on end, like yourself, had normal blood counts.

There are reports from France, from the MD Anderson in Houston; there are reports of similar patients in Denmark. And that’s very much intriguing in being off of a therapy, and the blood counts staying normal and in some cases the JAK2 mutations staying away. What we’d really like to learn is what is unique about a patient like that, who’s had such a remarkable response to Interferon? That is the intrigue with Interferon.

Interferon is not really a new idea, it’s been in use for polycythemia vera for 25 years, but there’s a renewed use, a renewed interest based on experiences like this one. And what we need to see next is how realistic is this when we study unique patience all over the world in large clinical trials. So what we’re really excited about with interferon is the, these studies are now underway.

They are being down all over the world, and we were hoping to, to participate as a site, but we were not able for logistical issues. But these are clinical trials that might define the role of interferon, a Phase III clinical trial, hopefully, minimizing the bias that can come from earlier clinical trials that are not randomized with long-term follow-up and very careful assessments of efficacy, safety and unique benefits. So we hope to learn that there are more patients like you taking interferon.

So we’re going to see that a clinical trial can perhaps define its role for patients with ET, polycythemia vera, even patients with myelofibrosis. Those clinical trials are opening as well. So a clinical trial is, is a controlled setting that helps us learn about the impact of perhaps an old therapy being used in a new way, or a brand-new therapy.

So, and I think we, we can talk about the pros and cons of clinical trials some, if we, if we might.

Jeff Folloder:

I think it’s important.

Dr. Stein:

So what are the, the pros I think are, are relatively clear. You might get early access to something new and unique that could really help you, okay? You might be in a situation where you’ve tried other medications that are available, and these medications have failed you and not helped you as much as we hoped. So it’s about access to something new, novel, and hopefully, exciting. Cost?

Most of the clinical trial drugs can be very, very expensive, and they’re covered. Sometimes there are other costs that you have to incur, for us, parking. And I think we learned that it’s even worse in New York City. But for here, if you have to come frequently for a clinical trial, I think that’s, it’s an issue.

Jeff Folloder:

And do you validate it?

Dr. Stein:

We do validate it, in fact, yeah. I can’t personally validate it. Some patients have asked me. No, we actually had someone who was applying for a job, who asked me to validate the parking, and I said, you’re probably asking the wrong person, and the person didn’t get the job. So the pros are access to something new, unique and novel. And sometimes, you can have many of the financial burdens relieved.

What are the cons? And I think Amy, as a former research nurse, can also talk about this. Inconvenience. You’re, a lot more is asked of you in a clinical trial, and more frequent visits than you would expect if you were getting a medication prescribed. There are more frequent tests expected of you, whether that could be blood draws, MRIs of the spleen, bone marrow biopsies. So it’s a balance.

So really, you know, this is the discussion that, it probably should be multiple discussions with your doctor and with a doctor who might be heading that clinical trial. This is not something that is a short visit. These are long visits, and sometimes many visits to go over the pros and cons to see if a clinical trial’s right for you.

Jeff Folloder:

Dr. Moliterno, is every patient a candidate for a clinical trial?

Dr. Moliterno:


Jeff Folloder:


Dr. Moliterno:

Well and I, I think Brady said it really well. And I had an experience as a principal investigator for a clinical trial that tested a tyrosine kinase inhibitor, in the treatment of PV and ET patients. And so we enrolled 36 patients over three sites, and these patients were similar to the group here. All very healthy, and in the middle of working, raising families, having children, caring for parents, caring for their grandchildren, and working a couple jobs at the same time. So asking patients to come in, this kind of a patient, for a clinical trial, it’s a big commitment on the patient’s part, right? And this trial was looking at a new tyrosine kinase, not ruxolitinib (Jakafi), but a related compound.

And so we had to do pharmacokinetics, which means several times during the two-year trial observation, they had to come in for a day-long visit, where we drew blood every two hours, to test drug levels after administration. So this is a, a big commitment for everyone and, for the doctors, right, to, to organize this, and for the patients to come and take so much time from their lives to do this.

Most of the patients when they came in, in the clinical trial had the disease 10 years already, right? Because this is chronic myeloproliferative disease. So they’ve been living with this a long time, and, you know, over the two-year trial. So we all got to know each other really well. So I was glad to do it, but I could definitely, there was a lot of dropout in the trial because of toxicity of the drug.

It wasn’t the most easy drug to manage. There was a lot of diarrhea. And while that’s not a terrible side effect. If it’s bothering you every day, and you’re trying to raise your children and work and care for your parents, you’re not going to tolerate really much in the way of side effects. So we learned a lot from, from doing that. And I think this is a challenge for MPN patients and families.

You have your disease a long time. That’s a good thing, right? Meaning that, right, this is a disease where you have a natural history of decades. But it does make clinical trials challenging. You’ve had the disease a long time, so maybe that agent only works maybe early in the disease. And are we looking for a cure? Which we’d have to power the trial several years on the therapy, before seeing a cure.

Are we just addressing symptom burden, right? So I think these are all important factors as we consider, do I want to participate in a clinical trial? The other issue with clinical trials is that the trial is asking a question, right? The question may be, does this agent work to relieve symptoms? Sometimes the physicians participating in the trial, we really wish, wish there could be a different question. How about cure?

Or how about, yeah, a, a, a different question? But you have to comply, you know, these have to be multicenter trials to enroll patients. So sometimes the question may be not quite what you wanted or what your patients want. But we still have to do the trial to get information, and, and that’s often a, a real bone of contention for everyone.

And, and so when we say, well, we’re not going to offer this trial, and we can’t participate either because the question isn’t appealing to us, or the organization, the clinic that we work in, the university, the cancer center can’t afford executing that trial. And those are some of the real factors related to participation in clinical trials.

Please remember the opinions expressed on Patient Power are not necessarily the views of Northwestern Medicine, its medical staff 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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