MPN Treatments and Clinical Trials Archives

When it comes to treatment, MPN patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

More resources for Myeloproliferative Neoplasms (MPN) Treatments and Clinical Trials from Patient Empowerment Network

Living Well with MPNs – Are There New or Emerging Treatments That Could Be Right For Me?

Understanding Treatments for MPNs: Are There New or Emerging Treatments That Could Be Right For Me?

Understanding Treatments for MPNs: Are There New or Emerging Treatments That Could Be Right For Me? from Patient Empowerment Network on Vimeo.

What new treatments are in development for myeloproliferative neoplasms (MPNs)? What are the considerations when choosing a treatment plan? In this LIVE webinar, Dr. Bart Scott from Seattle Cancer Care Alliance and Dr. David Snyder from City of Hope will help viewers to understand the various treatment options for those living with polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF).


Beth Probert:

Hello and welcome to our Patient Power webinar today. Our topic is “Understanding Treatments for MPNs; are there new or emerging treatments that could be right for me?”

I’m Beth Probert, and I’m coming to you from Oxnard, California which is just northwest of Los Angeles. I am a polycythemia vera patient and patient advocate. I was diagnosed in April, 2016 by my specialist at the University of Southern California Norris Cancer Center. I was treated with Pegasys, which is pegylated interferon and a few phlebotomies for about 12 months. And I am happy to say that I am in remission.

But like many of our patients and caregivers in the Patient Power community, I am very concerned about the future of my condition and very interested to hear today about some of these new and emerging treatments and clinical trials that will give us hope for our future and for all of is in the MPN community.

Before I introduce our panel today, I do want to remind everyone that you can submit your questions and we will try to address every question in today’s show. If we don’t get to it, we certainly will address those questions in future webinars. You can submit those questions to

All right, I’d like to introduce our panel today. Joining us today from Seattle we have Dr. Bart Scott. Dr. Scott is a medical oncologist at the Seattle Cancer Care Alliance and a research associate in the Clinical Research Division at Fred Hutchinson Cancer Research Center. Thank you so much for joining us today, Dr. Scott.

Dr. Scott:

Thank you for having me. Hello, everyone.

Beth Probert:

Great. And I’d like to introduce our other doctor on the panel today, Dr. David Snyder. He is joining us from Duarte, California which is in Los Angeles County. Dr. Snyder is an Associate Chair in the Department of Hematology and Hematopoietic Cell Transplantation at City of Hope. Dr. Snyder, I hope I said that correctly. Welcome.

Dr. Snyder:

Thank you, I’m happy to be here.

Beth Probert:

Thank you. Now I’d like to introduce you to our patient panelist. Today we have James from Lubbock, Texas and James was diagnosed quite a few nears ago with the central thrombocythemia. James, welcome to our show today.


Hello and thank you.

Beth Probert:

James, I’d like to start with you. How long ago were you diagnosed with ET?


In October of 1994, I was 47 years old. I had a heart attack. If it had not been Monday morning and I was in the hospital visiting my mother, I would be dead. I had a blockage right at the aorta. But they pulled me out of it.

I’ve been active all my life. Even back then, I was running more than I do now. It just happened to me, and so eventually we got around to essential thrombocythemia, whatever that meant.

Beth Probert:

Well James, that’s such an interesting story because you’ve been dealing with this for several years. And in 1994, if I’m correct, we didn’t really have too much internet access. How did you and your family deal with this? How did you get information and educate yourself?


Well, just so you know, I’m 545 miles from Houston, 325 miles from Dallas, in a town of 250,000. But luckily, we have a medical school. And so I went to the library at the medical school and I befriended one of the librarians whose husband was an oncologist, or is, and she was very helpful in trying to guide me. But back then, there really was very little information.

It wasn’t current. It was from the polycythemia vera study group, and that’s real old data and things like that. But when the internet came on, I started playing around on Yahoo, came across MPN – or back then it was MPD – Digest, and read that franticly every night.

Beth Probert:

I can certainly imagine what a revelation that was to all of a sudden have information at your fingertips. If I’m not mistaken, when we were speaking earlier you mentioned that you also through the years have attended conferences. Can you speak a little bit about that?


Yes, it’s literally empowering to go – and my first one was in 1999, but to go into a room and find other people who are dealing with some kind of MPN. I was like wow; I really am not quite so alone. And over the years the quality of the information has increased tremendously, as it has in the whole field.

It’s still empowering. I have friends I’ve made by going to the Scottsdale conferences; you just learn a lot. It’s just good.

Beth Probert:

That’s very inspirational. I kind of want to shift gears a little bit here and ask you a couple questions. Do you see a specialist?


I see a hematologist/oncologist in Lubbock and I have right from the start. He started me on hydrea and soon to be 23 years, I’m still on it. I’ve varied my dosages from time to time, but I’ve seen no need to see what you would consider an MPN specialist. While hydrea is working, I’m goin to stick with it and when it doesn’t, I’ll start looking around and doing other things. There’s no one I would consider an MPN specialist in Lubbock. I’ve talked to some oncologists and I had to alert them about anagrelide. I would say they don’t quite understand Pegasys versus the normal interferon and things like that.

But that’s really kind of been immaterial to me so far because hydrea is working.

Beth Probert:

And you do have that challenge of being a little bit more remote, and I can certainly see how that makes a difference. I want to ask you one last question. You’ve been on hydrea a very long time. Do you have any side effects?


None really. My wife says I’m just not quite as wired as I used to be. But the things I read about, the possibilities that could be occurring, I don’t have to. I don’t know why; I just don’t.

Beth Probert:

That is fabulous news.


As a matter of fact, the doctors don’t know why either. They’d like to know; I would too.

Beth Probert:

Sometimes there’s just no explanation. James, I really appreciate getting to understand your story and we’re certainly going to come back to you and learn a little bit more about your journey. I’d like to go over to our doctor panel now.

I’m going to start off with Dr. Scott. We’ve heard a little bit about James’ story, and for our viewers could you explain a bit about ET; just help us understand really what it is and more importantly, what are we treating it with and what are the goals of treatment for ET?

Dr. Scott:

ET stands for central thrombocytosis or essential thrombocythemia, so it’s been called both of those names but the principle issue is overproduction of platelets. When you see the blood counts of the patient with essential thrombocytosis, their platelet counts are elevated. There can be a wide range of how elevated we’re talking about; it could be anywhere from 650 to even greater than 2 million.

When you have a platelet count that’s really high, like above a million, paradoxically there’s an increased risk of bleeding. That’s because the platelets have, on their cell surface, what’s called a von Willebrand factor cleaning protein. It actually cleans a portion of the blood that helps in clotting. So even though they have all of these platelets, and we associate platelets with preventing bleeding and bruising; when the platelets are extremely high like that, patients can have bleeding like nose bleeding, gum bleeding.

But the real problem with essential thrombocytosis is clotting. The No. 1 cause of death in people with ET is due to blood clots. And so James was just saying, he presented with this basically heart attack that he probably wouldn’t have survived if he hadn’t been in the hospital already. That can be a common presentation that you see with essential thrombocytosis blood clots.

So the reason to treat patients with essential thrombocytosis when they are treated is to lower their risk for blood clots; to lower the risk for thrombosis. The standard therapy for ET would be aspirin, a baby aspirin a day, and then you look at different types of risk factors to determine whether or not they need cytoreductive therapy. Cytoreductive therapy is basically given to lower the blood counts.

The risk factors that we would look at age, would be history of prior thrombosis, also white count is another risk factor that’s come out recently that can predispose people for risk of blood counts. But there is a prediction model; it’s called an IPSET prediction model and it’s an international prognostic model to determine the risk of thrombosis in patients with ET. You can look at that and you can see what risk factors this patient has.

Interestingly, JAK2 mutations, so having ET with a JAK2 mutation is another risk factor for thrombosis. But if they are high risk for thrombosis, either due to age or history of thrombosis, other risk factors like cardiovascular risk factors or due to the IPSET model, these are patients that are treated with cytoreductive therapy. There are many choices for cytoreductive therapy and two of them have already – actually, three of them have already mentioned.

That would be hydroxyurea, which is a common agenda that is used; another choice would be pegylated interferon or Pegasys; and then another choice would be anagrelide. There have been two randomized trials that I’m aware of comparing hydroxyurea to anagrelide. One showed a benefit with hydroxyurea over anagrelide; the other one was basically equivalent. But for me, my preference is hydrea unless they are a younger patient.

And in those patients I typically prefer Pegasys. I think there’s more data that’s needed to determine whether hydroxyurea or Pegasys would be the best first choice. There is a randomized trial that either has completed accrual or will soon complete accrual where they compared hydroxyurea to pegylated interferon; it’s frontline cytoreductive therapy for people with PV and ET. That will help us to answer the question which is better between hydroxyurea or pegylated interferon. But both of those would be choices for initial cytoreductive therapy.

Recently one of the big things that we’ve learned about with all myleoproliferative neoplasms is the underlying driver mutations. All of the myeloproliferative neoplasms share in common up regulation of the JAK-STAT pathway. And the same thing of course is true with ET.

And so there are three common mutations seen in ET: JAK2V617F would be the most common, and then calreticulin and then what’s called the MPL mutation. So you would test your patients for those mutations if you suspect a myeloproliferative neoplasm. They’re helpful not only from the standpoint of diagnosis but also prognosis. In regards to what’s coming out in the future, there’s a lot of understanding of other things that determine risks.

So as James was mentioning, he’s done well for such a long period of time. And then there are other patients who have more rapid progression to myelofibrosis, for instance, with a diagnosis of ET. We are looking at that and we’re starting to understand more about why that is the case. One of the things that have come out is what’s called secondary mutation that can develop in patients that potentially increases their risk of going into leukemia or myelofibrosis.

Beth Probert:

Wow, that’s been really helpful to understand. When you hear James has done so well for 23 years, the same medication, some fluctuation in his dosing, do you see that often? Do you have in your patient group; have you seen people have that same success as James? He kind of joked like nobody knows why I’m not having so many side effects and I’m doing well; what’s your take on that? Is that an anomaly or do you see that?

Dr. Scott:

We do see it, for sure and I will admit I’m what we call a tertiary referral center, which means I tend to be referred cases that are more recalcitrant that have failed other types of therapies. Among the patients that I see, I do have somewhat of a swayed pool of patients that typically have more severe presentations, more severe problems, that don’t respond as well to initial treatments and have more of a prolonged course with side effects and things like that.

But that just has to do with the nature of my referral base. But certainly among MPNs itself, there are many patients who do quite well with hydrea for many years.

Beth Probert:

Great feedback; thank you, thank you. Dr. Snyder, I want to talk to you a little bit about myelofibrosis. As I mentioned earlier, I have polycythemia vera and I do from time to time get a little concerned about progression. We’ll talk a little bit more about progression in a bit, but could you talk to us about myelofibrosis and give our viewers a summary of it, and also address the treatments and the goals of the treatments through the different therapies?

Dr. Snyder:

Sure. So we’re talking about the family of myeloproliferative neoplasms, and we started with ET; P vera of course is the other.

The third type is myelofibrosis. What we call primary myelofibrosis is patients who are diagnosed right from the beginning with myelofibrosis. But we know that both P vera and ET have the potential to transform over time to what we would call secondary myelofibrosis, meaning that they started with one condition and over time it transformed into myelofibrosis.

We see certain changes when that happens. As the name implies, there’s increased scarring, scar tissue in the bone marrow; that’s what myelofibrosis means. We tend to see decrease in some of the blood counts, particularly the hemoglobin with anemia and often the platelet count. And at the same time, often the white count is normal or it can be very elevated.

One of the clues for a patient with P vera for example, is say a patient was requiring a certain frequency of phlebotomies to maintain control of hematocrit or a certain dose of hydroxyurea and after awhile the doctor notices gee, we haven’t done phlebotomy in about five, six months and still the hematocrit hasn’t gone up.

Or, we’ve been on a certain dose of hydrea for a long time and everything has been stable, but now it looks like the hemoglobin is starting to drop. That’s the kind of clue that maybe it’s beginning to transform towards myelofibrosis and the bone marrow no longer is over producing red cells, but instead there’s a decreased production of red cells.

So along with that comes a number of other features. The spleen often enlarges around that same time and people may become aware of that enlarging organ in their abdomen. It may impact their ability to eat. There are also a variety of systemic symptoms that can occur. Sometimes we see this in patients with P vera and less so with ET/, but most commonly in patients with myelofibrosis.

Those are things like fevers, weight loss, night seats, fatigue, itching, and others. That’s kind of the clinical picture that you see when a patient either starts right off at the beginning or is progressing. So in terms of goals of therapy, there are a few issues. One, just like with the discussion about ET, patients with myelofibrosis are at increased risk for blood clotting and sometimes bleeding, as well. But our main focus is to help prevent blood clots from occurring. And so the same kind of baby aspirin is needed, is used.

In addition, there is a treatment called ruxolitinib or Jakafi is another name, that is FDA approved for treatment of patients with myelofibrosis. I’ll say that’s the only drug currently that is approved by the FDA for myelofibrosis, despite the fact that there have been many clinical trials that we may talk about with other drugs. But ruxolitinib was approved based on two main endpoints. One was a significant reduction in the size of the spleen for patients whose spleen is enlarged, with relief of symptoms from that big spleen. And second was control of some of these systemic symptoms that I mentioned. Those are the two main benefits that people can achieve.

There may be some other benefits such as gain in weight for people who have maybe lost weight. There may be some prolongation in survival; that’s a little bit of a soft call but some patients may benefit that way.

So that’s the main treatment that we think about for patients with myelofibrosis.

Beth Probert:

It sounds like from what you’ve described and of course what Dr. Scott described is the symptom burden is a big driver in how you’re going to treat patients and the symptoms are also a big indication of if it’s progressing, if it’s doing what it should be doing. You’ve both mentioned a little bit about some of the mutation. In previous programming, you’ve both talked about genetic testing. My next question is when someone is diagnosed, and I know when I was diagnosed with polycythemia vera, one of the first things that was done is that I was sent to the lab to get some genetic testing.

How important is genetic testing in both of your views? I’ll hop over to Dr. Scott real quickly. At what point are you suggesting genetic testing? Is it something you regularly do?

Dr. Scott:

Thanks for asking. So, it is part of the recommend workup now for myeloproliferative neoplasms; that all patients have this testing done. It’s important to realize that we’re talking about acquired mutations that occur in the vast majority of people. When the word “genetic testing” is thrown out, there’s this automatic misunderstanding that it means that it’s something that was inherited.

The reason why that’s important is because we don’t necessarily want to convey the false message that having a diagnosis of MPN means that your kids are going to get it, because that’s not what we’re talking about with this genetic testing.

These are acquired abnormalities in the vast majority of patients with MPN. There are very rare inherited cases of MPN that have been recorded, and in the vast majority it’s something that you acquire; it’s not something that you were born with. These mutations up regulate expression of a particular pathway in your body that’s called the JAK-STAT pathway.

There are three defied mutations to date, and they are JAK2. There are two different types; there’s the exon VC617F mutation, which most people with PV have, most people with MF have and most people with ET have. But there’s another mutation called the JAK2 exon 12, which is primarily only seen in PV.

That accounts for the small proportion of PV patients that don’t have the JAK2V617F. Those are the two mutations in JAK2. And then there’s an MPL mutation which can be seen in myelofibrosis, and in ET. Then the third mutation is what’s called the Calreticulin mutation, which is the newest one that’s been described. That’s seen in about a quarter of ET patients and about a quarter of myelofibrosis patients. Those are the three driver mutations that people acquire that’s been associated with myeloproliferative neoplasms. It is now part of the diagnostic workup for these diseases according to the revised World Health Organization criteria.

So that’s part of what we mean when we say genetic testing. That’s actually not the whole picture, because there are new types of mutations that have been described that I was talking about earlier that we believe are secondary events.

They have been associated with worst prognosis, higher risk of going into leukemia, and higher risk of going into myelofibrosis. One of those that has a negative prognosis connotation is what’s called the ASXL1 mutation. If you have that mutation, there’s data showing that these patients are at higher risk of complication like progression of myelofibrosis and like progression to leukemia.

So, I think that all patients should have mutational testing, what I’ll call mutational testing instead of genetic testing. And I think it’s important not only from the perspective of making the diagnosis but also in regards to prognosis. And honestly, it also helps a little bit with therapeutic decision-making. Because we know that ASXL1 mutations have a very bad prognosis, and that might be a patient that you would consider more aggressive interventions in, like stem cell transplant, for instance.

Beth Probert:

Thank you. Dr. Snyder, PV; we’ve talked about ET, we’ve talked about myelofibrosis and kind of flipping back to PV, when you find out that someone is positive and has a mutation, and let’s backtrack just a little bit. I realize I really didn’t delve into what PV is. Could you give our viewers a little background on PV, and then we’ll talk a little further about how the mutation plays a role with that and what we look for in that.

Dr. Snyder:

PV is polycythemia vera. It’s an over production of red blood cells. It shares the properties with its cousins, ET and myelofibrosis, the JAK-STAT pathway is over activated, usually because of the JAK2V617F mutation. So the cells in the bone marrow produce red blood cells become autonomous, if you will; they’re always turned on.

Normally the body regulates very well how many red cells are produced by the bone marrow. If the tissues in the body sense that there’s not enough oxygen coming and the message gets sent through a hormone called erythropoietin goes back to the bone marrow, stimulates more red cell production. More red cells bring more hemoglobin, brings more oxygen to the tissues and then erythropoietin production is turned off.

In P vera, that production of red blood cells becomes independent of the erythropoietin signal so those precursors are always churning out red blood cells, even though the body doesn’t need them. So the hemoglobin hematocrit can go very high, and that gets people into problems with risk of thrombosis as the main issue. Also, there are systemic symptoms that we talked about in myelofibrosis can also be seen in polycythemia vera.

So the treatment goals there are to again control the risk of thrombosis by keeping the hematocrit at a safe level, and there have been some well designed trials now showing that keeping the hematocrit under 45 percent is the desired goal. Some hematologists would even be a little more blasé about it and say well, it’s under 50 percent; that’s okay. But there are now clear data to show that you’re doing a disservice to your patient by allowing hematocrits to get over 45 percent. And frankly for women, it may even be better to shoot for 42 percent as the target.

And so the question is how do you get there, and phlebotomy is certainly the most direct mechanical way, if you will, of doing that. But cytoreduction with interferon is one option; hydroxyurea is probably the most common drug used in that setting.

 And now for the last few years, ruxolitinib is also FDA approved as a treatment for patients with polycythemia vera who have become intolerant to hydroxyurea or resistant to it.

Beth Probert:

Great. A couple of questions here, Dr. Snyder; how do you decide who gets what? For polycythemia vera, and I myself have seen this; I’m a member on so many different focus groups and such, and I see that there are people, one is getting Pegasys, one’s getting ruxolitinib, one’s getting hydrea; how do you decide who gets what?

Dr. Snyder:

The first question is does the patient need any of those drugs? We stratify patients with P vera into risk groups depending on age and the presence of other traditional cardiovascular risk factors or history of stroke.

But for a young person, say someone under 60 without risk factors, they can be maintained with baby aspirin and an occasional phlebotomy from time to time to maintain hematocrit under 45 percent. And they could go potentially for many years with that approach very comfortably.

The times that we say the cytoreduction is needed, there are a few things. One if it’s a higher risk patient, say over 60 with history of a stroke, other cardiovascular risk factors for example, or a patient who has other change sin their blood, not just the high hemoglobin but now maybe the white blood cells and the platelets are going higher, and also maybe the spleen is starting to get big. Phlebotomy is not going to help those features. That’s the time where you would start considering cytoreductive therapy.

We mentioned interferon, and Pegasys is the form of it that we now use. I do consider that more in younger patients because it’s a drug that may be harder for older patients to tolerate, so that’s the starting population. As Dr. Scott mentioned, there are trials going on to kind of compare head to head hydrea and interferon to see is one maybe better than the other. There are some suggestions that interferon may accomplish more than hydrea could in terms of some of the disease parameters. That’s not clear yet from the studies.

So that’s an option in a younger person. Hydrea really still is the main go-to drug for most patients who need cytoreduction. And then ruxolitinib, as I mentioned, it’s only FDA approved or indicated for patients who have already been on hydrea and have been either resistant to it or intolerant to that drug. So you can’t use ruxolitinib as frontline therapy.

The insurance companies won’t pay for it, and as we all know it’s a very expensive drug.

Beth Probert:

That’s a very, very common thing that we do here. We talked a few minutes ago about the mutations and doing some testing. Dr. Scott, I want to go back to you. How often do you suggest that patients have the initial genetic testing? I have found out that I’m a little unique compared to some of the other people I’ve been talking to about the frequency of genetic testing. So, how often do you suggest your patients get the genetic testing, Dr. Scott?

Dr. Scott:

I’m going to be honest with you; that’s a very difficult question to answer in a definitive way, because there’s really a lack of data to address the question that you ask.

And so, I think it’s a personal decision that I assist my patients in making in conversations with them. There are a lot of different factors that go into answering that question. So, first I’ll say that everyone should have it at diagnosis, and I think most people would agree everybody should have an extensive panel sent at diagnosis to not only include the three driver mutations, but also the associated mutational changes that can be seen like ASXL1 and EVH2.

Almost all patients now are having that done at diagnosis. In regards to how frequently, it depends on a lot of factors. One of the biggest ones would be what’s the underlying health of the patient? So, would there be a utility in knowing are things changing? One of the reasons why you would want to know that, for instance, would be maybe their disease is bad enough at this time to say okay, a transplant is warranted, but you’re going to follow the patient closely

 And if there’s new mutational changes or there are other signs or symptoms, then you might consider transplant at a later time. So when those patients are candidates for stem cell transplant, then I think one could argue that more frequent monitoring would be warranted. And so I might monitor those patients every six months to a year with mutational profiles.

I do think it should be done if there’s a change in symptomatology; if it looks like the disease is changing. So let’s say for instance you have an ET patient who’s been doing well on treatment for a long period of time, but they come in and they have low blood count now. And for the first time, they maybe need a red cell transfusion or maybe even a platelet transfusion. You’ve decreased their hydrea doses and their spleen has started to increase. You see immature cells in their blood smear. All of these are signs that maybe they’re going into myelofibrosis.


So if that were happening, that would be another reason that I would say okay, maybe we should do this mutational testing. So, I think it’s hard to be definitive; I think it’s an individual decision made after a consultation with the patient. And I think there’s a lack of data that we have to address your question, and that’s why you’re going to hear a lot of different opinions about when they should be done.

Beth Probert:

Absolutely. And James, I just want to go back to you for a few moments. Have you had genetic testing?


I think we all had it just to be sure about the Philadelphia chromosome. And I’m JAK2 positive, but other than that, no. I would like to interject sometimes the conversation has been once we find the right treatment; you’re going to stay on it. But if you get around a lot of patients, you’ll find that this drug works great for me for three years and now, for whatever reason I’m transforming; it isn’t working. Maybe I’m not transforming but it doesn’t work. But that’s the only thing I’ve had done.

Clinical trials and CALR and MPL I’m not sure right now what I’d change about my treatment. I haven’t gotten excited about that yet. But I’m aware of them through the conferences and through the Listserv.

Beth Probert:

Very, very interesting. You’re very connected and I know that when you and I talked before and we got to know each other, that you are very connected to what is out there and still kind of figuring out what do I need; things are going well. But James, you brought up a very good point. I’d like to ask Dr. Snyder; resistance. We hear the word so often, but could you talk to our viewers a little bit about what is drug resistance and what happens at that point?

Dr. Snyder:

Usually if you’re talking about resistance, say to hydroxyurea, a patient with P vera resistant to ruxolitinib, it usually implies – often implies that there is something new that’s occurred within those abnormal cells. We were talking about mutations, and that’s probably the main mechanism for the development of resistance; that a patient had a certain profile, let’s say they had the JAK2 mutation but no other secondary mutations.

Sometimes another mutation will develop in the course of the disease, and now even though for example with ruxolitinib, you may be inhibiting the JAK2 pathway pretty effectively; if a mutation has occurred that activates another pathway inside the cell, it may be able to bypass that blockade that the ruxolitinib was establishing and now the patient clinically becomes resistant. So it’s a time to wonder what’s happening at the molecular level and should we be looking at that.

Beth Probert:

If this fits into resistance and the whole issue, and we heard earlier from Dr. Scott talking about stem cell transplantation and I know that’s something that’s your area of focus. When you see resistance happening and you’re not finding another therapy that’s working, is that when stem cell transplantation is a viable option?

Dr. Snyder:

That’s a very good question, and not an easy one to answer.

Beth Probert:

I’m on a roll!

Dr. Snyder:

That’s the biggest question, really; well there are two. Who is the right candidate for stem cell translation, and when; when is the right time? We don’t want to compromise or jeopardize good quality of life that a patient may be experiencing with their current situation.

Stem cell transplantation, yes on the one hand it is a curative treatment potentially; that’s the goal of therapy with stem cell transplantation. But on the other hand, it’s a high risk approach as well, and there are risks of early death after transplant and if not death, then significant morbidity of complications that may affect the quality of life. So we certainly don’t want to rush into that approach. That’s the nuance of when is that right time.

I’ll just step back for a minute. We haven’t talked about DIPS or DIPS Plus, which I’m sure people are familiar with. That’s the Dynamic International Prognostic Scoring System for myelofibrosis that stratifies patients into low, intermediate one, intermediate two, and high risk. It helps to predict expected survival or average survival for a large group of patients with that category.

Generally speaking, once you get to intermediate two, the average predicted survival is under five years. That is to me sort of the minimum criteria to say we should be thinking about a transplant. But it’s not sufficient, because many people may be in intermediate two or even high risk and still have good quality of life. So I look for additional factors. One would be that a patient starts increasingly requiring red cell transfusion because of severe anemia; that’s one trigger. Because that would indicate that survival may be shorter.

The other is to look at the blasts in the blood. Patients may have none, or they may have a low number, say 1 to 3 or 4 percent; that hovers in that range. And then over time, something happens and now it’s 8 to 10 or 8 to 12 percent. Again, it may be one of those mutations that’s come along.

That gives you a sense that the patient is on the way towards transformation, so that’s another trigger to say this is the time to think about a transplant.

Beth Probert:

So definitely not a decision that is taken lightly; it is sort of a last sort of therapy, so to speak, a strategy to take because of the serious effects it could have on the quality of life, if I understand you correctly?

Dr. Snyder:

Again, in a sense it is. I like to say that I don’t like to take patients too early, nor too late to transplant. Because we want patients who are potentially going to benefit from it to have that chance. So too early means that a patient is doing well in their current therapy, they have a good quality of life, they’re doing the things that they need to do, that they want to do; we don’t want to interfere. Too late, that’s a relevant term because it’s hard to say that it’s ever absolutely too late.

But someone who’s transformed to leukemia, that’s a much more difficult situation. It’s not that we can’t transplant a patient in that situation; you need to go through treatments first to get the leukemia back into the more chronic stage. It complicates the whole picture; outcomes are not quite as good.

The other besides leukemia is the general organ function of patients as they get older and they have other issues. Their heart function, their kidneys, their liver, their lungs; those need to be in pretty good shape to be able to withstand the impact of a transplant.

Beth Probert:

So it’s a very intricate decision making process and highly specific, is what I hear you’re saying.

Dr. Snyder:

It is. I’d just like to get back a minute to what we’ve been talking about mutations.

As we’re learning more about what secondary mutations can be found and what their clinical impact is, they’re being incorporated into prognostic scoring systems so not just DIPSS, but there are things like MIPSS, molecular scoring.

So, incorporating those data along with the clinical parameters. We’re not ready yet, but there may be a time where we can define a patient’s risk through the genetic profile and allow us to say okay, this patient, even though clinically they’re doing well, we know that their risk for not doing well in the short term is X or it’s a much shorter timeframe, and we better not wait – or it would be better not to wait – to move to transplant since we had said that this patient is a candidate and they have a donor.

So we’re not going to wait and risk the chance that they would lose the benefit. We’re not there yet, but I think that’s the direction we’re hoping to head to in the coming years.

Beth Probert:

That’s great to hear about where we’re going with this; what we can expect in the coming years. It sounds like it’s going to get highly specific and very useful. Now I’d like to take the time to talk about some clinical trials. Dr. Scott, I’d like to have you start off with us. What’s new and promising? If you could talk about a few clinical trials that maybe are going on at SCCA, or that you’d like to share with us and then Dr. Snyder, we’ll go back to you and you can give us your feedback. So Dr. Scott, can you lead us into that subject?

Dr. Scott:

Sure. We have a trial with a drug called imetelstat for patients with myelofibrosis. The accrual is currently on hold. A single center phase II result was published in the New England Journal of Medicine showing there were some patients who were able to retain a remissionof their myelofibrosis with treatment with imetelstat.

This includes both molecular remission and morphologic remission. Molecular remission would mean that their abnormal mutations went away and it responded, and morphologic remission would mean that visually the fibrosis had improved significantly. The phase II trial is currently on hold and they’re evaluating data. I’m helpful that the drug will continue to be explored in clinical settings. It does have a novel mechanism of action; it’s what’s called a telomerase inhibitor.

As I said, the drug is known as imetelstat. There are many centers that were participating in that phase II study. We just opened a trial with pactritinib. Pactritinib is also a JAK inhibitor, and actually I think it’s probably better to call these drugs JAK-STAT pathway inhibitors, because not all of them actually work directly on the JAK receptor, so I think that’s important to know.

These basically inhibit the JAK-STAT signally cascade. This drug, pactritinib, is in clinical testing. It is not yet FDA approved. It’s somewhat similar to ruxolitinib but it does appear to cause less cytopenias, so less toxicities with lowering of the blood count. It could be potentially useful in patients with low platelets. That’s one of the chief toxicities that can be seen with ruxolitinib are Jakafi is lowering of the platelet counts.

So, I’m hopefully that this drug will be approved in the near future. It was put on hold for the FDA for a brief period of time, but as I said the drug is now being studied again in a phase II trial, looking at different dosings. There are many centers participating in that study.

And then we also have a transplant study that’s looking at giving JAK inhibitors before transplant in an effort to improve the overall condition of patients before they go into transplant.

This is specifically for myelofibrosis patients. Patients with myelofibrosis can have a higher treatment-related mortality with transplantation because of other things that are going on with their body like malnutrition, the fact that they have a very big spleen, and other factors such as organ involvement with fibrosis can lead to a higher treatment-related mortality. They also have a slightly higher risk of graft failure in comparison to patients with other types of myeloid malignancies.

So, we’re hopeful that giving a JAK inhibitor before transplant can help improve the post transplant outcomes. So those are the three major trials that we currently have open. Of course there are other centers with really exciting drugs in development, as well.

Beth Probert:

Wow, that sounds very exciting. I know that I can say just hearing that there’s such a focus with MPNs and these trials.

And Dr. Snyder, what is going on in your neck of the woods at City of Hope and other trials that you’d like to tell us about?

Dr. Snyder:

Yes, we have a number of trials. We have the pacritinib and the imetelstat trial as well. We have two other trials that are for patients who have failed or progressed on ruxolitinib that have totally different mechanisms of action sort of outside of the pathways we’re talking about. One is called SL401, Stem Line 401. It’s an interesting sort of an immuno toxin; it’s a dual functional molecule that has an IL3, interleukin 3 portion that’s linked to a diphtheria toxin. It’s somewhat like a Trojan horse type of thing.

The cells in myelofibrosis and other hematological malignancies have an interleukin 3 receptor on their surface, and this IL3 molecule will bind to that interleukin receptor. That complex is taken inside the cells and then the diphtheria toxin is released and is able to kill the cell from the inside. So that’s one mechanism.

There’s another approach; there’s a molecule called CD47 which has been referred to as the “don’t eat me” signal. So, macrophages which are big cells in the body and in the blood and tissues, their name means big eaters. They like to eat foreign cells, tumor cells, bacteria, etc.

But some of the tumor cells or the malignancies become very clever and they have this protein called CD47 on their surface that sends a signal to the macrophage: don’t eat me, stay away and helps the cells to survive. So there is an antibody against the CD47 that binds to the CD47 and interrupts that pathway and then allows the macrophages to do their jobs, which is to eat these abnormal cells.

So that’s another approach that’s being tested not just in myelofibrosis but in other conditions as well. There is some data, sort of preclinical, that this approach may actually reverse fibrosis in some models, so it’s kind of intriguing particularly for patients with myelofibrosis.

The thing I will mention, we know that ruxolitinib is the only FDA approved drug so far; there have been several others unfortunately that have gotten just so far and then because of toxicities have been taken off of the table. But to me, I think another approach is combination therapy that is taking ruxolitinib as the base and then combining it with the second drug that has a totally different mechanism of action, and the two of them then perhaps can synergize and kill off the cells.

Those are trials, a quite a few of them going on around the country and I think those have a lot of promise.

Beth Probert:

And when you mention combination therapy, you both have mentioned it; are we looking at more of a personalized medicine? What’s your feeling on that, Dr. Snyder? Is it at that point we’re getting more personalized and we’re looking at that one person and saying this is going to work more specifically for you?

Dr. Snyder:

I think that’s a very good point, and I think as we learn more about mechanisms of action of some of these drugs and we talk about targeted therapy, it is something that can be very individualized potentially.

We talked about the genetic profile of what mutation someone might have. And so there may be a second drug beyond ruxolitinib that targets one of these secondary mutations that a patient might have. And so for that person with that particular combination of mutations, ruxolitinib plus this second targeted therapy may be just the right thing for them.

Even a drug like imetelstat, at least on data based on small numbers from Dr. Tefferi’s work suggested that there may be a mutational profile that defines the best responder type of patient, and conversely, patients who are unlikely to respond at all to that drug. That would be terrific to be able to say okay, don’t waste your time with this drug for this patient, but go in this direction because this is much more likely to be effective.

Beth Probert:

It could be life saving; it could get to the result much quicker than going through another therapy that just is not going to do it.

Dr. Snyder:

For sure.

Beth Probert:

Very interesting. So, quick question for you James, before we move one. We’ve talked about a little bit about clinical trials. James, have you considered a clinical trial? Or you’ve been stable; would you consider one in the future if your condition changed?


Very definitely. But you know one thing we haven’t touched on and what drives the doctors crazy, probably, is the psychology of the diagnosis. We as patients quite often tend to think if I just took thing X or had therapy Y, I would be okay. And as these doctors know, it’s not that simple. But yeah, if I thought I would benefit from it and I needed to, of course.

Beth Probert:

You bring up an excellence point, James, because it is the psychology behind really how we… you’ve been doing this for 23 years and you obviously have educated yourself which helps you to understand what’s going on, which balances everything out. I know that I, as I mentioned, I’m in some support groups and I see people doing combination therapy. And it’s the old adage: he’s doing two things and I’m doing one, and I’m sure doctors don’t want to hear that.


There are other patients; the last thing they want to know is anything about the technical side. Just: I’ll go to the doctor and the doctor says this, and I do that.

Beth Probert:

There is, indeed.


I think even I’m on one extreme and they’re on the other.

Beth Probert:

Definitely. We have a little bit of time, and Dr. Scott, I’d like to go to you.

We have a question from Pauline, and she asks: is there any way to determine declining blood counts such as anemia and thrombocytopenias are due to drug side effects or the disease process? And she goes on to say: my husband has been on Jakafi for just over a year, and these declining cell counts began a couple of months ago. So let me know if you need to repeat that. Could you comment?

Dr. Scott:

No, I’ve got it. There are things that are helpful to distinguish between drug toxicity and to these progressions, and one is timing. The cytopenias that are experienced with ruxolitinib, as long as the dose has remained the same are usually early side effects. So, most of the cytopenias are during the first eight weeks of therapy. So if you see early cytopenias, it’s more likely to be a drug effect. If you see later cytopenias, it makes you more concerned about disease progression. And to know definitively of course, you could repeat a marrow aspirate or a biopsy and that can be helpful.


And then other co-associated symptoms or size like increasing spleen size, or return of puritis can all be signs that the ruxolitinib is no longer working. But as I said, the side effects of drug-related are usually early events within the first eight weeks when treated with ruxolitinib.

Beth Probert:

Great. I think that’s going to be some good feedback for Pauline and some talking points that she can bring back to her specialist. We are coming to a close, and what I’d like to do now is ask each of our panelists just for some final thoughts or comments. You know, I’m feeling very optimistic from this discussion this evening to know that there’s just constant thought on these rare diseases, and there are clinical trials and research. But Dr. Snyder, let me start with you and if you could just give us some thoughts you’d like to share with us that you think would be meaningful?

Dr. Scott:

Sure. These are obviously difficult diseases because they’re hard to cure. But I will focus first on the role of stem cell transplantation as the only current only curative approach. We are trying to improve both the efficacy and the safety of that approach, and Dr. Scott mentioned studying the role of JAK2 inhibitors in the peritransplant time, for example, as one. It turns out that ruxolitinib also is an effective drug to treat one of the main complications, which is graft versus host disease. We have a trial actually looking at it as a prophylactic way to prevent graft versus host disease.

So just to say that there’s a lot of work being done on that front to try to improve outcome for patients with the transplant approach. But of course we’d all love to have – we all think about CML, that was mentioned; the Philadelphia chromosome. The Gleevec story is kind of the model that we wish we could duplicate with many of the diseases that we treat, recognizing at the same time that it’s very unlikely.

Because in a way, patients and doctors were lucky with CML because it’s a very simple biology. And if you come up with a drug like Gleevec that targets really the definitive driver of that disease, you can have dramatic clinical benefit.

Not quite the case with the myeloproliferative neoplasms; more complicated. Ruxolitinib, we all hoped this is going to target the JAK-STAT pathway; it’s going to shut it down and restore normal hematopoiesisIt’s not quite that simple.

But we certainly have hope and optimism that with many of these trials that are going on, particularly as I said clinical trials with combination therapy, that that is going to get us closer to that point. I’m a transplanter but I would love to be put out of business because we have drugs or a drug that is so effective, transplant is just a thing of the past. Hopefully, one day that will be the case.

Beth Probert:

Wow, and that is very powerful. I will always remember those words; that’s really great. Dr. Scott, can you give us some final thoughts on just anything to let us know again what we should be looking for, what’s in the future, some optimism; whatever you feel is meaningful?

Dr. Scott:

I think there is a lot of hope. When you compare what we know about myeloproliferative neoplasms now with what we knew about them six years ago, it is really remarkable. It was in 2005 that the first publications were published about JAK2, and we began our understanding of the underlying mechanisms of myeloproliferative neoplasms.

And over those 11 years there have been a lot of advances. So, I’m very encouraged by not only the clinical trial work that has been done, but also the basic science and the expansion of our understanding of these diseases. So when you compare what we have now with what we had ten years ago, it is really remarkable. I also do transplants but I do non transplant as well and I hope to be driven out of business. I would be okay with that.

Beth Probert:

That’s wonderful to hear; again very optimistic. James, you and I were chatting a little bit before our show started. James was commenting how even from 2014 to 2017, that there have been so many changes in progression and therapies. And James, I’ve got to say you’re like an MPN warrior.

You’ve been dealing with this for 23 years, you’ve been not only optimistic; you’ve been so resourceful, you are a born researcher. I would love to turn the stage over to you right now. And if you could give us some of your thoughts, some things you’d like us to know and help us feel more optimistic as we go forward.


Well, I’m very optimistic; I mean it’s just amazing compared to 1994. Actually, I was one of the data points in some of those 2005 studies. But there’s the MPN Research Foundation – let me throw in a plug for them, patients; they have funded some really specific studies. If you can’t go to conferences nearby or Scottsdale or New York – Dr. Silver runs one in New York; I know they’re in Seattle and all over now, get involved in a focus group. I drive to Dallas just to be around a focus group of those people. It’s tremendously empowering to talk to other people. Because your friends are like, you don’t really have a problem; you don’t look sick or anything, you know?

Beth Probert:

That is something that MPN patients hear quite a lot. But go ahead, James.


I’m just excited. When I have to get more knowledgeable and detailed about these things, I will but I’m not there yet. My goal is like the physicians here; I intend to die of something else. It didn’t get me the first time; I’m planning on no second time.

Beth Probert:

I think that is wonderful. You know, I would like to say that optimism is what is going to get us through this condition. I don’t tell too many people because I’m very optimistic, but I do agree with you James; I really connect well with people in focus groups. I drive sometimes about two hours each way into Los Angeles to see my specialist because that’s of value to me. If I ever could give advice, it would be make sure you’re connecting well with your specialist.

And that they are, like Dr. Scott and Dr. Snyder, very well versed in their field and in their clinical trials and things like that. So, wrapping up, a big thank you to our panel; I really believe that this webinar this evening gave us a great deal of information. Dr. Scott, Dr. Snyder, you both have very busy schedules so appreciate it. And James, you too took time out tonight so thank you all.

We have an upcoming webinar I’d just like to talk about really quickly on September 20th, and it’s what you can do to advance MPN research so I hope everyone will join us then. We will also be showing this video again and again. So thank you for our panel and thank you all for taking the time to watch Patient Power. Good night.

Clinical Trial Mythbusters: Are Clinical Trials a Gamble for Me or My Loved One?

Is a clinical trial right for me or is it a gamble with my health? How will my loved one be affected? Do the risks outweigh the benefits? Watch as a panel of experts, including an oncologist, trial coordinator, and patient advocate as they debunk some of the myths around clinical trials. Listen to hear the patient voice and perspective for getting the best care-making decisions about clinical trials.

Watch to learn:

  • What are common clinical trial myths?
  • Why should patients participate?
  • How can patients navigate the system?
  • How can I or my care partner work with my medical team?

Clinical Trial Mythbusters: Are Clinical Trials a Gamble for Me or My Loved One? from Patient Empowerment Network on Vimeo.


Living Well with MPNs – Tips and Strategies for Managing Symptoms and Side Effects of MPNs

Tips and Strategies for Managing Symptoms and Side Effects of MPNs

As part of our Living Well with MPNs webinar series a panel of MPN experts and patients discussed managing life with an MPN. The panel shared advice on managing fatigue, itching, night sweats, enlarged spleen and other symptoms. The experts explained why symptoms occur and stressed the importance of communication with your healthcare team. Tune in to learn more.

Clinical Trial Mythbusters: Are Clinical Trials a Last Resort Treatment Option?

Are clinical trials only for patients who run out of treatment options? Watch as our expert panel answer questions as they debunk common myths around clinical trial participation. Tune in to hear the patient perspective and expert advice for making decisions about clinical trials.

Watch the video and learn:

  • What is a trial and when should I consider one?
  • What are common clinical trial myths?
  • If my cancer center does not offer a trial, what should I do?
  • How can I stay informed?
  • Is there financial assistance to be in a trial?

Clinical Trial MythBusters: Are Clinical Trials a Last Resort Treatment Option? from Patient Empowerment Network on Vimeo.


Andrew Schorr:

Hello and welcome to this Clinical Trials MythBusters program.  I’m Andrew Schorr from Patient Power joining you all the way from Barcelona, Spain.  We’re here for a conference.  You’re about to meet folks from across the U.S. and wherever you’re joining us.  Thank you so much for joining us.

Thanks to our wonderful partner, the Patient Empowerment Network, and also the Coalition for Clinical Trial Awareness and the Alliance for Patient Access.  And thank you to our sponsors, they all start with A, Astellas, Amgen and AbbVie.  They help make this program possible.

We have a lot to talk about in helping debunk the myths about clinical trials and hopefully raising awareness and understanding for you and your family, so you can consider a clinical trial and see whether it’s right for you.  And I can tell you, in so many areas of cancer now there’s exciting research going on. But if you want to get the possibility of tomorrow’s medicine today, and it happened for me with chronic lymphocytic leukemia, being in a Phase II trial way back in 2000, 10 years before the drug combination I received was approved.  I know it was life-saving for me.

And I want you to meet our first guest.  It was life-saving for him, and that is Pat Gavin.  Pat joins all the way from Marne, Michigan, which is outside Grand Rapids.  Pat, thank you so much for joining us on this program.

Pat Gavin:

Thanks for having me, Andrew.  Glad to be here.

Andrew Schorr:

Now, Pat, I want to go over a little background about you.  I believe that you’ve been treated for three cancers, right?  Pharyngeal head and neck cancer, in 2007, right?

Pat Gavin:


Andrew Schorr:

And also you were treated for melanoma 2008, and then in 2014 prostate cancer.  Now, you were in a Phase II trial for that pharyngeal head and neck cancer.  Do you believe it made a big difference for you?

Pat Gavin:

Well, that trial is absolutely the reason I’m here today.  My oncologist described it as we had the experience of witnessing a miracle.

Andrew Schorr:

Let’s meet one of our medical specialists who is joining us, who has been on our Patient Power programs before and our lung cancer programs, and that’s Dr. Charu Aggarwal.  She joins us from Penn Medicine, the Abramson Cancer Center in Philadelphia.  She’s a lung cancer specialist and also a head and neck cancer specialist, very active in trials.  Dr. Aggarwal, thank you for joining us.

Dr. Aggarwal:

Thank you, Andrew.  Thank you for having me here.  I’m delighted to be part of this program.

Andrew Schorr:

Dr. Aggarwal, you have a lot of research going.  It takes patients wanting to participate for us to ever have approved medicines, right?

Dr. Aggarwal:

Absolutely.  And I think that’s key in clinical trial participation, to get drugs to patients early.

Andrew Schorr:

All right.  And certainly in the area of lung cancer and many other cancers now there’s a lot happening, and smart researchers like Dr. Aggarwal are trying to prove some things that really seem like they would make a lot of sense, but we patients have to participate, be their partner.  I’ve seen that.

Dr. Aggarwal, lung cancer is a good example, but you’re an oncologist, and you see many different areas that are changing fast.  What would you say to patients about the opportunity, like I said, did it give me tomorrow’s medicine today?  Or, Pat, who feels he’s alive because of that.  You must see that a lot.  Doesn’t always work out, but it’s happening more and more, isn’t it?

Dr. Aggarwal:

It is definitely happening more and more.  Clinical trials are really accelerating our ability to get patients, like you said, tomorrow’s medicine today.  In the last five years, we’ve had upwards of eight to 10 drugs approved for lung cancer alone, and it would not have been possible without patients’ participation on clinical trials.

As we understand the biology of diseases better and as more medicines are available to us, the only way to access them and the only way to get FDA approval is through clinical trials.  And we’ve certainly seen that for lung cancer, but we’ve also seen that for head and neck cancer, and immunotherapy is now possible because of clinical trial participation.

Andrew Schorr:

Right.  And I’m living with two cancers, chronic lymphocytic leukemia, where there are many new drugs now, and now we’re looking at trials with combinations of new drugs.  And then I have another condition, scarring in the bone marrow, myelofibrosis, and I was very grateful that a new drug had been approved for that. And I’ve taken that drug now four-and-a-half years, a genetic inhibitor, and I’ve met patient number one, who is in that trial, and I give him a big hug.

Now, Pat, what do you think are some of the myths?  You know, you meet people all the time.  What do you think are some of the things that people just think are true but really aren’t?  Maybe you could tick some off.

Pat Gavin:

Well, one of the big myths out there is that there’s going to be a placebo arm, and there are not placebo arms to treatment trials, unless the standard of care would be a wait and watch, which is relatively rare.  So you’re always going to get either standard of care or a combination that includes standard of care and the test drugs or the—test drugs.  You’re never going to be left out there with just taking a sugar pill.

Andrew Schorr:

Right.  So let me go over that with Dr. Aggarwal.  People I think are—have heard about trials for other illnesses, but we’re talking about cancer now.  Your patients don’t get just a little white pill with nothing in it, right?  They either get quality care, standard of care, or they get something new.  Is that correct?

Dr. Aggarwal:

That’s correct.  So the era of placebo?controlled trials is almost over, and I say almost because in the metastatic setting or in the stage IV setting or incurable setting we almost always never use placebo anymore.  We are either randomizing patients to standard of care or meeting standard therapy, the chemotherapy, be it a pill, be it targeted therapy or immunotherapy, and we compare patients to that approach and introduce the experimental approach on the other hand.

Now, if there are patients that have standard of care as observation, then, of course, that observation arm does become our randomized arm.

Andrew Schorr:

Okay.  And may I ask what Pat was taking, or like I know in leukemia we have people who are in watch and wait.  So we have some people who are in watch and wait, okay.  So I get that.

Pat, what’s another myth, do you think?  So one was where you get a placebo, so we heard no.  So what’s another myth, do you think?

Pat Gavin:

I think there’s always a feeling that I’m going to be just a guinea pig, and that’s the one thing I think I hear most often from people is I don’t want to be a guinea pig.  I want to make sure that I’m getting a treatment and not being exposed to things that are unsafe.  Of course, there’s always a certain amount of risk with any trial that we participate in, but the chances of some of the things happening that you might see on the comedy TV shows just aren’t going to be there.

Andrew Schorr:

Okay.  Dr. Aggarwal, let’s go over that.  So, first of all, you’re at a major university center, University of Pennsylvania and Abramson Cancer Center.  What sort of panels in decision?making of smart minds are there going to whether you’re even going to go ahead with a certain trial?  I think you call it an investigational review board.  Tell us a little about the process of deciding whether you’re going to have a trial at your institution at all.

Dr. Aggarwal:

Yes.  So there’s a very thorough review of clinical trials, and these are vetted through several committees both in terms of ethical review as well as scientific review.  And, you know, when my patients say to me I don’t want to be a guinea pig, I really try and figure out what is it about the trial that they don’t want to do?  Is it the fact that they don’t want to get the investigational drug, or is it the number of tests that are involved in association with receiving that drug?

And I think, you know, most of the time, 80 to 90 percent of the time, I’m able to answer patients’ questions and concerns regarding their guinea pig concern, and most of the times actually it’s related to the fact that they don’t want to undergo extra tests or procedures that they wouldn’t have otherwise.

As soon as they hear that this is actually a drug where it may benefit them and they’re not just going to get a sugar pill, most patients are actually interested in clinical trial participation, because they’re here to really help themselves and to get something that can help their cancer.

Andrew Schorr:

So, Pat, another concern—well, I guess one limitation of people being in trials is people don’t even know about them, you know, not only don’t understand what a trial is but have not even been told that it’s an option, and that’s a problem in the U.S. today, isn’t it?

Pat Gavin:

Absolutely.  I even think it was a problem for me.  I didn’t know that a trial was going to be available in my home town.  If it wouldn’t have been for my oncologist recommending it to me, I probably wouldn’t have joined.  Fortunately, today I think patients are getting more knowledgeable about trials that are out there, and they’re hearing more and have the interest in joining a trial, and they’ll recommend it to their oncologists and tell them that they are interested.  But not knowing about them is a big problem.

Andrew Schorr:

Okay, Pat.  So for our viewers today, what question or questions would you urge cancer patients or family members to ask today so that they have the awareness of trials that might be right for them?

Pat Gavin:

Well, the first thing I would do is I would offer to my oncologist that I’m interested in being in a trial.  And I would ask what type of trials are available for people with my cancer, and what would you recommend as far as the trials that you see out there that you think is right for what I’m facing today?

Andrew Schorr:

Okay.  All right.  Well, now joining us I think is Mary Ellen Hand, who has been at the Rush University Medical Center in Chicago for many years and also works with lung cancer patients but has been in oncology for many years.  Mary Ellen, first of all, thank you so much for being with us.

Mary Ellen Hand:

You’re welcome.  Sorry for the technical difficulty.

Andrew Schorr:

It’s okay.  Thank you for being with us, Mary Ellen.  So from your point of view, what’s a myth that comes up a lot for people?  We’ve been talking a little bit with our other guests about whether with you get a placebo, no, whether you’re a guinea pig, no.  Are there other myths that you can think of that you really want to talk about now?

Mary Ellen Hand:

I think that people sometimes come to this thinking I don’t want to do something because I don’t—as you’re saying, a guinea pig or be in uncharted territory as opposed to having an opportunity to have a therapy that may be more impactful in their disease and help control their cancer better.

And, secondly, I will have people who have an out?of?network insurance or something that doesn’t allow them the flexibility to maybe even come to our institution or somewhere else for their therapy, and they think cancer trials are free care, anything you get if you’re on a trial is free.  And what is true is that ordinary customary charges for things like blood tests and scans and doctor appointments and the medicines that are approved are billed to your insurance, and what the company might provide that’s being tested would be the thing that’s provided free to you.  And so I think that that’s a misconception that many people have.

Andrew Schorr:

Okay.  Now, can a major medical center like yours help a patient discover the financial issues related to them, maybe even work with their insurance company to see are there options for them related to being in a trial?

Mary Ellen Hand:

I think over the last couple years in particular things have become much more complicated for people.  You know, some people sign up for Medicaid or Medicare replacement policies in the different states.  There’s Medicaid with places—Medicaid policies that don’t allow people flexibility.  But certainly that’s our job is to help people find out where they could go and if they’re eligible for a trial to help them get to that trial, and some of that is people who have—fit a particular niche.

And some people need to be well enough to travel, you know, if they need to—if the trial is out of our ZIP code.  Here in Chicago we’re very collegial in head and neck cancer and lung cancer and, you know, multiple other cancers.  You know, if the trial exists five miles from here, we’ll facilitate the patients getting on that trial there.

I think that medical records, one of the many—one of the most common medical records systems is available at many institutions across the country, so people can have access to the reports for another hospital.  Otherwise, there are coordinators and people who can make sure that all of that gets to the research nurse and gets in the hands of the person who is going to take care of that.

And then at our hospital, and I’m sure across the country, we make sure that they get the imaging so that they have something to compare it to, and then that’s uploaded into your chart, you know, at the other facility so that everybody has the right information to take care of the patients.


Andrew Schorr:

Okay.  Dr. Aggarwal…

Dr. Aggarwal:

I would just add…

Andrew Schorr:

Go ahead, please.

Dr. Aggarwal:

I would just add that this is a very common concern about the financial responsibility for clinical trials.  And here at Penn we are actually trying to make this process very, very transparent so that when I discuss a clinical trial with a patient actually our consent forms reflect what will be the standard of care costs and what will be sponsored by the clinical trial.

And, in fact, we do facilitate meetings with a financial counselor so that if a patient has concerns about what will be covered versus what will not be covered will be discussed at length with a financial counselor.  And that actually has gone a long way in allaying some of the concerns that patients have when going on clinical trials.  So, you know, it goes hand in hand with what Mary Ellen was saying, that I think once patients hear from the oncologist that there’s another level of—from a finance person I think that really goes a long way.

And I would urge patients to actually discuss and ask the facility where they’ll be treated if there is such a person who can discuss with them, because most academic cancer centers do have this facility.

Andrew Schorr:

So many people are treated, you know, by a local oncology clinic, but often they can work in partnership with an institution like yours, Chicago, Philadelphia, others around the country.  How does that work?  How can that work where they can be in your trial but maybe some testing or some other things, or do they have to commute to your institution maybe from a distance all the time?  Let’s start with you, Dr. Aggarwal.  Can there be more partnership, or are more trials available now in the community as well?

Dr. Aggarwal:

So a lot of partnerships exist between community physicians as well as academic physicians, so I see patients for my community oncology colleagues all the time, and the goal really is to make access easier, you know, the access to clinical trials and drugs easier.  So while the administration of the drug and the monitoring of the drug may happen at the academic center, there are many tests and imaging procedures that can occur in the community.

And the goal is also to make this easier for patients.  So if a patient is 25 miles away, I try not to drag the patients here just for a clinical exam or just for a scan.  You know, so I would facilitate them getting scans closer to home with their outpatient oncologist and then ask them to perhaps bring a CD with them for review.  They can get their blood work done closer to home.

So there are lots of things and lots of procedures where we work synergistically with their community physician hand in hand to try and facilitate all of these procedures so that they don’t have to keep traveling all the time.  So we certainly do that.

Andrew Schorr:

In Chicago, too, Mary Ellen?

Mary Ellen Hand:

Certainly.  You know, there are some things that the study requires.  If an infusion needs to be done onsite, that’s what happens.  You know, we have patients who travel across the country that might have a genomic mutation.  They may be looking for second or third generations of drugs, and so those people may travel.  So they have their local oncologist, meaning near, whether that’s someone in the community or someone in the academic center.

I think that’s another thing, is that patients are concerned that their doctor, whom they’ve forged this relationship with and the nurse, they think they will be upset if they go somewhere else.  And then instead of knowing that it’s a great opportunity for us to advance the body of knowledge but it’s also—we’re always encouraging and hopeful that people can get onto a clinical trial.

And so I think it makes them feel really good that people have these connections.  I think they like to know they’ve talked, they like to know that everybody’s on the same page and this many more layers of care take care of that.

Andrew Schorr:

Pat, let’s pick up on that.  So…

Mary Ellen Hand:

Their problem, their knowledge, all of us together, so.

Andrew Schorr:

Right.  Well, Pat, let’s talk about that.  So people have a doctor, maybe the one who diagnosed them, and they have a close relationship with them, and they’re afraid of losing them.  What do you say to people?  Mary Ellen spoke about that but from your perspective.

Pat Gavin:

Well, I think it depends somewhat on the trial and where they’re going to be available.  I received all of my care through the clinical trial locally at the Lacks Cancer Center here in Grand Rapids.  It was a trial like many others that are in the national clinical trials network, and the NCI-sponsored trials are generally available at the NCORP sites, and there are a lot of those around the country.  I was fortunate to have one of the original ones here in Grand Rapids by the Cancer Research Consortium, and those trials are available in academic centers, they’re available in community cancer centers like I had.  So it depends on the trial.

Now, some of the pharma trials may be a little more isolated and localized to specific hospitals for some of their early?phase trials.  Talk to your oncologist again.

Andrew Schorr:

We are getting questions.  And so I mentioned I have chronic lymphocytic leukemia.  This came in from George, who also has CLL.  He said, I’ve had no treatment, but it’s likely that it will be needed very soon, and it seems that Medicare patients are treated somewhat unfairly when it comes to available financial assistance for oral chemo, oral drugs that are now in trials often, Mary Ellen.

And so he says, are we likely to run into problems with clinical trials if you’re on Medicare?  So, Mary Ellen, any guidance about that, Medicare patients and trials?

Mary Ellen Hand:

No.  I think that we’re an aging America, so I think that we want to be sure that patients who are on Medicare have access to clinical trials.  So I think what he’s speaking particularly to is the co?pay of some of these medications is so very high, and co?pay assistance programs are not always built to support and help them in this.  But I think that if he’s going to be eligible for a clinical trial, he should, you know, number one see if he’s eligible. And then hopefully the place that he’s at will be able to help navigate that for him so that he can be—you know, be eligible to participate in that trial.

Andrew Schorr:

Okay.  One other question, Dr. Aggarwal.  This one comes from Stacey.  Stacey also has leukemia, and we’ve had oral drugs being developed a lot now for various leukemias, this is CLL.  And so there’s a clinical trial underway combining two of these oral agents, ibrutinib (Imbruvica) and venetoclax (Venclexta), is underway at MD Anderson in Houston, and the same trial is supposed to begin at Northwestern in Chicago near where Mary Ellen is, and that’s in May.

And she says since there’s a four? to five?month period before the introduction of venetoclax following ibrutinib, what would be the chance that I could join the trial at Northwestern in May, or would this be something I would have to direct to the doctor leading the trial?  She’s wondering about since the drugs get combined but sort of one after the other, can you sort of start, and how does that work?

Dr. Aggarwal:

So I would say that each clinical trial is different in terms of how they’re designed and what eligibility criteria are outlined.  I would really encourage participation—or I would encourage her to speak with this—speak about this trial with a physician or contact the PI of the clinical trial at MD Anderson…

Andrew Schorr:

Principal investigator.

Dr. Aggarwal:

…principal investigator to try to get some clarification of that.  There are some trials that prohibit previous therapies or previous ibrutinib, for example, prior to enrolling in a combination clinical trial, and there are some trials that allow prior participation.  In some instances, they may see progression on ibrutinib prior to combination therapy that is ibrutinib and venetoclax.  So I think it’s a matter of finding what the check boxes on the trial are, and talking to the principal investigator would be the best way to go about it.

Andrew Schorr:

Okay.  Here’s a question for Pat.  So, Pat, one of the things I wonder about is there are people who are on modern therapy now like for instance some of these drugs we mentioned, people are doing well on ibrutinib or people are doing well on venetoclax, or people are doing well on some of the lung cancer drugs.  Now, none of us know how long they’ll last, so they say, well, why should I even think about trials if it’s not broken now?  What would you say to them?

Pat Gavin:

We have to as patients look at clinical trials as a form of treatment, and it should be something that should be considered right from the beginning.  I hear people saying that, well, I’m going to go with what I’ve got so far, and if that doesn’t work and nothing else is an option, then I’ll use it as a last resort.  Now, in some cases a clinical trial may be a last resort, but in other cases, like you mention, there may be things about early treatment that would disallow you from participating in a clinical trial later on, so it’s best to be talking about clinical trials as an option right from the beginning.

Andrew Schorr:

Well, you know, this is a series we’re doing, and so we’ve covered some ground today, and I just want to recap a couple of things.  We talked about the phases of trials.  We talked about financial issues, and there are counselors to help you related to that.  Pat and I told the story of each of us thinking we wouldn’t be alive if we hadn’t been in a trial.  We talked about genomics.  So we’ve covered a lot in our first one, and we have another program coming up late in June.  Dr. Aggarwal, was this a good start?

Dr. Aggarwal:

This was an excellent start.  I think we definitely look forward to more patient participation on further trials, further programs like this.

Andrew Schorr:

Okay.  And, Mary Ellen, do you think we made a good start today, and hopefully people will now consider trials?

Mary Ellen Hand:

I think that it’s always just good to have more education, so whatever venue we can give that to people, whether it’s talking one on one with their physician or nurse or whether it’s online, to give people permission that they can get more information.

I think the other important thing to know is the criteria we talked about is you can’t—if you are truly getting a second opinion, you should get it before you start something, because you don’t want to have started a treatment that you get one dose of something that blocks you from access to a clinical trial.  Or you don’t want to have your genomic testing done yet, and yet you’ve started chemotherapy.  So sometimes it’s just educating people that, you know, if they’re not very sick, there’s time to get more information.

Andrew Schorr:

Good, good point.  Pat, what’s a final comment from you?  What do we want to leave people with, hopefully have more people think about trials, get access to them and have greater participation?

Pat Gavin:

Well, every time I talk I say I’m alive today by the grace of God and the fact that I participated in a cancer clinical trial.  They make a difference.  They’re the reason why you and I are alive today.  They need our support.  If clinical research is going to advance, we have to have patients in clinical trials.

Andrew Schorr:

Right.  So if we want progress and cures for the cancers that we’re living with, we’ve got to work with folks like Mary Ellen, Dr. Aggarwal and the other folks involved in research around the world, really.

Well, I’m over here in Europe, today in Barcelona.  This is a worldwide broadcast we’re doing.  Pat Gavin, I want to thank you so much for joining us from near Grand Rapids and wish you good health, Pat.  Thank you for being with us.

Pat Gavin:


Andrew Schorr:

And, Mary Ellen Hand, thanks for joining us again on our programs and in Chicago and 34 years of devotion to us, Mary Ellen, you keep going.  Thank you for being with us.

Mary Ellen Hand:

You’re welcome.

Andrew Schorr:

Okay.  Dr. Charu Aggarwal from Penn Medicine, the Abramson Cancer Center in Philadelphia, thank you for being with us again on one of our programs.  And thanks for the research that you’re moving forward with and your devotion to patients with cancer.  We hope that—well, we know it makes a big difference, and we look forward to you having great discoveries in partnership with patients moving forward.

Dr. Aggarwal:

Thank you for having me.

Andrew Schorr:

Great program.  Our next program will be coming up on June 21st, and we’re going to discuss are clinical trials a gamble?  So how do you decide as a patient, you and your family?  We’ll talk about that in June.  Thank you so much for being with us.

Paying It Forward: Volunteering for Clinical Trials

Editor’s Note: This blog and video is from the Alliance for Aging Research. The Alliance for Aging Research is dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health.

Getting medical discoveries from the research lab to patients depends on clinical trials and the people who volunteer to participate in them.  Volunteering in a trial may help society at large by bringing new treatments one step closer to patients, and could help a loved one if you have a genetic disease or condition.  Volunteering may also give you access to a cutting-edge treatment and medical team that carefully monitors your health.  But clinical trials can’t happen without volunteers, and 37% of trials don’t enroll enough patients to move forward.  Clinical trials need volunteers like you so watch this short film to find out more about why they are important, how to get involved, and what it means to participate.

How to Read and Understand a Scientific Paper

In a previous article, How to Read Beyond the Headline: 9 Essential Questions to Evaluate Medical News, I recommended you should always try to read an original study (if cited) to evaluate the information presented. In this follow-on article, you will learn how to read a scientific research paper so that you can come to an informed opinion on the latest research in your field of interest.  Understanding research literature is an important skill for patient advocates, and as with any skill, it can be learned with practice and time.

Let’s start by looking at what exactly we mean by the term “scientific paper”. Scientific papers are written reports describing original research findings. They are published in peer reviewed journals, which means they have been refereed by at least two other experts (unpaid and anonymized) in the field of study in order to determine the article’s scientific validity.

You may also come across the following types of scientific papers in the course of your research.

•       Scientific review papers are also published in peer reviewed journals, but seek to synthesize and summarize the work of a particular sub-field, rather than report on new results.

•       Conference proceedings, which may be published in a journal, are referred to as the “Proceedings of Conference X”. They will sometimes go through peer review, but not always.

•       Editorials, commentaries and letters to the editor offer a review or critique of original articles. They are not peer-reviewed.

Most scientific journals follow the IMRD format, meaning its publications will usually consist of an Abstract followed by:

•       Introduction

•       Methods

•       Results

•       Discussion


Let’s look at each of these sections in turn.

(a) Introduction  

The Introduction should provide you with enough information to understand the article. It should establish the scientific significance of the study and demonstrate a relevant context for the current study.  The scope and objectives of the study should be clearly stated.

When reading the Introduction, ask yourself the following questions:

·       What specific problem does this research address?

·       Why is this study important?

(b) Methods

The Methods section outlines how the work was done to answer the study’s hypothesis. It should explain new methodology in detail and types of data recorded.

As you read this section, look for answers to the following questions:

  • What procedures were followed?
  • Are the treatments clearly described?
  • How many people did the research study include? In general, the larger a study the more you can trust its results. Small studies may miss important differences because they lack statistical power. Case studies (i.e. those based on single patients or single observations) are no longer regarded as scientific rigorous.
  • Did the study include a control group? A control group allows researchers to compare outcomes in those who receive a treatment with those who don’t.

 (c) Results

The Results section presents the study’s findings.  It should follow a logical sequence to answer the study hypothesis.  Pay careful attention to any data sets shown in graphs, tables, and diagrams. Try to interpret the data first before reading the captions and details.  If you are unfamiliar with statistics, you will find a helpful glossary of terms hereClick here for an online guide to help you understand key concepts of statistics and how these concepts relate to the scientific method and research.

Consider the following questions:

  • Are the findings supported by persuasive evidence?
  • Is there an alternative way to interpret these findings?

(d) Discussion 

The Discussion places the study in the context of the broader field of research. It should explain how the research has moved the body of scientific knowledge forward and outline the next steps for further study.

Questions to ask:

•       Does the study have any limitations? Limitations are the conditions or influences that cannot be controlled by the researcher.  Any limitations that might influence the results should be mentioned in the study’s findings.

  • How are the findings new or supportive of other work in the field?
  • What are some of the specific applications of the study’s findings?

The IMRD format provides you with a useful framework to read a scientific paper. You will need to read a paper several times to understand its findings. Consider your first reading of the study as a “big picture” reading.  Scan the Abstract for a summary of the study’s principal objectives, the methods it used and the principal conclusions. A well-written abstract should allow you to identify the basic content of an article to determine its relevance to you.  In describing how she determines the relevance of a study, research RN, Katy Hanlon, focuses on “key words and phrases first. Those that relate to the author/s base proposal as well as my own interests”.  Medical writer, Nora Cutcliffe, also scans upfront “to gauge power and relevance of clinical trial data”. She looks for “study enrollment (n), country and year”. It’s important to note the publication date to determine if this article contains the latest findings or if there is more up-to-date research available. Cutcliffe also advises you should “note author affiliations and study sponsors”.  Here you are looking out for any potential bias or vested interest in a particular outcome.  Check the Acknowledgments section to see if the author(s) declare any financial interests in the research which might bias their findings. Finally, check if the article is published in a credible journal.  You will find reputable biomedical journals indexed by Pubmed and Web of Science.

Next, circle or take note of any scientific terms or keywords you don’t understand and look up their meaning before your second reading. Scan the References section – you may even want to read an article listed here first to help you better understand the current study.

With the second reading you are going to deepen your comprehension of the study. You’ll want to highlight key points, consult the references, and take notes as you read.  According to the scientific publisher, Elsevier, “reading a scientific paper should not be done in a linear way (from beginning to end); instead, it should be done strategically and with a critical mindset, questioning your understanding and the findings.”  Scientist, Dr Jennifer Raff, agrees. “When I’m choosing papers to read, I decide what’s relevant to my interests based on a combination of the title and abstract”, she writes in How to read and understand a scientific paper: a guide for non-scientists. “But when I’ve got a collection of papers assembled for deep reading, I always read the abstract last”. Raff explains she does this “because abstracts contain a succinct summary of the entire paper, and I’m concerned about inadvertently becoming biased by the authors’ interpretation of the results”.

When you have read the article through several times, try to distill it down to its scientific essence, using your own words. Write down the key points you have gleaned from your reading such as the purpose of the study, main findings and conclusions. You might find it helpful to develop a template for recording notes, or adapt the template below for use. You will then have a useful resource to find the correct reference and to cross reference when you want to consult an article in the future.

In the example below I have taken an article published in 2015, as an example. You can read the paper Twitter Social Media is an Effective Tool for Breast Cancer Patient Education and Support: Patient-Reported Outcomes by Survey on PubMed.

Template for Taking Notes on Research Articles



Further reading

Clinical Studies and Research Opportunities For Patients

Essential Thrombocythemia (ET)


QuintilesIMS Global Services, a worldwide consulting firm, is conducting a research study to better understand the experience of ET patients. QuintilesIMS plans to interview patients who have been diagnosed with ET one‐on‐one over the phone. QuintilesIMS is looking for interested people to share what they have gone through since being diagnosed with ET.


The overall purpose of the study is to learn which symptoms of ET are the most common, and how these symptoms typically affect the lives of patients like you who live with the disease. This understanding may help improve how the patient’s experience is accounted for in clinical trials that measure the effect of disease treatments. It also may help patients and their families by highlighting the consequences of the disease that most need greater attention, possibly leading to the development of new programs and support services.

Your Involvement

If you qualify, you will be invited to participate in a 75‐minute telephone discussion with one of QuintilesIMS’ healthcare researchers. You will not need to do anything to prepare, other than being ready to describe what your life has been like while living with ET. At no point before, during, or after the interview will you be contacted to purchase anything or be asked to take any medication. You will be one of 20 patients participating in the study and your information will be kept confidential by removing any personal identifiers.

To qualify, patients must have the following criteria:

1. Patient is ≥18 years of age

2. Patient has a current diagnosis of Essential Thrombocythemia (ET) and fulfills one of the following:

· Patient has received hydroxyurea treatment in the past and discontinued it for any reason


· Patient could not receive hydroxyurea because it was contraindicated for the patient

3. Patient has some self‐reported disease related symptom burden

4. Patient is physically and mentally able to participate in an 75 minute interview in English to discuss signs, symptoms, and impacts related to ET

5. Patient is not currently participating in a research study where an investigational agent is being administered

6. Patient has no prior treatment with any oral JAK inhibitor


In return for your voluntary participation in the interview, your time and effort will be compensated with a $125 Visa gift card.

Next Steps

To participate, please call Michael Posey from QuintilesIMS, at +1 412‐973‐3162 (Monday‐Friday, 9:00am ‐ 5:00pm EDT), or write to Michael will ask for your consent to participate in the study, see if you qualify, then schedule your interview.

Essential Thrombocythemia (ET)

A Double-Blind, Double-Dummy Phase 2 Randomized Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.

Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

You can also visit and enter NCT03123588 to learn more.

Multiple Myeloma

Help Us Better Understand Multiple Myeloma Treatment Experiences

Pinpoint Patient Recruiting is seeking individuals with Multiple Myeloma to participate in a market research study about patients’ experiences with treatment decisions for Multiple Myeloma. The feedback will be used to help future patients and their caregivers.

If you are currently being treated for Multiple Myeloma and are receiving your second, third or fourth course of drug therapy (because previous courses of therapy either did not work, stopped working or you experienced a relapse), you may qualify to participate.

If you qualify and complete the online survey, you will receive an honorarium of $75 for your time and participation. All information and responses will remain confidential.

To see if you qualify or to get more information visit:

You may also contact Kim Slusher via email at


A Phase 2 Study of the Safety, Tolerability, and Efficacy of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis

The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of INCB050465 and ruxolitinib in subjects with myelofibrosis

Talk to your doctor if you are interested in participating in this study.

To find a study center near you, call , then select option 1.

You can also visit and enter NCT02718300 to learn more.

Understanding Itching and Night Sweats With MPN

From the Understanding Myeloproliferative Neoplasms (MPNs) Town Meeting, a panel of experts explains why MPN patients have to deal with itching and night sweats and what they can do to treat those side effects. The panel includes:

  • Olatoyosi Odenike, MD, Associate Professor of Medicine at The University of Chicago Medical Center
  • Julie Huynh-Lu, PA-C, Physician Assistant, Department of Leukemia at The University of Texas MD Anderson Cancer Center
  • Srdan Verstovsek, MD, PhD, Professor, Department of Leukemia, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center
  • Carmelita Escalante, MD, FACP, Professor and Chair, Department of General Internal Medicine at The University of Texas MD Anderson Cancer Center

Please check out the full video below to hear from the experts.

Understanding Itching and Night Sweats With MPN from Patient Empowerment Network on Vimeo.

How Do You Find Out About Clinical Trials?

Interview with Larry Anderson, Jr., MD, PhD, Assistant Professor, Department of Internal Medicine, Division of Hematology/Oncology University of Texas Southwestern Medical Center, Patient Advocate, Lynette, and Robert Orlowski, MD, PhD, Director of Myeloma and Professor in the Departments of Lymphoma/Myeloma and Experimental Therapeutics The University of Texas MD Anderson Cancer Center

From  the Virtual Town Meeting: Understanding the New World of Myeloma Treatment, Andrew Schorr first ask Dr. Anderson about how patients can find out about clinical trials, whether that be a governmental website, advocacy groups, or each institution’s individual website. Later he gets Lynette and Dr. Orlowski’s opinion on the matter. Check out the full video below to hear from three myeloma experts.

How Do You Find Out About Clinical Trials? from Patient Empowerment Network on Vimeo.

What Can Help Fight Fatigue With MPNs?

Interview with Carmelita P. Escalante, MD, FACP, Professor and Chair Department of General Internal Medicine The University of Texas MD Anderson Cancer Center

From the October 2016 MPN Town Meeting, the question, “Is there any drugs or maybe even vitamin B-12 that can be used to combat the fatigue associated with an MPN?” Check out the full video below to see how MPN expert, Dr. Carmelita Escalante, answers.

What Can Help Fight Fatigue With MPNs? from Patient Empowerment Network on Vimeo.


[toggle title=”Full Transcript Here” state=”closed”]


Jeff Folloder: I have a question for Dr. Escalante. Mark from the internet wants to know if there’s any drugs or maybe even vitamin B-12 that can be used to combat the fatigue associated with an MPN.

Dr. Carmelita Escalante: For fatigue – specifically fatigue we look at other conditions. So, we try to treat those conditions, whether they’re depression, etc. Specifically, for fatigue, we would not use B-12 unless your B-12 deficient. There’s not literature to say that B-12 supplements are helpful unless you’re low on B-12.

The drugs that we have used and there is mixed evidence, is stimulants. Stimulants are drugs like methylphenidate, which is Ritalin, which has been used in children with ADHD, attenuation deficit disorders. It’s probably the most studied of all the stimulants. We’ve also used provigil and nuvigil, which is modafinil and armodafinil. They are approved for sleep dysfunctions; such as sleep apnea.

There’s been a scattering of others that have been used, like Adderall. The data is mixed. There is probably more negative trials than positive. But, in severe fatigue, there is a small – there’s some data that shows improvement.

I specifically try to get the behavioral and exercise treatment going, but for some patients the stimulants can be very helpful. And we use them in fairly small doses. There’s very negligible side effects. So, I think it’s a win if I use it and the patient feels better and can do more.

There is a big placebo effect. So, when we do trials with it we always have to have a placebo on. But, the bottom line is if it’s helpful and the patient is tolerating it and doing the other things that we’ve prescribed to try to improve the fatigue and we have everything controlled that we can, and it helps, I think it’s a good thing. Especially, I’ve noticed if there are cognitive deficits, which is – which methylphenidate has been used for in kids to try to get them to focus, it can be very helpful.

We use it a lot in our brain tumor patients. I’ve used it especially inpatients that have jobs where they really have to focus and they tell me I just can’t do my work because I can’t keep focus, and it can be extremely helpful for them.

Andrew Schorr: Okay, what about the obvious one? I’m drinking a lot of coffee. Coffee’s okay?

Dr. Carmelita Escalante: Coffee’s okay. It’s a fairly week stimulant. I think if you have very mild fatigue and it helps you. I’d be careful in those energy drinks because sometimes, especially if you’re on other medications or if you have heart conditions, you need to be very careful about drinking several a day – may not be in your best interest, especially if you feel your heart racing or you feel jittery.



Nothing About Us Without Us: Patient Involvement in Research

Until recently, patient participation in research was limited to their involvement as subjects enrolled in research studies, but there is a shift occurring as funding bodies increasingly look for evidence of patient and public involvement (PPI) in research proposals. The rationale for this is increasing evidence that PPI in the provision of healthcare leads to improved outcomes and better quality of care.

Assumptions are made every day about patients; assumptions which may lead to a failure to deliver optimum care. When these assumptions extend to research, quite often there is a mismatch between the questions that patients want answers to and the ones that researchers are investigating. As an example, the research priorities of patients with osteoarthritis of the knee, and the clinicians looking after them, were shown in a study to favor more rigorous evaluation of physiotherapy and surgery, and assessment of educational and coping strategies. Only 9% of patients wanted more research on drugs, yet over 80% of randomized controlled trials in patients with osteoarthritis of the knee were drug evaluations. PPI recognizes that patients bring a unique perspective and experience to the decision-making process in research. It is paternalistic and patronizing to rely on speculation about patient experience. By considering the actual experience of patients, researchers can make more informed research decisions. Involving patients is an important step in ensuring that the real life experiences of patients are considered when it comes to setting research priorities. This in turn will increase the relevance of research to patients and improve research quality and outcomes.

As an advocate you may be asked to become involved in a research project, so it is important to have a clear understanding of what PPI is – and what it isn’t. PPI is not about being recruited as a participant in a clinical trial or other research project, donating sample material for research, answering questionnaires or providing opinions. PPI describes a variety of ways that researchers engage with people for whom their research holds relevance. It spans a spectrum of involvement which may include any of the following:

  • Being involved in defining the research question
  • Being a co-applicant in a research proposal
  • Working with funders to review patient-focused section of applications
  • Being an active member of a steering group for a research study
  • Providing your input into a study’s conception and design
  • Contributing to/proofing of documentation
  • Assisting in the implementation and dissemination of research outcomes
  • Improving access to patients via peer networks and accessing difficult-to-reach patients and groups

Effective PPI transforms the traditional research hierarchy in which studies are done to, on, or for participants into a partnership model in which research is carried out with or by patients.  PPI should always involve meaningful patient participation and avoid tokenism. The Canadian Institutes of Health Research Strategy for Patient-Oriented Research (SPOR) describes PPI as fostering a climate in which researchers, health care providers, decision-makers and policy-makers understand the value of patient involvement and patients see the value of these interactions. Underpinning this framework are the following guiding principles for integrating patient engagement into research:

  • Inclusiveness:Patient engagement in research integrates a diversity of patient perspectives and research is reflective of their contribution.
  • Support:Adequate support and flexibility are provided to patient participants to ensure that they can contribute fully to discussions and decisions. This implies creating safe environments that promote honest interactions, cultural competence, training, and education. Support also implies financial compensation for their involvement.
  • Mutual Respect:Researchers, practitioners and patients acknowledge and value each other’s expertise and experiential knowledge.
  • Co-Build:Patients, researchers and practitioners work together from the beginning to identify problems and gaps, set priorities for research and work together to produce and implement solutions.

Derek Stewart, a patient advocate and Associate Director for Patient and Public Involvement at NIHR Clinical Research Network, sees a growing momentum of actively involving patients and public in research gathering pace worldwide. “It is really pleasing to hear researchers saying how valuable it has been to involve patients and the public in their work”, he says. “It has equally improved the quality of the research and enriched their own thinking and understanding.”

Earlier this year, PCORnet, the National Patient-Centered Clinical Research Network, announced its first demonstration study which reflects PCORnet’s aims of patient engagement and open science. ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-Term Effectiveness) will compare the effect of two different aspirin doses given to prevent heart attacks and strokes in high-risk patients with a history of heart disease. Seeking input at every critical step, from consent design and protocol development, through dissemination of final study results, the project represents a new research paradigm. Unprecedented in the design of clinical trials, the final consent form and protocol were shaped with input from patients, local institutional review boards, physicians, and study coordinators.

Another noteworthy example of PPI can be found in the Metastatic Breast Cancer Project a direct-to-patients initiative launched at the Broad Institute of MIT and Harvard last October. Corrie Painter, an angiosarcoma patient and Associate Director of Operations and Scientific Outreach at Broad Institute, explains that “the project seeks to greatly accelerate the pace of biomedical research by empowering patients to directly contribute to research and was built in lock step from design to consent language with dozens of patients.”

To what extent you may wish to be involved in PPI will depend on several factors. Do you have professional experience (e.g. project management, clinical experience, etc.) which would be useful? Are you happy to work as part of a team? Or would you prefer to work on your own? You should also take into consideration your other work or family commitments. For instance will you need to take time off work to attend meetings? Consider also at what point you are in your own health journey. Will participation in research place an added burden on your treatment or recovery? In making the decision to become involved in research, you should always balance your own health needs with the desire to be supportive of research and the research process.

Useful links



Metastatic Breast Cancer Project


Who Is Eligible and How Can I Learn More About Clinical Trials?

From the Lung Cancer Town Meeting in September 2016, Janet Freeman-Daily interviews a panel of lung cancer experts about who is eligible for clinical trials and how you can learn more about them. The panel includes the following experts:

  • Nisha Monhindra, MD Assistant Professor of Medicine, Hematology/Oncology Division, Feinberg School of Medicine Robert H. Lurie Comprehensive Cancer Center of Northwestern University
  • D. Ross Camidge, MD, PhD, Director Thoracic Oncology Clinical and Clinical Research Programs University of Colorado Denver
  • David D. Odell, MD, MMSc, Assistant Professor, Thoracic Surgery Robert H. Lurie Comprehensive Cancer Center of Northwestern University
  • Timothy J. Kruser, MD, Assistant Professor, Radiation Oncology Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Check out the full video below to hear all of the lung cancer experts advice.

Who is Eligible and How Can I Learn More About Clinical Trials? from Patient Empowerment Network on Vimeo.

How to Read Beyond the Headline: 9 Essential Questions to Evaluate Medical News

Ben Goldacre writing in Bad Science classified science reporting as falling into three categories – wacky stories, scare stories and breakthrough stories; the last of which he views as ”a more subtly destructive category of science story”. Whether you get your news through digital or traditional means, you can’t fail to notice the regularity with which journalists report on the latest medical breakthroughs. Some of these reports are sensationalist (“coffee causes cancer”) and fairly easy to dismiss; but do you know how to separate fact from fiction when it comes to less sensationalist headlines?

The foundation of empowered patient-hood is built on reliable health information. This means not only knowing where to find medical information, but being able to evaluate it and knowing how it can be applied to your own, or your loved-ones’ particular circumstances. Headlines often mislead people into thinking a certain substance or activity will prevent or cure chronic disease. As patient advocates we must learn to read beyond the headlines to filter out the good, the bad, and the questionable. The following questions are designed to help sort the signal from the noise next time you read the latest news story heralding a medical breakthrough.

1. Does the article support its claims with scientific research?

Your first concern should be the research behind the news article. If an article contains no link to scientific research to support its claims, then be very wary about treating those claims as scientifically credible.

2. What is the original source of the article?

If the article cites scientific research you should still treat the findings with caution. Always consider the source. Find out where the study was done. Who paid for and conducted the study? Is there a potential conflict of interest?

3. Does the article contain expert commentary to back up claims?

Look for expert independent commentary from doctors or other healthcare providers to explain the findings (there should be an independent expert source quoted – someone not directly connected with the research).

4. Is this a conference presentation?

Journalists frequently report on research presented at large scientific meetings. It’s important to realize that this research may only be at a preliminary stage and may not fulfill its early promise.

5. What kind of clinical trial is being reported on?

If the news relates to results from a clinical trial, it’s important you understand how, or even if, the results apply to you. Quite often, news publications report on trials which have not yet been conducted on humans. Many drugs that show promising results in animals don’t work in humans. Cancer.Net and American Cancer Society have useful guides to understanding the format of cancer research studies.

6. What stage is the trial at?

Research studies must go through several phases before a treatment can be considered safe and effective; but many times journalists report on early phase trials as if these hold all the answers. The testing process in humans is divided into several phases:

  •  Phase I trials: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
  • Phase II trials: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
  • Phase III trials: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.


7. How many people did the research study include?

In general, the larger a study the more you can trust its results. Small studies may miss important differences because they lack statistical power.

8. Did the study include a control group?

A control group allows researchers to compare outcomes in those who receive a treatment with those who don’t. The gold standard is a “randomised controlled trial”, a study in which participants are randomly allocated to receive (or not receive) a particular intervention (e.g. a treatment or a placebo).

9. What are the study’s limitations?

Many news stories fail to point out the limitations of the evidence. The limitations of a study are the shortcomings, conditions or influences that cannot be controlled by the researcher. Any limitations that might influence the results should be mentioned in the study’s findings, so always read the original study where possible.

Useful Resources

  • Gary Schweitzer’s Health News Review website provides many useful resources to help you determine the trustworthiness of medical news. To date, it has reviewed more than 1,000 news stories concerning claims made for treatments, tests, products and procedures.
  • Sense about Science works with scientists and members of the public to equip people to make sense of science and evidence. It responds to hundreds of requests for independent advice and questions on scientific evidence each year.
  • Trust It or Trash is a tool to help you think critically about the quality of health information (including websites, handouts, booklets, etc.).
  • Understanding Health Research (UHR) is a free service created with the intention of helping people better understand health research in context. It gives clear and understandable explanations of important considerations like sampling, bias, uncertainty and replicability.

MyLifeLine: Learn About Clinical Trials

Editor’s Note: This post was originally published here on The mission of is to empower cancer patients and caregivers to build an online support community of family and friends to foster connection, inspiration, and healing through free, personalized websites.

Learn About Clinical Trials


Why consider a cancer clinical trial?

What clinical trials can offer, from the care you receive to the impact you can make.

Clinical trials offer a chance to receive investigational medicines or procedures that experts think might improve the treatment of cancer. This important option is not limited to people who have run out of choices. In fact, there may be clinical trials for every stage of disease in dozens of cancer types. In this video, patients and doctors share their perspectives on why joining a clinical trial may be an option worth considering.

“To have the opportunity to go on a clinical trial for a patient is extremely exciting.” —Sandra Swain, MD; oncologist

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I don’t want to be a guinea pig for an experimental treatment.
The Truth:
Cancer clinical trials are developed with high medical and ethical standards, and participants are treated with care and with respect for their rights.

I’m afraid i might receive a sugar pill or no treatment at all.
 The Truth:Cancer clinical trials rarely use placebo alone if an effective treatment is available; doing so is unethical.

Cancer clinical trials are only for people with no other treatment options.
 The Truth:Trials can study everything from prevention to early- and late-stage treatment, and they may be an option at any point after your diagnosis.

I’m worried that I won’t receive quality care in a cancer clinical trial.
 The Truth:Many procedures are in place to help you receive quality care in a cancer clinical trial.

People might access private information about me if I participate.
 The Truth:In nearly all cancer clinical trials, patients are identified by codes so that their privacy is protected throughout and after the study.

I’m afraid that my health insurance will not help with the costs of a cancer clinical trial.
 The Truth:
Many costs are covered by insurance companies and the study sponsor, and financial support is often available to help with other expenses; talk to your doctor to understand what costs you could be responsible for.

Informed consent only protects researchers and doctors, not patients.
 The Truth:
Informed consent is a full explanation of the trial that includes a statement that the study involves research and is voluntary, and explanations of the possible risks, the possible benefits, how your medical information may be used, and more. Informed consent does not require you to give up your right to protection if the medical team is negligent or does something wrong.

I’m afraid that once i join a cancer clinical trial, there’s no way out.
 The Truth:
You have the right to refuse treatment in a cancer clinical trial or to stop treatment at any time without penalty

How to know if a cancer clinical trial is right for you.

There are many factors to keep in mind when considering a cancer clinical trial.

As with any important decision, it’s a good idea to think about the risks and benefits of joining a cancer clinical trial. This video encourages you to ask your medical team about all of your treatment options, including cancer clinical trials. Trial participants, doctors, and patient advocates explain the factors you’ll want to keep in mind as you consider your treatment plan.

“I’ve always advised patients…when the circumstances weren’t urgent, to take time to understand their disease and to evaluate the alternatives.”  —Sandra Horning, MD; oncologist and chief medical officer

What to ask your doctor(s)

Asking The Right Questions Keeps You Involved In Your Care

A cancer diagnosis is often overwhelming, and it’s sometimes hard to gather your thoughts and know the right questions to ask. This video talks you through some of the questions it will be helpful to ask about your cancer, your treatment options, your doctor, and about whether participating in a cancer clinical trial is right for you.

“Talk to your doctor and say, ‘Tell me my full options.’ Raise questions. Be a pain in the neck. That’s what the doctor is there for.” —Arthur Caplan, PhD; medical ethicist

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Rules And Procedures Are In Place So That You Will Receive High-Quality Care

Before a single patient can join a trial, many different experts must approve every detail of the study—from why it’s being done to how often patients should be monitored. Once the trial begins, more unbiased experts provide oversight to check that the rules of the trial are being followed and patients’ rights are protected. This video features doctors and patient rights advocates explaining the high standards by which trials are developed and run.

“I explain…that when they’re on a clinical trial, they’re going to be followed very closely by…specific guidelines.” —Daniel P. McKellar, MD; surgeon and Commission on Cancer chairman

Informed Consent Describes The Study Process, Potential Risks And Benefits, And Your Rights As A Participant

If you are eligible and decide to join a trial, you will be required to review and sign the informed consent forms. This can be an overwhelming process, but it is how you will learn all the details of the trial, including the potential benefits and the possible risks, and give your permission to be treated. This video features patients, doctors, and patient rights advocates who offer tips and insights to help you navigate the process of informed consent.

“When I received the stack of papers…it made me realize this is really serious. But then…it was actually a good feeling to know that this was not something that was being done lightly.” —Rose Gerber; trial participant

Information And Support Are Close At Hand

Because so many people have been affected by cancer, there are many reliable and helpful resources to help you through your cancer journey. In this video, trial participants and doctors help you find the people and resources that may be helpful in educating you about cancer clinical trials.

“The first thing is to hold on tight and be optimistic and to get very engaged and educated about your cancer.” —Jack Whelan; trial participant

Reliable Resources To Help Along The Way

First, talk to your doctor

Your healthcare team is the best source for information about your treatment options, including cancer clinical trials. There are many questions you’ll want to ask your healthcare team when you’re ready to discuss treatment options. Print this helpful Discussion Guide and bring it to your next appointment so that you don’t forget anything important. Record your answers on the form and keep it handy for future reference.

Where to find information about cancer clinical trials

These clinical trial resources will help you find trials that might be right for you.

Support services

These trustworthy sources provide assistance with trial-related costs, which may not always be covered by insurance.

Practical support

Financial support

Additional nationwide support organizations

Don’t go it alone

There are millions of people just like you who are ready to ACT against cancer. These organizations provide advocacy, information, awareness, fundraising opportunities, and a community of like-minded people touched by cancer.

Heading Off Cancer Growth on the Cellular Level

Cancer cells are like all the cells in our body, in that they need certain basic building blocks – amino acids – in order to reproduce. There are 20 amino acids found in nature. The amino acid serine is often found in abundance in patients with certain types of breast cancer, lung cancer, and melanoma. The overproduction of this amino acid is often required for the rapid and unregulated growth characteristic of cancer.

Scientists at the Scripps Research Institute (TSRI) wondered if there was a way to take advantage of the relationship between cancer cell proliferation and serine. Amy GrayThey examined a large library of molecules -numbering 800,000 – to find an enzyme that inhibited serine production. After much research, the group found 408 contenders that could possibly work. This list was again narrowed down to a smaller set of seven, ending with one promising candidate. This molecule, 3-phosphoglycerate dehydrogenase (PHGDH), seemed to inhibit the first step in a cancer cell’s use of serine to reproduce itself.

Luke L. Lairson, assistant professor of chemistry at TSRI and principal investigator of cell biology at the California Institute for Biomedical Research remarked, “In addition to discovering an inhibitor that targets cancer metabolism, we also now have a tool to help answer interesting questions about serine metabolism.”

What does this mean for cancer patients in the future?

Discovering an enzyme that inhibits serine production means that a key process in cancer cell proliferation can be slowed down or even stopped.   Interfering with cancer cell metabolism could be a pathway to treatment. Potentially, adding the molecule PHGDH to cancer cells disturbs the basic need of cancer cells to divide and reproduce rapidly. Obviously this finding points to years of further research and drug development. But discovering this key relationship between serine over-production and a molecule that slows it down could be a model for new cancer treatments in the future.