Tag Archive for: ruxolitinib

Should All MPN Patients Undergo Molecular Testing?

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Should All MPN Patients Undergo Molecular Testing? from Patient Empowerment Network on Vimeo.

Dr. Gabriela Hobbs discusses the necessity of molecular testing for myeloproliferative neoplasm (MPN) patients, including the pros and cons of this in-depth testing for patients with polycythemia vera (PV) and essential thrombocythemia (ET).

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

How useful is having a genetic panel done? Should all patients get molecular or genetic testing? 

Dr. Hobbs:

Great question. And I think that it is very important to have genetic testing.   

And genetic testing involves more than just testing the JAK2 mutation. So, we know that the JAK2 mutation is the most common mutation in patients with MPN. But that being said, there are other mutations that also occur such as the calreticulin mutation and the MPL mutation.   

And so, I think having genetic testing that at least tests for those three mutations is very important so that we can actually help a patient know that they have an MPN. In addition to those three main mutations, many clinicians now have access to what’s called extended next-generation sequencing, where there’s a panel that tests for many different genes at the same time and can test for a variety of other mutations.  

And this is particularly relevant for patients with myelofibrosis. As we know that having other mutations, like, for example, mutations in IDH or ASXL1 and others, can increase the risk of that disease in terms of its risk of transforming to leukemia or how long a patient may live with their myelofibrosis. 

And so, I do recommend having extended next-generation sequencing done at least at diagnosis.  

When I generally think about repeating that, if there’s something that looks like it’s changing within the patient’s disease, to be honest, also on the flipside of that argument, sometimes this next-generation sequencing will mostly contribute to adding anxiety and will not necessarily directly impact how a patient is treated. And this is particularly true in patients with PV and ET, where we’ll sometimes order these tests, and we get a bunch of mutations back, but we don’t know what to do with that information yet.  

And so, as a researcher – not a clinician – as a researcher, I think it’s very important to have that information so that we can then do studies and understand the patterns of mutations and how that affects outcome. But as a clinician, and you as a patient, you need to really be aware of how that’s going to impact the patient in front of you and how that may impact you as a patient. Do you want to know if you have these mutations if nothing can be done about it? So, I would say, take a moment to reflect upon what I said and also to ask your clinician, how is this information going to help me? Do I need to have this information?  

Maybe you want to have it done so that it’s in your record. But maybe you don’t necessarily want to know those results. And everybody’s very different. And I think it’s absolutely wonderful to talk to my patients about all the information. But there may be some patients that really are just, like, do the test but don’t tell me the results, because I know that I’m just going to be very anxious knowing that I have something that I can’t do anything about. So, just take a minute to talk about it with your doctors. I think that’s really important.  

What Are the Long-Term Effects of JAK Inhibitors?

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What Are the Long-Term Effects of JAK Inhibitors? from Patient Empowerment Network on Vimeo.

MPN expert Dr. Gabriela Hobbs discusses what researchers know about the long-term safety of JAK inhibitors and options for patients if the treatment loses effectiveness over time.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Should All MPN Patients Undergo Molecular Testing

Should All MPN Patients Undergo Molecular Testing?


Transcript:

Katherine:

What are the long-term effects of JAK inhibitors? And what happens when JAK inhibitors are no longer effective? 

Dr. Hobbs:

Yeah. Great question. So, so far the patients that have been on JAK inhibitors for a long time don’t seem to have the development of additional toxicities that we didn’t know about.  

So, I’ll just comment on some of the things that we do know about. Weight gain is a common complaint that I have from patients, especially those that have polycythemia vera, because maybe they didn’t want to gain weight when they were put on a JAK inhibitor compared to the myelofibrosis patients who maybe had lost a lot of weight before being on a JAK inhibitor.  

There are certainly higher risk probably of developing infections with some of the JAK inhibitors. And we see, for example, shingles reactivation being a common one. And there’s the concern of development of skin cancers, which has been seen with some JAK inhibitors. But generally speaking, long-term use seems to be safe. That being said, ruxolitinib (Jakafi), which is the oldest one to be approved, has only been around since 2011, so we don’t have decades worth of experience to know.  

When JAK inhibitors stop working – to answer the second part of your question – until fairly recently we really didn’t have a whole lot to offer because there was only one JAK inhibitor. Now we have two others. We have fedratinib (Inrebic) and also pacritinib (Vonjo). And we know from the studies that have been done with both of these agents that some patients that lose response to Jakafi, meaning that their spleen starts to grow or their symptoms start to come back, can be treated with these other JAK inhibitors.  

And many patients will, again, have control of their spleen and symptoms. Now losing response to a JAK inhibitor can come in many different ways. And so, some patients may also develop signs of having leukemia or progression of their disease to leukemia. And, unfortunately, for those patients, being on another JAK inhibitor doesn’t make sense. So, those patients may need to receive other types of medications or a stem cell transplant. 

Advances in Myelofibrosis Research

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Advances in Myelofibrosis Research from Patient Empowerment Network on Vimeo.

What are the recent developments in the study and advancement of myelofibrosis treatment? MPN researcher Dr. Gabriela Hobbs discusses ongoing clinical trials for new JAK inhibitors, BET inhibitors, and anemia therapies, among others.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

What about myelofibrosis, Dr. Hobbs? What advances are being made in the care of patients with this more advanced MPN? 

Dr. Hobbs:

Yeah. So, in myelofibrosis, I would say it is almost difficult to keep track of how many clinical trials are currently open. So, in 2011, we had ruxolitinib approved, or Jakafi. That was the first JAK inhibitor. Since then, we’ve had two more JAK inhibitors approved, fedratinib (Inrebic) and most recently pacritinib (Vonjo). And we’re currently awaiting the fourth JAK inhibitor to be approved, and that’s called momelotinib.   

And in addition to the JAK inhibitors, there are lots of other clinical trials underway right now that are either alone – a drug by itself or a drug in combination with ruxolitinib.  

So, there are several Phase III studies. And the reason why that’s important is that after Phase III we usually see a drug approval. So, we can expect, hopefully in the next couple of years, to see many more drugs available on the market to treat patients with myelofibrosis. Some of those include agents that block different pathways within a cell. And that includes a drug called parsaclisib. There’s a drug called pelabresib (CPI-0610), which is a BET inhibitor.  

There’s another drug called navitoclax (ABT-263), which is a cousin of venetoclax (Venclexta), which is a drug that we’ve been using a lot in leukemia. So, there’s lots of different drugs that are being used in combination with ruxolitinib. There’s also a drug called luspatercept (Reblozyl) that’s also been approved for myelodysplastic syndromes. And I suspect that that’ll be approved as well to help patients with anemia. So, really, there’s lots of drugs that are being studied right now. And I think the question that we’re all asking is, well, how are we going to use all of these different drugs? So, I look forward to seeing the results of those studies.  

Katherine:

Mm-hmm. Will some drugs work better for some patients and others not? 

Dr. Hobbs:

That is such a good question. And so, what I’m hoping to see is exactly that. I’m hoping to see that for patients, for example, with anemia, perhaps we’re going to be using luspatercept and momelotinib. Perhaps we’re going to see that patients with certain mutations may respond better to certain medications like the BET inhibitors or navitoclax or the PI3 kinase inhibitor, parsaclisib. But as of now, we don’t have enough information.  

We haven’t seen enough results of these studies to start to be able to know, you know, what is the patient that’s going to do better with two drugs versus one drug? And so, I think that over the next couple of years we’re going to start to have answers to those questions.  

Advances in Polycythemia Vera Research

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Advances in Polycythemia Vera Research from Patient Empowerment Network on Vimeo.

What are the recent developments in the study and advancement of care for patients with polycythemia vera (PV)? Dr. Gabriela Hobbs reviews recently approved PV treatments as well as those currently in clinical trials.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

There was recently an interferon approved for use in patients with PV. What other studies are showing promise for patients with PV?   

Dr. Hobbs:

Yeah. So, we as a community, there’s been a lot of excitement about this new interferon that was approved, the ropeginterferon (Besremi) study. And there are still some ongoing studies utilizing ropeginterferon to see if we can use it differently.  

Because currently the way that that drug is approved is that it has to be titrated up very slowly to get to the maximum dose. So, that’s something that is still ongoing. In addition, there’s a new drug that’s being studied called Rusfertide (PTG-300) from a company called Protagonist. And this drug has been very interesting. It acts through iron metabolism.  

And so far in preliminary results, it has shown that a lot of the participants that receive this medication no longer need phlebotomy. And I think what’s exciting about this is that phlebotomy is a very archaic way of treating patients.  

And I hope that we can stop utilizing it. So, it’s nice to have a compound that’s specifically asking that question. And the other thing to keep in mind is that this drug has been used in combination with other drugs, which is really reflective of how participants or patients show up to clinics.  

Some patients are not going to be on any medications. Some patients may be on hydroxyurea (Hydrea).  

Some patients may be on an interferon. Some patients may be on ruxolitinib (Jakafi). And these trials allow participants to be on a variety of different medications. So, that’s an exciting new compound. 

Advances in Essential Thrombocythemia Research

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Advances in Essential Thrombocythemia Research from Patient Empowerment Network on Vimeo.

Are there new treatment developments for patients with essential thrombocythemia (ET)? Dr. Gabriela Hobbs shares an update on ET therapies in clinical trials and discusses when it might be appropriate for a patient to join a clinical trial.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

Let’s talk about ET for a moment. Is there any research being done to help better manage this condition? 

Dr. Hobbs:

Yeah. I would say that of the three MPNs, ET is certainly the one that has the least amount of drugs that are being currently studied for this group. But that doesn’t mean that there isn’t any research. Ropeginterferon (Besremi), which was recently approved in polycythemia vera, is now being studied in essential thrombocythemia.  

So, I would expect in the next couple of years, if those trials are successful, to have ropeginterferon as a therapy to offer patients.  

There is also a clinical trial that we have at our site. We’re using ruxolitinib or Jakafi for patients with ET that have symptoms of their disease to see if it can help them in the same way that it can help PV or myelofibrosis patients. So, there’s definitely some research going on in ET. But probably less than for PV and myelofibrosis.  

Katherine:

Mm-hmm. While ET is typically well-managed, what patient type might benefit from joining a trial? 

Dr. Hobbs:

It really depends on what the patient is experiencing. I think there are some patients that really are very asymptomatic and can expect to have an excellent outcome with their disease. But they can also participate in research, for example, by participating in a tissue bank and offering a sample of their blood or if they have a bone marrow by offering some bone marrow if there’s extra.  

Because that can really help to understand the disease biology, if a patient is going to progress from ET to myelofibrosis.  

So, we can learn a lot from that. But then there are maybe some ET patients that need to be on a medication to reduce their blood counts or a cytoreductive agent.  

And that’s a patient that could ask about participation in a clinical trial. For example, the ropeginterferon study or, like I mentioned, there may be some patients that maybe are already on a medication, and their blood counts aren’t well-controlled on the first drug that was used. 

So, before considering switching to a second-line agent or a second medication, that could inquire with their clinician if there’s a clinical trial available for second-line use. Or those patients that have a lot of symptoms with ET, they could potentially be eligible for a study that addresses just symptoms.  

How Driver Mutation Research Is Advancing MPN Treatments

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How Driver Mutation Research Is Advancing MPN Treatments from Patient Empowerment Network on Vimeo.

How do driver mutations affect MPN care? MPN researcher Dr. Gabriela Hobbs shares an update on what’s being learned about the JAK mutation and how researchers are working towards targeted therapy for MPNs.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

There have been huge developments in the last 10 to 15 years in the field of MPN. So, I’d like to dig a little deeper. We hear about the common driver mutations in MPNs like JAK2, CALR, and MPL. How are these being studied , and what is being discovered?  

Dr. Hobbs:

Yeah. So, it’s amazing how in the last 15 years really so much has been discovered. You know. The JAK2 mutation was first published out in 2005 and calreticulin in 2013. So, those are relatively recent discoveries. And I think a lot of efforts has been put into learning about what these mutations are doing and how they lead to disease. And so, we have the JAK inhibitors, which block the signaling through a pathway called JAK-STAT. And all of these mutations will activate that pathway within cells.  

And so, many of the approved drugs, for example, ruxolitinib (Jakafi), fedratinib (Inrebic), and pacritinib (Vonjo), work on blocking that pathway.  

But since then, we’ve also learned that there are other mutations and other pathways that are likely involved in the development of myeloproliferative neoplasms and also their progression. And so, what we’re seeing now is that many of the clinical trials that are being conducted don’t just target the JAK-STAT pathway or the pathway that’s influenced by these main mutations.  

But also block other pathways to try to really block all the variant expression of signaling in the myeloproliferative neoplasms. And so, we’re trying to attack it by many different angles.  

Katherine:

Yeah. Is there a possibility of specific targeted therapies at MPNs similar to those in AML such as FLT3 inhibitors? 

Dr. Hobbs:

Absolutely. So, similarly to AML, we know that we have mutations in similar types of genes called tyrosine kinases. So, these are enzymes that are turned on and always active. And so, I think there is definitely hope that we can develop some targeted agents. For example, ruxolitinib or the other JAK inhibitors are similar. They’re tyrosine kinase inhibitors where they block an enzyme, specifically the JAK2 enzyme.  

But I think that we can definitely do better and develop more specific inhibitors, for example, a molecule that just blocks the JAK2 mutation and not just every JAK2 molecule in every cell. Similarly to AML, there are mutations, for example, in enzymes called IDH.  

And we have IDH inhibitors for AML. And there are some studies that are using IDH inhibitors for MPN. So, I think we’re going to continue to see more targeted therapies specific to the mutations that occur in MPN. 

How MPN Researchers Collaborate to Advance Patient Care

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How MPN Researchers Collaborate to Advance Patient Care from Patient Empowerment Network on Vimeo.

MPN specialist and researcher Dr. Gabriela Hobbs discusses how collaboration and data sharing among researchers around the world impact MPN treatment advances.

Dr. Gabriela Hobbs is a hematology-oncology physician specializing in the care of patients with myeloproliferative neoplasms (MPN), chronic myeloid leukemia, and leukemia. Dr. Hobbs serves as clinical director of the adult leukemia service at Massachusetts General Hospital. Learn more about Dr. Hobbs.

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Transcript:

Katherine:

I’d like to start by discussing your role as an MPN researcher. You’re on the front lines for advancements in the field. What led you to there, and why is it so important to you?  

Dr. Hobbs:

Many things in my life led me to becoming an MPN clinician. First, I wanted to be a clinical investigator since I was very little, and I read a Louis Pasteur book about – you know. And I was fascinated by the fact that you could be both a scientist and a clinician. And after that, I had phenomenal teachers and mentors. And I was really always drawn to patients with hematologic malignancies. I thought that that interaction was very intense and intimate.  

And I was honored to be a part of that interaction. And then from a research perspective and from a scientific perspective, I very clearly remember seeing when the first targeted therapy, Imatinib, was approved when I was an undergrad. And I just thought that was the most fascinating thing. And so, I’ve basically continued to feel that way as I’ve gone through my training, and I’m thrilled to be able to have actually become an MPN clinician so many years later.   

Katherine:

With the American Society of Hematology or ASH meeting taking place this month, it demonstrates how researchers work together around the world to advance care.  

Can you share with the audience how this collaboration works?  

Dr. Hobbs:

Yeah. So, the American Society of Hematology meeting – or the ASH meeting – is really one of my favorite events of the year.  

And it really highlights what you said. It is such a positive environment, and it’s so exciting to use that opportunity to talk to my collaborators from across the globe. And I really think that that’s where the scientific community shines because really all of us are actually trying to figure out how to work together and overcome sometimes a lot of obstacles – bureaucratic obstacles, regulatory obstacles – to make sure that we can share data, do it the right way. But really we always have one thing in mind.  

And that is to be able to advance the care that we give our patients. And so, that collaboration and really that collaborative environment is always very positive. And I always come back home very energized from that. And then just seeing all my colleagues presenting all the wonderful things that they are working on and getting updates on their research is just an exciting environment.   

 Katherine:

In your view, why is it essential to present and share data at these larger conferences like ASH? 

Dr. Hobbs:

So, for many different reasons. I mean, there are many different ways of presenting data that can be done through just publishing a paper. But the nice thing about conferences – and especially large conferences – is that you really get an opportunity to present work in progress. And some of these research projects may not end up turning into bigger projects or they may not become bigger trials. But all of them have at least an opportunity to learn something from them, whether or not they worked or they didn’t work.   

Oftentimes when things are published in journals, especially the high-impact journals, we are seeing trials that had positive results. But sometimes we don’t see those smaller trials that never went anywhere. And so, having a forum when we can discuss work that’s ongoing, discuss about projects that are maybe having issues, all those things actually really help us to change our research questions or develop new research questions based on what’s working and also really what’s not working. And so, having this large forum to present all of that data, I think, is really, really important to helping us design future clinical trials and projects. 

How Clinical Trials Advance MPN Treatment and Research

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How Clinical Trials Advance MPN Treatment and Research from Patient Empowerment Network on Vimeo.

MPN expert Dr. Angela Fleischman provides a deeper understanding of how clinical trials advance myeloproliferative neoplasm (MPN) research and treatment, explains safety protocols in place for trials, and addresses common misconceptions associated with clinical trial participation. Dr. Fleischman also shares an update on emerging MPN research.

Dr. Angela Fleischman is a physician scientist and assistant professor in the Department of Medicine at the University of California, Irvine. Learn more about Dr. Fleischman.

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Transcript:

Katherine:

Hello, and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to discuss how clinical trials advance research for myeloproliferative neoplasms, or MPNs, and we’ll talk about what MPN patients should know about participation. 

Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. 

Well, let’s meet our guest today. Joining me is Dr. Angela Fleischman. Dr. Fleischman, welcome. Would you please introduce yourself? 

Dr. Fleischman:

Thank you very much for the invitation. Hi, everyone. My name is Angela Fleischman. I’m what’s called a physician scientist, meaning, I do research as well as see patients, and my focus for my entire career thus far has been on myeloproliferative neoplasms, specifically their role of inflammation in MPN. And I am at the University of California, Irvine in Southern California. So, nice to be here today. 

Katherine:

Well, thank you so much for joining us and taking the time. Before we get into the discussion about clinical trials, because you’re so heavily involved in research, let’s talk about the latest developments in the field. What MPN clinical trials are you excited about right now? 

Dr. Fleischman:

So, I would say, there’s a lot of new clinical trials in the field for myelofibrosis, which is the most severe form of myeloproliferative neoplasm. 

There tend to be more clinical trials because that’s a patient population in – I don’t want to say in more need, but they do have more need in terms of necessitating better treatments. 

Drugs that are quite far along in clinical trials – and in order for a drug to make it to market, one needs to go through multiple clinical trials to demonstrate the safety, as well as efficacy. Things like a BET inhibitor are very, very promising in moving forward in clinical trials. Other medications for other diseases, such as polycythemia vera, not anymore in clinical trials, but excitingly, newly FDA-approved, was ropeginterferon for polycythemia vera. 

So, that’s a real exciting development for Polycythemia Vera patients. 

And now, we have – outside of the context of clinical trials, because I want to talk about what’s actually available to patients now, we now have three JAK inhibitors available for myelofibrosis patients. And really, since 2011, we had only had one, and then, more recently, a second JAK inhibitor, but now, we have three. So, now we’re moving into an era where we can tailor a specific JAK inhibitor for a specific myelofibrosis patient, depending on what their particular needs are. So, I think that that’s very promising. And then, there are lots of clinical trials combining JAK inhibitors with new drugs. 

Katherine:

So, how does it work? How do clinical trials advance MPN research and treatment? 

Dr. Fleischman:

Well, there are multiple stages of clinical trials. One needs to have some rationale for testing a specific drug in patients. You just can’t say, I just want to take something off the shelf and see if it works for myeloproliferative neoplasms. 

There could be different ways that things sort of enter into clinical trials, either preclinical data from in vitro, meaning, in the lab, in the liquid media, with cells, that makes somebody think that it might work in humans, or that it works in a similar disease to myeloproliferative neoplasm. So, it’s a little bit of a stretch, but a very rational stretch, to then test it in a new population. 

First and foremost, safety needs to be evaluated, because as physicians, one of our primary objectives is to do no harm to patients. So, at very early stages of clinical trials, the primary objective is to see what the appropriate doses, what’s tolerated, what the side effect profile is. 

And then, moving on to efficacy. So, maybe it’s tolerated, but does it actually work at the next stage of clinical trials. Then, a much larger clinical trial would be to do a head-to-head comparison between, in most cases, standard of care versus drug X. 

And I think, for clinical trials, in particular, for myeloproliferative neoplasm, it’s very important to understand what the stated, primary end point is, in particular, for myelofibrosis patients, that myelofibrosis patients may have different problems. Some myelofibrosis patients, their primary issue may be anemia. And so, if they’re looking for a clinical trial to address their anemia, they would probably want to be looking for one whose primary end point is transfusion, freedom from transfusions, or improving the anemia, not necessarily – there was another trial that’s primarily looked at spleen reduction, but they didn’t have an enlarged spleen, that, necessarily, wouldn’t be appropriate for the patient. 

So, I think it is particularly important in myeloproliferative neoplasm to identify what the primary end point is, and whether what you’re going for is that primary end point. 

Katherine:

Mm-hmm. Any advances that are being done in MPN research require MPN patients to participate in clinical trials, right? 

Dr. Fleischman:

Of course. 

Katherine:

So, to start, let’s talk about where clinical trials fit into the treatment plan for ET, PV, and MF patients. When should a patient consider participating in a clinical trial? 

Dr. Fleischman:

Okay, well, I guess a patient could really consider participating in a clinical trial at any point if they had a very altruistic philosophy, that understanding that their participation may not necessarily help them at this moment in time, but may help others in the future, and we’ll gain knowledge about myeloproliferative neoplasms. 

That’s one approach. 

Another approach, which is probably a more usual approach, is when a patient has already tried standard therapies and they haven’t quite worked for them, or they’re in a class where, maybe, we don’t have really great standard therapies for somebody. 

For example, a myelofibrosis who may not be doing too well and may not necessarily be a candidate for a transplant, I think that’s a very reasonable population to go out and seek clinical trials, because there’s really not necessarily a great standard of care treatments for that patient population, or ET or PV patients who have tried standard of care and, maybe, can’t tolerate standard medications, or they’re just not working for them. 

But really, anytime somebody can do a clinical trial, if that’s what they feel is important to them.  

Katherine:

What are the benefits and risks of a trial participation? 

Dr. Fleischman:

So, the benefits are that you’re getting a drug that, potentially, is better than standard of care, that could be standard of care five to 10 years from now, but you’re getting it early.  

As investigators, ethically, we can’t start a clinical trial if we believe that the drug that we’re testing might have negative side effects on the patient, or maybe worse than standard of care. I mean, ethically, that’s not appropriate. So, ethically, we believe that what we’re testing may be better than what we’re currently giving patients, but we don’t know that. So, that’s the purpose of a clinical trial. 

So, a clinical trial, it’s a new drug. So, could have side effects that are on unanticipated, including death. I mean, that’s just the reality. That would be a very uncommon scenario, but it’s an unknown, so it’s an unknown. 

Other things that I think are very important to discuss are the financial implications of a clinical trial. On the pros, one could be getting a free drug that is outside of standard of care, and many of the tests that are done for the purposes of the research are covered. However, drugs, say, if it’s a combination drug, standard of care plus a new drug, the standard of care drug is usually billed to insurance. And so, the patient would need to pay for that, or if there are studies that would be considered standard of care, the patient would need to cover them. 

So, I think it, really, is important to discuss the financial implications. What money is it going to save you by participating, and may there be extra costs, or hidden costs, potentially, involved by participating? 

Katherine:

Yeah. Let’s talk about safety in clinical trials. Would you review the safety protocols that are in place before a clinical trial even begins? 

Dr. Fleischman:

So, before a clinical trial begins, there, usually, needs to be safety information in animals. Also, a lot of drugs have been tried in other diseases first. Either, they’re, have been studied in clinical trials and maybe not found to be very efficacious, but at least we have the value of the safety data in another population. 

So, we’re entering, again, into clinical trials with the understanding that it would not be harmful to humans with the data that we have available in animals, or in liquid culture. But again, we just don’t know that. And then, also, for many clinical trials, starting off at lower doses, and then, increasing the dose slowly in different cohorts of patients, to see what’s the maximally tolerated dose. 

As well as, when somebody is on a clinical trial, safety and side effects are very closely monitored, and even small side effects that likely have nothing to do with the drug, really do need to be investigated fully, just to make sure that they’re not related to the drug. 

Katherine:

Yeah. How do you know if the medicine is safe prior to starting a human trial? 

Dr. Fleischman:

That’s a great question. 

Based on what the molecule looks like, as well as, many times, they’ve been tested in animals to see – for example, for myeloproliferative neoplasm, it would be important to know, does it change a healthy rat’s blood count? Does it harm their liver? Those sorts of things, and safety information is usually available for a new drug. 

Katherine:

Are patients monitored more closely when they’re in a trial? 

Dr. Fleischman:

Yes, definitely. And for the purposes, mainly, of paying very close attention to even small side effects that, if somebody was not watched closely, may be missed because they’re so subtle. 

Katherine:

What if a patient decides to leave a trial? Does that negatively impact their care? 

Dr. Fleischman:

No, and I think that’s a very important point, that, ethically, as investigators, we cannot – and we do need to make it a point to communicate this fully with the patient, that when we’re asking the patient, or informing them about a potential clinical trial, we need to inform them that whether or not they participate will have nothing to do with the way that we treat them. We will treat them equally, regardless of whether or not they participate, as well as, anytime during the clinical trial, a patient has the absolute right, for whatever reason, they can decide to leave the clinical trial. That’s the most – I don’t say that’s the law, but those are the rules of clinical trials, as well as, a patient cannot be treated differently if they decide to leave a clinical trial.  

We have to be fair. I mean, this is – you have to be fair to all patients, and all patients deserve excellent treatment, regardless of whether they participate in the clinical trial. 

Katherine:

Dr. Fleischman, we’ve been talking about what happens when people participate in trials. But what if they don’t? Why is it crucial that patients participate in trials? 

Dr. Fleischman:

Because without participation in clinical trials, we are not going to further our understanding of myeloproliferative neoplasm. Many of the drugs that we use today in myeloproliferative neoplasms, as well as other diseases, the reason why we use them today is because people 10, 20 years ago participated in the clinical trial and demonstrated a benefit of these medications. So, people don’t participate, we’re not going to have new drugs for myeloproliferative neoplasms.  

Katherine:

All right. We know that much of the reason that people don’t participate is because of various stigma associated with clinical trials, and I’d like to talk about that with you. 

Let’s start with the word “experiment.” Why does this word not pertain to clinical trials? 

Dr. Fleischman:

So, I think the word “experiment” may have a negative connotation, and making the patient think, maybe they’ll say, a guinea pig. The only way that we can identify whether a drug is going to be beneficial is to test it out in humans with a particular disease. 

So, I mean, on one hand, it is an experiment, because we don’t know what’s going to happen, but we’re doing the experiment for the benefit of people who are suffering from the same disease. 

Katherine:

Yeah. Yeah. That’s a good explanation. What would you tell patients who are worried that they will receive a placebo? 

Dr. Fleischman:

So, that is part of a clinical trial, and it is also important to look how your clinical trial that you’re interested in is structured.  

So, some clinical trials do receive, or split into placebo, or active drug, and double-blinded means that the patient doesn’t know, nor the physician knows. So, no one knows, and that’s important because we don’t want to sway any subconscious things that, if you know you’re getting the drugs, then you’re going to say your symptoms are getting better, things like that. 

Again, ethically, in a clinical trial, we cannot not give somebody treatment that they – we can’t keep treatment from somebody. So, for example, if a person with polycythemia vera was a, per guidelines, should be on a cytoreductive agent, we cannot, ethically, treat them without a cytoreductive agent. So, it would be – they would have standard of care plus placebo, or drug X. 

So, maybe I’m not explaining this correctly, but if a placebo study is done, the placebo can’t take the place of something that we know is good for the patient. 

We can’t leave them hanging without any treatment, unless, for their specific situation, there’s not, necessarily, a known standard treatment, that it would be very reasonable to treat them with nothing.  

Katherine:

Another myth we often hear is that trials should only be considered if you have no other options. Why is that false? 

Dr. Fleischman:

I think there is a place for patients with no other options that – they may be more inclined to participate in, I want to say, higher risk studies, in which there’s less data to support a particular medication. But that’s why we look at these drugs in patients with no other options, because there’s no other reasonable thing to give them. 

But the patients with no other options may not be an accurate representation of the patient population, as a whole. So, it is important for people who may have other options, but maybe they want to think about, well, I do have a standard option, but maybe there’s something better out there for me, to participate in clinical trials. 

Katherine:

What if an MPN trial isn’t offered at the center where a patient receives care? What can they do?  

Dr. Fleischman:

Many times, specific clinical trials are only open at specific universities. And so, it’s very likely that your university, or the place where you receive care, may have a few clinical trials, or maybe one, or maybe zero for MPNs, but may not necessarily fit your exact circumstances. 

So, what I would recommend is, doing searching on your own, either through clinicaltrials.gov, or the MPN Research Foundation also has some nice resources, but doing some research on your own to identify some potential clinical trials that you’re interested in, and then go to your primary oncologist and say, “Hey, I printed these out. I think these might look really interesting to me.” 

And usually, on clinicaltrials.gov, they would have where they are, and you can actually, also, search for your state. So, maybe bring some that are close to you, and discuss with your primary oncologist the pros and cons of them. And then, ask your primary oncologist to make a referral to the location where they offer that specific trial. 

And a lot of times, you can – there’s a phone number you can call and be pre-screened. Say, “Hi, I’m a 55-year-old man with myelofibrosis,” and there are specific inclusion, exclusion, criteria that they can ask you. And if you don’t meet the inclusion criteria, then it’s not worth your time to go and have an actual visit, but if you do meet the inclusion criteria, then you could go and have an actual visit, and learn a little bit more.  

Katherine:

Oh, that’s great information. Thank you. Here’s a question we received from an audience member, prior to the program. Susan wants to know, “How can I get my community oncologist on board with trial participation? I’m interested in participating in a clinical trial that’s based in Chicago, and I’ll need her help in coordinating care with the team from a distance. Any advice for how to talk to my local doctor about that?”  

Dr. Fleischman:

So, that may be a tough one. So, many times, if somebody has to travel for a clinical trial, it does require some coordination. There are specific – and it’s clinical trial specific. There may be specific things that actually need to be done at the study site. For example, specific labs that would be drawn, and say, need to be frozen within two hours, or specific tests, for example, MRIs, if you need to look at the spleen size, you would need to do it on the same machine for everyone. 

So, there are specific things that have to be done at the location, or if it’s written to the protocol, you have to come to the location for a physical exam on this day and this day, and if it’s not within a two-to-three-day window, then there’s a deviation, and the data is not valid. 

So, what I would say is – sorry, this is a long answer here, but where certain things, if they’re written in the protocol that say a CBC could be drawn at any institution at week four, then that would be reasonable to have your primary oncologist do. But in the context of clinical trials, certain things are really set in stone as to the exact dates that needs to be done, and the exact location. And if they’re not done exactly, to a tee, then your data will not be – your data cannot be used for the analysis. 

Katherine:

Mm-hmm. But then, there’s also the issue of patients being willing and able to travel a distance to a teaching university where a clinical trial might be happening.  

Dr. Fleischman:

Correct, yes. And I think that, for some clinical trials, when the protocol is made, understanding that trying to minimize the trips to the actual site, and working the protocols, working some sort of wiggle room in the protocol, such that lots of stuff, or hopefully, lots of stuff, can be done remotely. But sometimes, it’s just not possible.  

Katherine:

Yeah. I’d like to turn our conversation to health disparities, Dr. Fleischman. Based on American history, some people believe that they won’t receive equitable or safe care if they participate in a trial. 

How can you reassure those people who are concerned they’ll be treated fairly? 

Dr. Fleischman:

Now, I think that this is a very important point, and something that there’s been a lot of emphasis, to try to improve diversity in clinical trials, because our American population is quite diverse. However, the participants that, in general, participate in clinical trials are, unfortunately, still have not a very diverse population in our clinical trials. 

I think what we need to first start doing is education, to reach out to underrepresented communities, to start to build the trust amongst these communities, to tell them about the value of clinical trials. And I think it’s going to take some time to build trust first, because it does take quite a bit of trust to participate in the clinical trial. 

But I don’t have a great answer for that, other than, we need to work hard to, first, build trust, and then, I think the diversity will come. 

Katherine:

Mm-hmm. How does holding on to some of these beliefs lead to limitations in care and create disparities? 

Dr. Fleischman:

So, and rightfully so, if a patient is scared, or has some reservations of participating in a clinical trial, they may – that’s offered to them, that they provide them with, potentially, something better than standard of care. They may be missing out on a potential opportunity. 

Also, potentially, if a patient, if they’re asked about a clinical trial and they have a negative connotation about them, they may lose trust with their physician, if they say, oh, my physician is asking me to participate in a clinical trial. 

This means that they’re thinking of me as an experiment, and maybe they’re not really thinking of me as patient. And so, they may not have that trust with their physician, and so, may not be as open, in terms of communication, with their physician.  

I think it all boils down to trust, and as physicians, we need to demonstrate that we are worthy of the patient’s trust, and we really are ingrained in us to treat every patient the same. I mean, that’s what our oath is. That’s what we’re supposed to do, and I think that the vast majority of patients, they have, ethically, are treating patients exactly the same, regardless of their circumstances. 

Katherine:

Yeah. Health equity means that no matter what a patient’s circumstances, whether it be race, income issues, lack of education, that they should have access to the best care. What is being done by the medical community to address this issue? 

Dr. Fleischman:

So, yes, this is a significant issue, and in particular, with myeloproliferative neoplasms, in whom there are lots of oral drugs – or with interferons, it’s injectable, but you get the prescription, and you give it to yourself – that there can be quite high copays, in some cases, exorbitant amounts, which, really, are not able to be paid for by the vast majority of people. 

So, many companies do have copay assistance programs. Also, foundations have copay assistance programs. So, I think that is, at least, one step in trying to make things more equitable, to get people who need a drug, their drug, at a very reasonable cost. Again, it does take some time, some legwork on the part of the patient, to seek out these programs, or to find an advocate for themselves to seek out these programs for them. 

Katherine:

Yeah. Would a healthcare team be part of that process, though? Would they be able to help the patient? 

Dr. Fleischman:

They will be able to help the patient in terms of saying, “Hey, there’s this program for this drug. Why don’t we fill out the form together?” Or, “Why don’t you call this,” you know. Many times, the patient needs to initiate the process. So, I think the healthcare team can sort of guide the patient in saying, this is what’s available, we can help. We can fill out our portion of the form, you fill out your portion of the form. But no, it does need to be – the patient needs to be an active participant in seeking out the support. 

Katherine:

Mm-hmm. Before we end the program, Dr. Fleischman, I’d like to close with some advice from you. What do you want to leave MPN patients with, relating to clinical trial participation? 

Dr. Fleischman:

I would say that MPN patients today are the key to our future treatments. 

Without participation in clinical trials today, there’s going to be no new drugs for myeloproliferative neoplasms. They’re just not going to appear. We need to test them in patients before them actually coming to market, and before really knowing whether they work or not. So, I would say that the MPN patients today are the key to the future of MPN treatments.  

Katherine:

Dr. Fleischman, thank you so much for joining us today. 

Dr. Fleischman:

My pleasure. As always, I really enjoy connecting with MPN patients, and I think this was a very important topic to discuss.  

Katherine:

Yeah. And thank you to all of our partners. To learn more about MPNs, and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us today. 

Advice for Hesitant MPN Clinical Trial Participants

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Advice for Hesitant MPN Clinical Trial Participants from Patient Empowerment Network on Vimeo.

What should MPN patients know about clinical trials? Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares information about the varying degrees of clinical trials and advice to those who are hesitant about clinical trials.

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How Can MPN Patients Become More Proactive in Their Care?


Transcript:

Dr. Nicole Rochester: 

It’s said that clinical trials are tomorrow’s medicine today, and you’ve already kind of alluded to the importance of clinical trials as it relates to MPN. What would you say to an MPN patient who is on the fence or may be concerned or afraid of participating in a clinical trial?

Dr. Claire Harrison: 

It’s right to be cautious and, you know, careful because ultimately it’s a huge privilege as a clinician that involves patients in clinical trials that my patients trust me and trust my team to look after them with something that is experimental, but remember there are varying degrees of experimental. Most clinical trials are not first in man, you’re not a complete guinea pig, it may be a drug, for example, navitoclax is in clinical trials mainly for myelofibrosis also ET and PV but that is a drug that has been used for thousands of patients, for another indication so talk to your healthcare team, if you don’t find the answer from the primary person that you’re used to dealing with, find someone else, be linked to somebody you trust and that you have a good relationship with, take someone with you to the consultation, write down the questions I’m so sure you say this all the time, don’t you Nicole to the people that you talk to, but write down your questions, don’t be afraid to ask them again, there is no stupid question in this context, you will be given a 30-plus page booklet to read, and I lost count of the number of times, my patients go, yeah, I’ve got this, or I trust you.

Actually, you know, you need to read it…we are experimenting on you, and you need to read that and understand. And you need to understand, what happens if I go on the control arm, will I be able to cross over? How many visits will I have, will I have to pay for those visits, etcetera. It’s all really important. But ultimately the relationship with your healthcare provider is important, and using an advocate is really important too.

Dr. Nicole Rochester: 

I agree 100 percent. So important, these are things that I talk about all the time, so I really appreciate that you highlighted that, and just the importance of patients taking an active role in their medical care and also the trust that is required between the patient and their treating providers. So I really appreciate that. Do you have any examples, Dr. Harrison, in your own practice of successes with MPN patients who have participated in clinical trials? 

Dr. Claire Harrison: 

Oh yes, I think I started doing clinical trials, well golly, a long time ago. I think my first clinical trial, probably the records were written parchment to be honest, but we’ve still learned a lot from that, so that was an ET study. It was from that study we understood about the JAK2 mutation, and we understood how patients behave differently. I think probably the most gratifying thing for me was being involved in the JAK inhibitor studies in myelofibrosis and being involved in delivering ruxolitinib and Jakafi to patients and seeing the benefits for those patients. 

Big things, you know, there are patients who are alive because they took part in that trial today, I think, but there are also patients for whom small things were also really important, so as a patient, that’s important to define what is the benefit you want to get. So one of my first patients, you haven’t been able to have a bath or a shower for years, because he had terrible what we call aquagenic pruritus, itching induced by contact with water, we called him two days after he started ruxolitinib (Jakafi), and he was in tears, he could take…or you can take it out.

These things are really important. Like myself, I can imagine not being able to dig it out, I would either be very tough for another patient, it was, well, I looked really skinny because I’d lost loads of weight and I put weight on, and body image was really important as well, but then the small things like being able to be…participate more in family activities is really, really important too. 

What Are the Unmet Needs in Access to MPN Care?

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What Are the Unmet Needs in Access to MPN Care? from Patient Empowerment Network on Vimeo.

Which areas of MPN care still need improvements to access? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London explains patients who still experience barriers to care and what can be done to reduce access issues.

See More from Best MPN Care No Matter Where You Live

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Advice for Hesitant MPN Clinical Trial Participants


Transcript:

Dr. Nicole Rochester: 

What would you say are the unmet needs in access related to MPN and care, specifically as it relates to clinical trials, and what can we do to address those unmet needs?

Dr. Claire Harrison: 

Well, I think there is a problem with rare diseases in terms of geographical access to trials, and we often find patients have to travel a long way. I know that’s true in North America as well as in Europe. And we’re very lucky in our geographical locations, but in some parts of the world, some companies or doing not open clinical trials, so I think there’s an access issue. 

I think also there is something about patients have to meet rigid entry criteria for clinical trials, and so oftentimes in myelofibrosis, for example, commonly patients who fail ruxolitinib (Jakafi) have a lower platelet count, and that is often an exclusion criteria. Those criteria are there to try to get a uniform population of patients in a trial, but it can feel like you’re excluded as a patient, and it can feel very tough and for your health care team that we can’t include you in a clinical trial. We also have to remember that it is there for safety purposes, so if there is a lower limit for platelet count, that’s often because the drug might affect platelet count. It is really important that we have a broad spectrum of trials available and that we try to increase the availability of trials for patients. 

I also want to say a word about inequality of access and thinking about accessing some different ethnicity, so often non-white MPN patients are under-represented in clinical trials, and I know that a focus in the UK and also in North America as well. And it is really important that patients have access to a clinical trial if they need it, and also that we understand how investigational products will work in people of different backgrounds. So for example, we know that probably, Nicole, your blood count assuming it’s a healthy, normal blood count may well be different from mine for background, racial genetic differences, so drug metabolism might be different, so this is really important, and we need to work hard as a community, the clinical community and the patient community to raise awareness and improve access for patients. 

Dr. Nicole Rochester: 

Well, as someone who does a lot of work in health equity, Dr. Harrison, I really appreciate you pointing that out. It’s certainly an issue here in the United States, as you mentioned, differential access to clinical trials, and we’ve learned that not only our patients, often not aware, but often the providers, at least here in the U.S., are not offering clinical trials as an option for patients from marginalized and minoritized communities. So I really appreciate you bringing that up. 

How Can MPN Patients Stay Up to Date With New Treatments?

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How Can MPN Patients Stay Up to Date With New Treatments? from Patient Empowerment Network on Vimeo.

What are some ways for MPN patients to stay updated on new treatments? MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares resources for MPN treatment news and the benefits that data sharing brings for all MPN patients.

See More from Best MPN Care No Matter Where You Live

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How Are MPN Treatments Changing for Low-Risk vs High-Risk Patients?


Transcript:

Dr. Nicole Rochester: 

Dr. Harrison, how can patients best keep up with the new treatments and communicate with their doctors in a way that makes sure that they have access to these new therapies?

Dr. Claire Harrison: 

Well, I think patient advocacy groups are really important here, and use of social media and the Internet, you’re only a few clicks away from updated data, programs like these, but you also have to trust your team and trust your doctor. We all have to keep up to date. That is a professional requirement, and we also are all networked, so I’d probably get 10 to 15 emails a day from colleague saying, “Hi Claire, can I talk to you about this?” There was an email just the other day about a patient in Washington, actually a very young child, we are all connected. 

We all want the best for our patients. But do you remember that you can contribute as a patient to advance this in your field, and I know many patients are really interested in this, if you are asked to submit in a blood sample, giving permission for us to use your data. So if we can touch maybe on the field of real-world data and real-world data collection here would be good, so what on the real world data? What does that mean? And so this is becoming a really important way that’s recognized by the FDA and other approval agencies in the world, so in Europe, we have EMEA, for example, and in the UK, we have NHRA as a way of collecting data on agents, so once they are approved, we collect data with regard to how the patients do. 

We’ve traditionally done this, but increasingly, as we use electronic data for our patients, we’re more able to collect real-world data, how does my patient who is with myelofibrosis on ruxolitinib (Jakafi) in my clinic inside East London do? So if we can pull that data, we learn a lot more about how these agents are working in patients outside of clinical trial, so you will be contributing if you allow us to collect that kind of data, we discovered JAK2 mutations, CALR mutations, etcetera from samples collected from patients and data. If you want to be part of a clinical trial, then by all means, ask your healthcare team, many MPN centers have lists of trials, and you can always look at clinicaltrials.gov, but boy you throw up a lot of different options when you search in that…on that website. 

Dr. Nicole Rochester: 

Thank you. I think it’s important to talk about real-world data because in this day and age, many of us are very protective about our personal health information and we should be, but as you stated, having access to that data is really a key way to advance the science and technology in the treatment of some of these conditions, so I really appreciate you sharing that.

Dr. Claire Harrison: 

I think just to point out, and I’m sure the audience is acutely aware of what did we learn about COVID? We learned an awful lot about COVID from real-world data from all you MPN patients who gave samples, who told us about how you did with COVID, that’s how we learned about what happened to patients during COVID with MPN, how they responded to vaccination, etcetera. It’s really powerful. And your data will be anonymized, it won’t be linked back to you, Nicole Rochester, or me, Claire Harrison it will be completely anonymous. 

How Can I Get the Best Myeloproliferative Neoplasm (MPN) Care?

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How Can I Get the Best Myeloproliferative Neoplasm (MPN) Care? from Patient Empowerment Network on Vimeo.

 There are many exciting developments in the myeloproliferative neoplasms (MPNs) research pipeline, but how can patients get the best possible MPN care? Internationally respected MPN expert Dr. Claire Harrison from Guy’s and St. Thomas’ Hospital in London shares information about research updates, treatment strategies, clinical trials, and how to achieve patient-centered care for you or your loved one.

See More from Best MPN Care No Matter Where You Live

Related Resources:

How Do I Best Communicate My Concerns Without Feeling Dismissed


Transcript:

Dr. Nicole Rochester:

Hello and welcome. My name is Dr. Nicole Rochester, I’m a pediatrician and a professional health advocate, and your host for today’s Patient Empowerment Network program. We are thrilled that you have decided to tune in. With so many exciting developments in the research pipeline from myeloproliferative neoplasms or MPNs. We’re going to answer several important questions during today’s program, how can you as a patient access the best possible MPN care? Will there be alternative treatment strategies for MPN patients who have failed previous therapies? Should you consider a clinical trial as a path to enhancing your care? Whether you’re living with polycythemia vera, essential thrombocythemia or myelofibrosis, we have so much to unpack and we are joined by world-renowned and highly respected MPN expert, Dr. Claire Harrison. Thank you so much for joining us, Dr. Harrison, it’s an honor and a privilege to join with you.

Dr. Claire Harrison:

It’s a great pleasure and it’s a privilege to join you as well.

Dr. Nicole Rochester:

Following this program, you will receive a survey and we’d be delighted if you take some time to provide your feedback that helps inform future MPN programs that we produce. Please remember that this program is not a substitute for seeking medical care, so please be sure to connect with your healthcare team to determine what is best for you. Now, let’s dive right into this very important topic, how can you get the best myeloproliferative neoplasm care. 

Dr. Harrison, we know that the treatment landscape for MPN is rapidly changing and keeping up with the pace of the developments can be challenging both for healthcare professionals and certainly for patients and their families, so I was wondering if you can give us a general overview of the treatment landscape and maybe highlight anything that’s new and in development that you think would be helpful for the audience.

Dr. Claire Harrison:

Well, sure, I think this is obviously really interesting, very important. It’s a fast-moving field. And one of the first changes actually completely hot off the press is a brand new diagnostic criteria that has been produced by two separate groups that are shortly about to be published, kind of just seeing pre-published and those of us that have been privileged to be involved, have seen them. So that’s gonna really focus the mind of clinicians on how do we achieve the diagnosis, and that’s the first thing that’s really important, get an accurate diagnosis, don’t just accept well, you’ve got an MPN, actually, it’s really important to know what type you’ve got. 

Maybe as much do you tell as possible in terms of the genetic mutation or change that underlies the disease, because that is increasingly important in what we call prognostication, understanding what the risk of events happening due to the disease actually is. When we make a diagnosis, we sometimes can’t be accurate and we might have to go back and revisit them, so some patients don’t get a PV, ET or MF diagnosis, they might get an MPN unclassified. That’s okay, but it’s important to keep revisiting that.

Once we’ve made a diagnosis, then it’s really important to think about what the risk is to the patient, and we’ve had some changes to our risk classifications recently, right from the ET end of the spectrum where we’re thinking more and more actually, those patients who are under the age of 60 with a lower count and the CALR mutation, do we even need to give you aspirin because we might be increasing the risk of bleeding. 

To the other end of the spectrum for patients with myelofibrosis, a more aggressive disease, we want to know more about your mutational profile, so we’re doing more powerful genomic tests and assessing them, your prognosis, and then what the features of your disease are that need treatment. And there are lots of changes, which I think we’ll get into later in our conversation here and lots of new options, which are really important, I just also don’t to leave this segment without saying to all of you who are listening, it’s important that you understand your disease, it’s important that you understand the diagnosis, prognosis, etcetera, and you get the best care.

But that’s maybe not enough, you need to know that you’re taking good care of you, and that’s something that’s really important to all of us, so you need to know that you’re managing your vascular risk don’t just think about your blood. Think about the fact that you know if you’re smoking, quit smoking, if you’re drinking too much, cut it down, if you’re not walking enough,  walk more, lose weight. The majority of patients with MPN actually have a problem with a blood clot, not a further complication of their disease, so maybe we’ll stop there and then we can dive in a bit more deeply

Dr. Nicole Rochester:

Thank you, I appreciate you pointing out the changes with the diagnosis. I’m really excited to learn more about that, and also pointing out the importance of self-care and some of the other risk factors that individuals with MPN can mitigate. So I think that’s extremely important. Can you share a little about what’s in the robust pipeline of potential therapies for patients with MPN, what is it that you’re excited about? And is the future bright in this area?

Dr. Claire Harrison:

I think the future is really bright from the point of view of de-escalating treatment as well as newer treatment, so I think it’s important to point that because all treatments have potential complications inside of it, so as we understand that some of these conditions may have very low risk for patients, it’s important to understand that, and de-intensify, I call that calreticulin (post ET?), I would also call out ET, essential thrombocythemia, which lacks a known driver mutation, so-called triple-negative ET, emerging data suggests that may have very low risk for patients, but what you all want to hear about, of course, is what’s new treatment-wise. So I think just to call out, I’m really excited that there will be a new trial this year for ET patients with Bomedemstat, which is an LSD-1 inhibitor, new target, new molecule. We’ve been testing it in myelofibrosis and we’ve tested it now in a bunch of patients with ET, and it seems to be very efficiently reducing the platelet count not affecting hemoglobin and patients appear to get a good benefit with regard to fatigue, which we know is the number one symptom for patients with MPN, so I’m excited about that because it’s been a long time since we’ve had a new treatment for patients with ET. And then for patients with PV, increasingly across the globe, the availability of this newer formulation of interferon, Besremi is becoming more available and the latest data with that agent suggests that it may be superior to standard therapy such as hydroxyurea, hydroxycarbamide in terms of clotting, et cetera, is really important.

Interestingly, and we may both have to think back to our med school days on this, we’ve been targeting the iron pathway in for patients with PV. So I always tell my patients with PV, do not let anyone give you iron tablets without speaking to one of our team because that’s like putting oxygen on the fire, it’s like feeding the red cell production. So there is a new agent called Rusfertide PTG-300, which targets the iron pathway and allows iron to build up in the body, but it doesn’t allow it to get to the bone marrow and so, this is a new treatment for PV patients, which might reduce the need for iron removal by phlebotomy or venesection, and has been also shown to give symptomatic benefit, and then of course, there’s a bunch of new treatments for patients with myelofibrosis, that’s probably the busiest part of the portfolio at the moment. 

We’ve just seen positive data with me Momelotinib, which is one of the fourth JAK inhibitors, very strong data from the momentum study, good results in patients even with low platelet counts down to 25, and then I’m really excited to see strong data coming with Navitoclax and Pelabresib, which are other agents targeted at are the pathways in myelofibrosis and then finally, in Denmark, they’ve been looking at vaccination strategies, and I know my patients are really interested in vaccination and (inaudible). I don’t have anything new to say (inaudible), but I do have something new to say on vaccination.

So in Denmark, they’ve been looking at producing a vaccination against the calreticulin mutation Nicole, because it’s expressed on the surface of the blood cells, so antibodies can find it. So this is ongoing, no positive result as yet, but it’s still ongoing and there are newer taking off with regards to vaccination structures, and I think that’s really exciting.

Dr. Nicole Rochester:

Wow, there is a lot on the horizon for MPN patients. That is extremely exciting, Dr. Harrison, how can patients best keep up with the new treatments and communicate with their doctors in a way that makes sure that they have access to these new therapies?

Dr. Claire Harrison:

Well, I think patient advocacy groups are really important here, and use of social media and the internet, you’re only a few clicks away from updated data, programs like these, but you also have to trust your team and trust your doctor. We all have to keep up to date. That is a professional requirement, and we also are all networked, so I’d probably get 10-15 emails a day from colleagues saying, Hi Claire, can I talk to you about this? There was an email just the other day about a patient in Washington, actually a very young child, we are all connected. 

We all want the best for our patients. But do you remember that you can contribute as a patient to advance this in your field, and I know many patients are really interested in this, if you are asked to submitting a blood sample, giving permission for us to use your data. So if we can touch maybe on the field of real world data and real-world data collection here would be good, so what about the real world data? What does that mean? And so this is becoming a really important way that’s recognized by the FDA and other approval agencies in the world, so in Europe, we have EMEA for example, and in the UK, we have NHRA as a way of collecting data on agents, so once they are approved, we collect data with regard to how the patients do. 

We’ve traditionally done this, but increasingly, as we use electronic data for our patients, we’re more able to collect real-world data, how does my patient who is with myelofibrosis on Ruxolitinib in my clinic inside East London do. So if we can pull that data, we learn a lot more about how these agents are working in patients outside of clinical trial, so you will be contributing if you allow us to collect that kind of data, we discovered JAK2 mutations, CALR mutations, etcetera from samples collected from patients and data. If you want to be part of a clinical trial, then by all means, ask your healthcare team, many MPN centers have lists of trials, and you can always look at clinicaltrials.gov, but boy you throw up a lot of different options when you search in that… On that website.

Dr. Nicole Rochester:

Thank you. I think it’s important to talk about real-world data because in this day and age, many of us are very protective about our personal health information and we should be, but as you stated, having access to that data is really a key way to advance the science and technology in the treatment of some of these conditions, so I really appreciate you sharing that.

Dr. Claire Harrison:

I think just to point out, and I’m sure the audience is acutely aware of what we learn about COVID? We learned an awful lot about COVID from real-world data from all you MPN patients who gave samples, who told us about how you did with COVID, that’s how we learned about what happened to patients during covid with MPN, how they responded to vaccination, etcetera. It’s really powerful. And your data will be anonymized, it won’t be linked back to you, Nicole Rochester, or me, Claire Harrison it will be completely anonymous.

Dr. Nicole Rochester:

Absolutely, thanks for clarifying that. I want to go back to treatment strategies, you’ve mentioned earlier about low-risk versus high-risk patients, and that some of those criteria are changing. How are treatment strategies changing for low-risk and high-risk patients with MPN?

Dr. Claire Harrison:

It’s complicated because we need to think across the entities, and we don’t have an answer to that for patients with MPN unclassified and we don’t actually have a good answer to that for this entity called pre-fibrotic myelofibrosis which does appear and is strongly recognized in the new diagnostic criteria, but for ET, for example, low-risk patients I mentioned triple negative, calreticulin, m-positive, young patients, platelets less than 1500, not too much changing their queries about aspirin or not, and then for PV patients, we haven’t really changed all kind of high-risk criteria and for both ET and PV, the questionnaire is, should we use the treatment above aspirin or above aspirin (inaudible). And for the most part, that would be hydroxycarbamide, hydroxyurea, which is the commonest treatment used worldwide or Interferon, and these are the right treatment for some patients and not the right treatment for other patients, so some patients can be very fixated on interferon is the absolute best, but there is no clear evidence of that, and there are some patients who interferon is not the right treatment, but low versus high risk becomes even more important for myelofibrosis patients.

And here, we’re thinking about using a risky strategy like transplantation for those patients who have higher risk disease, and we’re using, as I mentioned to you, these molecular markers and newer prognostic tools to stratify patients, and it is important to remember is a patient leave if someone puts your data into a prognostic tool and that comes up with five years, but it doesn’t mean to say five years on the dot your times up, that’s an average. 

And if we put your data into a slightly different tool, we might get something else. So for the most part, we make decisions like transplants,  we are learning more about transplantation and outcomes from that, and then in some countries, some treatments are used for patients who fall into intermediate or high-risk categories, and some clinical trials are based on that as well. I would want to say about myelofibrosis, and something I think I would really like to see changed, not changing yet, but changed, is that we should be able to intervene for patients with a low-risk disease. If my myelofibrosis patients have breast cancer, we would not be going there, then you’ve got low-risk disease we’ll put you on watch and wait, watch and wait is really hard for our patients, we know that I can see you nodding.

Dr. Claire Harrison:

You know that too, right? So if these were patients with breast cancer we would not say, We’ll just watch and wait. So I would really like to see in the next five to 10 years a treatment that we could use earlier in the disease course, but there is nothing at the moment, but we’re looking at that. The other thing we’re looking at, if we’ve got a minute or so is the different endpoint, so we’re trying to understand what does it mean if you’re a (?), so the amount of abnormal genes you’ve got goes down, the amount of bone marrow fibrosis you’ve got goes down. And again, this is something we’ll collect in a clinical trial, but also from real-world data.

Dr. Nicole Rochester:

Wonderful. Wow, thank you. What about for patients who have failed therapies, are there any treatment strategies for MPN patients who have failed traditional therapies?

Dr. Claire Harrison:

Yes, in fact, actually, that’s where we’re evaluating new therapies across all of these entities, so if you’re a PV or an ET patient and you failed a therapy, then this is where, for example, in ET, we would be looking at Bomedemstat or we’re looking at the bromodomain inhibitor Pelabresib, and there’ll be other agents that we’ll be looking at, or we might be looking at vaccination. And for MF patients that while there are a bunch of different therapies for patients who you have not tolerated or progressed through standard therapy. So actually, there’s a lot of options, some of them are already approved and some of them are in clinical trials.

Dr. Nicole Rochester:

And you mentioned clinical trials, and so I think this is a perfect opportunity to transition and start to talk more about clinical trials as a treatment option for NPM patients and really focusing on treatment access, what would you say are the unmet needs in access related to MPN and care, specifically as it relates to clinical trials, and what can we do to address those unmet needs?

Dr. Claire Harrison:

Well, I think there is a problem with rare diseases in terms of geographical access to trials, and we often find patients have to travel a long way. I know that’s true in North America as well as in Europe. And we’re very lucky in our geographical locations, but in some parts of the world, some companies or doing not open clinical trials, so I think there’s an access issue. I think also there is something about patients have to meet rigid entry criteria for clinical trials, and so oftentimes in myelofibrosis, for example, commonly patients who fail (?) have a lower platelet count, and that is often an exclusion criteria. Those criteria are there to try to get a uniform population of patients in a trial, but it can feel like you’re excluded as a patient, and it can feel very tough and for your health care team that we can’t include you in a clinical trial. We also have to remember that it is there for safety purposes, so if there is a lower limit for platelet count that’s often because the drug might affect platelet count. It is really important that we have a broad spectrum of trials available and that we try to increase the availability of trials for patients. 

I also want to say a word about inequality of access and thinking about accessing some different ethnicity, so often non-white and patients are under-represented in clinical trials, and I know that a focus in the UK and also in North America as well. And it is really important that patients have access to a clinical trial if they need it, and also that we understand how investigational products will work in people of different backgrounds. So for example, we know that probably Nicole, your blood count assuming it’s a healthy, normal blood count may well be different from mine for background, racial genetic differences, so drug metabolism might be different, so this is really important and we need to work hard as a community, the clinical community and the patient community to raise awareness and improve access for patients.

Dr. Nicole Rochester:

Well, as someone who does a lot of work in health equity, Dr. Harrison, I really appreciate you pointing that out. It’s certainly an issue here in the United States, as you mentioned, differential access to clinical trials, and we’ve learned that not only our patients, often not aware, but often the providers, at least here in the US, are not offering clinical trials as an option for patients from marginalized and minoritized communities. So I really appreciate you bringing that up. It’s said that clinical trials are tomorrow’s medicine today, and you’ve already kind of alluded to the importance of clinical trials as it relates to MPN. What would you say to an MPN patient who is on the fence or may be concerned or afraid of participating in a clinical trial?

Dr. Claire Harrison:

It’s right to be cautious and you know, careful because ultimately it’s a huge privilege as a clinician that involves patients in clinical trials that my patients trust me and trust my team to look after them with something that is experimental, but remember there are varying degrees of experimental most clinical trials are not first in man, you’re not a complete gene page, it may be a drug, for example, Levetoclax (?) is in clinical trials mainly for myelofibrosis also ET and PV but that is a drug that has been used for thousands of patients, for another indication so talk to your healthcare team, if you don’t find the answer from the primary person that you’re used to dealing with, find someone else, be linked to somebody you trust and that you have a good relationship with, take someone with you to the consultation, write down the questions I’m so sure you say this all the time, don’t you Nicole to the people that you talk to, but write down your questions, don’t be afraid to ask them again, there is no stupid question in this context, you will be given a 30-plus page booklet to read, and I lost count of the number of times, my patients go, yeah, I’ve got this or I trust you.

Actually, you know, you need to read it… We are experimenting on you, and you need to read that and understand. And you need to understand, what happens if I go on the control arm, will I be able to cross over? How many visits will I have, will, I have to pay for those visits, etcetera. It’s all really important, but ultimately the relationship with your healthcare provider is important and using an advocate (inaudible) is really important.

Dr. Nicole Rochester:

I agree, 100%. So important, these are things that I talk about all the time, so I really appreciate that you highlighted that, and just the importance of patients taking an active role in their medical care and also the trust that is required between the patient and their treating providers. So I really appreciate that. Do you have any examples, Dr. Harrison in your own practice of successes with MPN patients who have participated in clinical trials? 

Dr. Claire Harrison:

Oh yes, I think I started doing clinical trials, well golly a long time ago. I think my first clinical trial, we probably the records of written parchment to be honest, but we’ve still learned a lot from that, so that was an ET study. It was from that study we understood about the JAK2 mutation on and we understood how patients behave differently. I think probably the most gratifying thing for me was being involved in the JAK inhibitor studies in myelofibrosis and being involved in delivering Ruxolitinib and Jakafi to patients and seeing the benefits for those patients. 

Big things, you know, there are patients who are alive because they took part in that trial today, I think, but there are also patients for whom small things were also really important, so as a patient, that’s important to define what is the benefit you want to get. So one of my first patients, you haven’t been able to have a bath or a shower for years, because he had terrible what we call aquagenic pruritus itching induced by contact with water, we called him two days after he started Ruxolitinib and he was in tears, he could take… Or you can take it out.

These things are really important. Like myself, I can imagine not being able to dig it out, I would either be very tough for another patient, it was, Well, I looked really skinny because I’d lost loads of weight and I put weight on, and body image was really important as well, but then the small things like being able to be… participate more in family activities is really, really important too.

Dr. Nicole Rochester:

Wonderful, so what advice would you give for patients so that they can really take a proactive approach to their healthcare and feel more confident in talking about their concerns and communicating with their healthcare team, you’ve shared with us how important that is. Do you have maybe two or three specific tips or maybe questions that every MPN patient should ask their healthcare provider?

Dr. Claire Harrison:

I think the first thing to say is, in my personal view is you do not have to be under an MPN expert to get the best care. I know some people differ with regard to that, but these are chronic conditions, there are national and international guidelines, clinicians are connected. We all talk about patients over time, as we like to do that, we like to get the best for our patients, so a local center with a clinician who you trust, who you get on with… Where you can get there easily. You trust their team, you know their logistics work for you, maybe it’s a nurse who work who you get on with, well, who comes to the appointment with you, that is just as good as being under the best professor in the state, where you might not actually see them  when you turn up and go to the unit, so that’s really important, understanding your condition, and if you don’t understand being empowered to ask questions, and if you’re in a position where you can’t ask a question, something’s wrong. So don’t be afraid, take somebody with you, write it down. Sometimes it can be a mistake to do a troll on the internet, so I wouldn’t always encourage that because what’s on the internet is not always accurate, but go to a trusted website as the clinician… Where can I go to find out more information? Some patient advocacy groups run buddy systems that can also be very helpful and it can be very empowering to meet another patient with the same or similar condition, so I think those are all helpful tips from my perspective, also don’t expect to get all the answers all the time, it can be really tricky as a clinician, maybe you get a patient who comes with a big long list of questions, and say What is your top question that you really want answers to.

Dr. Nicole Rochester:

Those are awesome, awesome tips. I’m just gonna repeat a few of them, just to highlight, you mentioned prioritizing your concerns which is incredibly important, and acknowledging that the clinician doesn’t have unlimited time, and so really focusing on the things that concern you the most, you mentioned bringing a buddy to appointments, which is something I fully endorse, so that there’s someone else that’s taking notes or… It can be your eyes and ears during that appointment, things that you may have missed either because of anxiety or stress, and you mentioned writing things down, taking notes, even as the patient asking questions, which is so incredibly important, and really the way that I feel patients demonstrate their involvement in their disease and being an active member of the team, so I really, really appreciate those tips, Dr. Harrison, I think that you have given us so information, so much information about how to empower MPN patients and their families so that they can really get the best care at the outset. So it’s time to wrap things up, Dr. Harrison, I’d love to close with any closing thoughts that you have, any takeaway messages you’ve given us so many already, but if there’s anything else that you have not had the opportunity to share with the audience, I love for you to go ahead and do that now.

Specifically anything related to how they can advocate for themselves or any other important messages that you wanna leave the audience with.

Dr. Claire Harrison:

I think I would want the listeners to feel empowered and to feel very hopeful, this is the time where there’s a great change. We’ve been through a really difficult couple of years, but actually, we group together really well as a patient in a clinical community, and we’ve learned a lot, so trust your clinical team if you don’t trust them if there’s a problem. Move on. And don’t be afraid to do that, don’t be afraid to ask for another opinion, actually, we as clinicians like somebody else to give an opinion on our patients, that’s another thing we haven’t covered, and do connect with patient advocacy and keep up-to-date. Do you ask for a copy of your letter, don’t be afraid to ask for copies of your diagnostic information, you will properly outlive the relationship with your clinician. I will probably retire before my patients move on from my practice, so keep the information and understand it as much as you can.

Dr. Nicole Rochester:

Wonderful, thank you. So Dr. Harrison and you’ve just left us with a message of hope and a message of empowerment, and I think those two things are incredibly important, so I really appreciate you taking time with us today, and thank you so much for sharing your insights and your expertise. And I wanna thank you all for tuning in to this Patient Empowerment Network program. 

Notable New MPN Treatments

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Notable New MPN Treatments from Patient Empowerment Network on Vimeo

What’s the latest in myeloproliferative neoplasm (MPN) treatments? Dr. Kristen Pettit from Rogel Cancer Center gives updates about treatment developments for myelofibrosis, polycythemia vera (PV), and essential thrombocythemia (ET) care including JAK inhibitors, BCL-2 inhibitors, BCL-XL inhibitors, BET inhibitors, and others.

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Transcript:

Dr. Kristen Pettit:

There are so many new treatments in MPNs that I’m excited about, so one that is investigational that has been moving through the pipeline is momelotonib, which is a newer JAK inhibitor for patients with myelofibrosis. The mechanism of action is slightly different than the other JAK inhibitors, ruxolitinib (Jakafi), baricitinib (Olumiant), and pacritinib that’s improved as well.

The hope with momelotonib is that it will do some of the good things that the other JAK inhibitors do reduce symptoms and reduce spleen size, but also potentially improve anemia for patients who struggle with anemia, which is such a high proportion of our patients with myelofibrosis. I think that’s one exciting thing, another exciting avenue are new potential up-front treatment strategies that are being studied, so there are a number of different clinical trials going on right now, testing the strategy of either standard ruxolitinib or Jakafi by itself compared head-to-head versus a combination of Jakafi plus another medication.

Those other medications that are being tested in trials include the BET inhibitor called parsaclisib, also BCL-2, BCL-XL inhibitor called navitoclax, and a Pi3 Kinase inhibitor called parsaclisib. These upfront head-to-head treatment strategies are going to be very important to keep an eye out for over the next couple of years. The hope is that these combination strategies could deepen responses and potentially prolong responses when a new drug is combined with the JAK inhibitor as the first treatment option for patients with myelofibrosis. In the later line setting, one thing that’s exciting is Imetelstat, which is a telomerase inhibitor.

This is the first study in my myelofibrosis that is trying to specifically prove whether or not it is linked in survival for patients with myelofibrosis. So, I think that’s very exciting and something to keep an eye out for.

In polycythemia vera, one newer treatment option that’s getting a lot of excitement is the Hepcidin mimetic called rusfertide, this medication will hopefully harness the body’s iron metabolism pathway and act as sort of a chemical phlebotomy as opposed to an actual therapeutic phlebotomy in order to control the hemoglobin and hematocrit for patients with PV as well as improve symptoms.

In ET the newer agents that are being investigated include the BET inhibitor parsaclisib, is also being studied for myelofibrosis as well as an LSD-1 inhibitor called bomedemstat. Both of these look exciting so far, as far as their ability to both control platelet count and improve symptoms for patients with ET.

An MPN Expert Defines Clinical Trial Types

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An MPN Expert Defines Clinical Trial Types from Patient Empowerment Network on Vimeo.

 What are the types of clinical trials? Dr. Ruben Mesa explains the differences and discusses what patients should expect with each type.

Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as executive director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert, here.

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Transcript:

Katherine:                  

You touched upon the various types of clinical trials and I would like to look at each one of them individually. So, let’s start with the double-blind clinical trial. What is that?

Dr. Mesa:                   

What that refers to is that neither the patient nor the physician knows which of the two comparator arms of the trial are more that a patient receives. So, with a trial, let’s say that there’s more than one way that someone can be treated. Let’s say arm A is drug X, and arm B is drug X plus drug Y, where drug Y is the experimental drug, and drug X is our standard of care. The reason they are double-blinded is that if the physician or the patient know exactly which arm they’re on, that might have an influence in terms of the physician assessing the response of the patient, the patient filling out questionnaires regarding response in symptoms.

It’s natural for us if we’re on a drug to say, “Oh, wow, you know, I automatically feel better. I’m excited about being on this,” and lead to what we call a bit of a placebo effect. Where there have been studies in the past where someone got a placebo but believed they were already feeling better even though nothing different had occurred. So, that they had somewhat kind of convinced themselves they were going to be doing better. So, the intent is, again, just to get the most objective set of response both from the physician and from the patient.

Katherine:                  

What is a randomized clinical trial?

Dr. Mesa:                   

A randomized trial means that when there is more than one arm, which treatment that a patient receives is random, is not chosen by the physician. Why that is the case is, again, we truly want to see which approach is better.

If the physician got to choose, they may inadvertently put all the sicker patients on one arm, or put all the less sick patients on one arm. In either case, it would make the value of the clinical trial less.

The value of the clinical trial, the entire reason we do them, is to try to, in the best way that we can, figure out which approach was better, whether that’s a treatment for your MPN, whether that’s figuring out whether a COVID vaccine helps to prevent to COVID, whether it’s figuring out whether a cholesterol lowering medicine is a good medicine to be on. Regardless of the reason, we want to know, is it the right way to go?

Because after that, there will be a lot of people who receive that treatment.

Katherine:                  

And finally, what is a controlled clinical trial?

Dr. Mesa:                   

A controlled clinical trial is, again – is following these same pieces where it has a comparator, where that comparator arm is sometimes also called the control, meaning that’s kind of the baseline – and, again, you’re looking to see, does that make a difference a baseline. So, let me use an MPN analogy. When ruxolitinib or Jakafi was first tested, there were no approved drugs for myelofibrosis.

So, how that worked – it was a controlled study. There was randomized placebo control. Half the group got ruxolitinib, half got placebo. After 24 weeks, people could then go on to get the ruxolitinib.

So, everyone eventually got the ruxolitinib. But, for those 24 weeks, we were able to compare what did the standard of care, which was really nothing, against ruxolitinib and saw a dramatic benefit. Now, the newer trials, now that ruxolitinib (Jakafi) is approved, ruxolitinib has been the control.

So, when there was a ruxolitinib and momelotinib trial to see if momelotinib was an effective drug, it was compared against ruxolitinib. Now, it was blinded, so that you didn’t know which of the two that you were on, but people were getting an active control. So, that is an active-controlled trial versus a placebo-controlled trial where the comparator is placebo.

Katherine:                  

What is an observational study, and how does that differ from the other clinical trials?

Dr. Mesa:                   

An observational study, as the name might suggest, is, again, where you’re observing a group of individuals, whether they start on a treatment, whether you’re trying to see how the disease behaves over a period of time.

But what it typically does not do is that you are intervening in a very specific sort of way where you are again changing how people otherwise would have been treated.

Clinical Trials As an MPN Treatment Option: What You Should Know

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Clinical Trials As an MPN Treatment Option: What You Should Know from Patient Empowerment Network on Vimeo.

 Should you consider an MPN clinical trial? Dr. Ruben Mesa provides an overview of clinical trials—including the phases—and defines common trial terms and types. Dr. Mesa shares advice on trial participation and gives an update on the latest advances in MPN research.

Dr. Ruben Mesa is an international expert in the research and care of patients with myeloproliferative neoplasms (MPNs). He serves as executive director of UT Health San Antonio MD Anderson Cancer Center in San Antonio, Texas. More about this expert, here.

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Transcript:

Katherine:                  

Hello and welcome. I’m Katherine Banwell, your host for today’s program. Today, we’re going to discuss research advances in myeloproliferative neoplasms, or MPNs, and review key information that patients should know about clinical trial participation.

Before we get into the discussion, please remember that this program is not a substitute for seeking medical advice. Please refer to your healthcare team about what might be best for you. Well, let’s meet our guest today. Joining me is Dr. Ruben Mesa. Dr. Mesa, welcome. Would you please introduce yourself?

Dr. Mesa:                   

Thank you so much. It’s a pleasure to be here. I’m Ruben Mesa. I’m the executive director of the Mays Cancer Center at UT Health San Antonio MD Anderson.

Katherine:                  

Great. Thank you so much for joining us today. As we move through this conversation, we’ll talk about the classic myeloproliferative neoplasms, essential thrombocythemia, polycythemia vera, and myelofibrosis. So, before we dive deeper into our clinical trial discussion, let’s talk about research advances. What are the latest developments in the field of myeloproliferative neoplasms?

Dr. Mesa:                   

There are many advances that are very important for patients to know about. First, we’re learning more about the biology of these diseases. Why do they occur? Why do they progress? Why are they different in different individuals? Indeed, the course of these diseases can be quite variable. So, these important pieces of biology are important for us to be able to better diagnose the disease, monitor the disease, and develop better therapies.

Second, I would say that there are many important new therapies that are in development. They are only able to be developed into therapies that patients can use by the process of undergoing through clinical trials. But these therapies are for patients with essential thrombocythemia, polycythemia vera, and myelofibrosis. So, a critical part, but many different scientific advances that are important and hopefully will have an important impact for patients with MPNs.

Katherine:                  

Mm-hmm. It sounds very promising. And, of course, these research advances wouldn’t be possible without patients actually participating in clinical trials. So, can you help us understand more about the trials? What is a clinical trial? Let’s start with that.

Dr. Mesa:                   

It’s a very good question. A clinical trial is a very structured way for us to be able to ask a question, whether that question is, is a new therapy safe?

Is it effective for a particular disease? Sometimes there are clinical trials that don’t involve treatments, that involve questionnaires, or other interventions, things like exercise or yoga or other things. But in general, it’s where we are having a patient do something in a structured way that we are able to then assess. Is it safe and is it effective? Is it reaching our goal in terms of trying to have an impact on that disease, whatever that is?

So, if it’s a blood pressure medicine, it’s probably about lowering the blood pressure. If it was about the COVID vaccines, did the vaccines help people from developing COVID or make COVID less severe? So, what they’re testing is variable. But the concept is the same.

It’s a very organized way for patients to be able to receive something that is closely monitored, that has been approved in advance as being a reasonable, safe, and ethical to ask patients to participate.

Katherine:                  

What are the phases of clinical trials?

Dr. Mesa:                   

So, the phases are particularly to treatment or drug use trials for developing new therapies. And they start with Phase I, which is typically the first time a drug is tested in human beings. It’s already gone testing in the lab to see whether it should work. It likely has had some animal testing to get a sense of dose and safety. But then, the first individuals who receive the drug, it’s on Phase I. What we’re really trying to understand the safety of the drug and to try to get around the dose.

There is the Phase II, typically where we’re testing a therapy in a group of people that are all similar to see what is the effectiveness of the treatment.

So, that first phase is, is the drug safe? What is the dose? The second phase is, is the drug effective? and however we define effective for that particular disease. And then, the third phase is where that new treatment is compared against how we otherwise normally would have treated the disease. So, if that’s in the setting of where we already have a drug that is approved, it’ll be compared against that drug.

If there’s never been a drug, then that comparator could possibly be a placebo, or an inactive part, or observation, or sometimes best alternative therapy the doctors can use.

There is, finally, a fourth phase. There are times that, after a drug is approved, the FDA will ask for additional information – safety information, effectiveness information – even after approval, and that’s something referred to as the fourth phase.

Katherine:                  

Let’s move on to some common language used around clinical trials. I’ll mention a few and then maybe you could define them for the audience. The first one is, informed consent. What does that mean?

Dr. Mesa:                   

Informed consent is that what is involved with participating with a trial, what the drug is, what do we know about its safety, what you might anticipate, either in terms of side effects, whether a likely side effect or a rare side effect.

And what’s involved with you in terms of a participation, whom to call if there’s an issue. It really is an extensive document. It looks like a contract, but actually it is not.

So, informed consent is not an obligation to participate in the trial, nor does it mean that you have to stay on the trial for any length of time.

It is truly to inform you, and then you sign it, saying that you have been informed. One important concept: in the clinical trial, you are always in the driver’s seat, so that if you take – you sign the consent and you choose not to participate, you’re done then and there. If you take one dose and you don’t want to take anymore – fine. So, you’re always in the driver’s seat. It looks like a contract; it clearly is not. It is not an obligation from your side.

Katherine:                  

That’s good to know. What about standard of care?

Dr. Mesa:                   

So, standard of care is the medical language we use to how we would treat you otherwise. So, a clinical trial, by definition, is we’re trying something new.

Sometimes it’s a drug that’s never been approved, but sometimes it’s a drug that’s approved that we’re testing in a different way. Standard of care is kind of the default care that you would normally receive anyway, that is kind of the medical standard for your particular condition.

Katherine:                  

What does adverse event mean?

Dr. Mesa:                   

An adverse event means any possible side effect or event, a hospitalization or something of that nature. And then, the doctor typically attributes whether it’s related or unrelated. So, let me use an example.

If you’re on a clinical trial with a drug, but you go skiing; you fall and you break your ankle. That is an adverse event. You were hospitalized and had a broken ankle during the conduct of the study.

Now, it likely is not attributed to the trial drug, and that’ll be discussed and investigated.

But maybe it was. Maybe you felt light-headed and you passed out because of the drug you were on and you were skiing. So, again, that is a determination that your doctor makes about an adverse event. But it’s an adverse event whether it’s related to the drug or whether it has nothing to do with the drug.

Katherine:                  

And what about HIPAA? What does that mean?

Dr. Mesa:                   

HIPAA relates to – and I forget the full acronym – but really, it’s around the integrity of your patient information and that that is not able to be disclosed in a way that is either harmful to you or to individuals that really are not authorized to receive that information, which typically includes your treating team with permission – if you give permission to another healthcare provider or system, to your insurance company, et cetera.

But it’s really around both portability, I believe, in terms of your patient record, but also in terms of privacy.

Katherine:                  

A big concern for patients who may be considering participating in a clinical trial is fear that they will receive a placebo. Can you define what a placebo is for the audience?

Dr. Mesa:                   

So, a placebo means a drug that is inert. So, historically, a placebo has been, let’s say, like a sugar pill.

So, one, it is a very small minority of trials in this day and age that have a placebo. So, one, it’s almost solely in the setting of a Phase III trial. So, in a Phase I trial, everyone gets the drug. In a Phase II trial, typically everyone gets the drug. In a Phase III trial, there is typically something that it is compared against.

Now, if there’s a standard of care approach, that’s likely the comparison group.

Now, the group that starts with standard of care may well then have a period where they “crossover,” where they are treated in one way for a certain amount of time, and then get kind of the drug in question. A placebo is truly meant to be the same as kind of getting nothing. Now, in a disease like MPN, the number of placebo control trials is really very few. Sometimes a situation that they are used is where the comparator is, let’s say, trying to use two drugs – so, let’s say, the standard of care plus a new drug – versus the standard of care alone.

Now, sometimes people will take both the standard of care and a placebo so that they are, what we call, blinded. So, they don’t know which treatment arm they were on. They’re still getting treatment. They’re still getting the treatment that they would’ve anyway, but they don’t get two treatments. So, the second part is a placebo.

But anything like this, one – any trial a doctor refers you to, one should fully understand exactly how the trial works. Is it a trial with a placebo? Is it not? And then, allow that to help kinda inform your own consideration.

Is this something that I’m willing to do? Does it make sense? Is there a different approach? You know, is there a different trial that does not involve a placebo? So, I think, as physicians, we clearly understand that we try to absolutely minimize the situation where placebos are used. And when they are used, they are only used in a way that we feel that no one is getting less than at least the standard of care therapy that they would otherwise.

You know, it is unethical for there to be a placebo that really would deny patient a therapy that we otherwise know would be helpful.           

Katherine:                  

You touched upon the various types of clinical trials and I would like to look at each one of them individually. So, let’s start with the double-blind clinical trial. What is that?

Dr. Mesa:                   

What that refers to is that neither the patient nor the physician knows which of the two comparator arms of the trial are more that a patient receives. So, with a trial, let’s say that there’s more than one way that someone can be treated. Let’s say arm A is drug X, and arm B is drug X plus drug Y, where drug Y is the experimental drug, and drug X is our standard of care. The reason they are double-blinded is that if the physician or the patient know exactly which arm they’re on, that might have an influence in terms of the physician assessing the response of the patient, the patient filling out questionnaires regarding response in symptoms.

It’s natural for us if we’re on a drug to say, “Oh, wow, you know, I automatically feel better. I’m excited about being on this,” and lead to what we call a bit of a placebo effect. Where there have been studies in the past where someone got a placebo but believed they were already feeling better even though nothing different had occurred. So, that they had somewhat kind of convinced themselves they were going to be doing better. So, the intent is, again, just to get the most objective set of response both from the physician and from the patient.

Katherine:                  

What is a randomized clinical trial?

Dr. Mesa:                   

A randomized trial means that when there is more than one arm, which treatment that a patient receives is random, is not chosen by the physician. Why that is the case is, again, we truly want to see which approach is better.

If the physician got to choose, they may inadvertently put all the sicker patients on one arm, or put all the less sick patients on one arm. In either case, it would make the value of the clinical trial less.

The value of the clinical trial, the entire reason we do them, is to try to, in the best way that we can, figure out which approach was better, whether that’s a treatment for your MPN, whether that’s figuring out whether a COVID vaccine helps to prevent to COVID, whether it’s figuring out whether a cholesterol lowering medicine is a good medicine to be on. Regardless of the reason, we want to know, is it the right way to go?

Because after that, there will be a lot of people who receive that treatment.

Katherine:                  

And finally, what is a controlled clinical trial?

Dr. Mesa:                   

A controlled clinical trial is, again – is following these same pieces where it has a comparator, where that comparator arm is sometimes also called the control, meaning that’s kind of the baseline – and, again, you’re looking to see, does that make a difference a baseline. So, let me use an MPN analogy. When ruxolitinib or Jakafi was first tested, there were no approved drugs for myelofibrosis.

So, how that worked – it was a controlled study. There was randomized placebo control. Half the group got ruxolitinib, half got placebo. After 24 weeks, people could then go on to get the ruxolitinib.

So, everyone eventually got the ruxolitinib. But, for those 24 weeks, we were able to compare what did the standard of care, which was really nothing, against ruxolitinib and saw a dramatic benefit. Now, the newer trials, now that ruxolitinib (Jakafi) is approved, ruxolitinib has been the control.

So, when there was a ruxolitinib and momelotinib trial to see if momelotinib was an effective drug, it was compared against ruxolitinib. Now, it was blinded, so that you didn’t know which of the two that you were on, but people were getting an active control. So, that is an active-controlled trial versus a placebo-controlled trial where the comparator is placebo.

Katherine:                  

What is an observational study, and how does that differ from the other clinical trials?

Dr. Mesa:                   

An observational study, as the name might suggest, is, again, where you’re observing a group of individuals, whether they start on a treatment, whether you’re trying to see how the disease behaves over a period of time.

But what it typically does not do is that you are intervening in a very specific sort of way where you are again changing how people otherwise would have been treated.

Katherine:                  

Are there other common terms that you think patients should understand and know about?

Dr. Mesa:                   

As you relate to adverse events, sometimes you would hear the term, serious adverse event, and this is sometimes to separate whether, again, as the name suggests, they are serious – and by serious, that sometimes has a threshold of requiring hospitalization, requiring a visit to the ED, emergency department – to potentially being life-threatening. Now sometimes these are associated with the disease or the medication. Sometimes, they’re unrelated. But these are ones we’re particularly sensitive of.

Again, as one looks at side effects of therapies, you’ll look at an informed consent and typically it will be a fairly long list of possible things. A relatively short list of things that we expect might happen being likely to occur, maybe can occur in greater than 20 percent of people, and sometimes some really rare things that are less likely to occur. But we also look at – when we look at a trial and look at all of the side effects that people had – were they related, were they unrelated, and were they potentially serious or not?

Katherine:                  

Let’s talk about safety. What are the risks of a clinical trial participation?

Dr. Mesa:                   

So, clinical trials are structured to try to have the safety be front and center in terms of caring for patients.

So, depending upon the therapy and how much is known about that therapy will dictate the frequency in which the patient is observed. If there’s specific side effects, how are we monitoring for those side effects so that, if they are starting to occur, we can discontinue it, discontinue the drug, lower the dose, etc. So, there are some times we do accept as patients and as physicians some potential new side effects in the hope that a therapy might be more effective against the disease.

So, if it might irritate the eye, do we have eyes exams? If it might cause the heart rhythm to be abnormal in some way, do we monitor electrocardiograms? If it might cause rash, do we have exams at a certain frequency to assess for rash? Is there more blood count tests done to assess for changes in the blood counts, irritation in the liver or kidney?

So, depending upon how the drug might impact someone, it really helps to dictate what monitoring is occurring in the conduct of the study to monitor for side effects.

And then there will always be a very specific plan. Well, if a side effect occurs, what do we do? Is the drug stopped? Is the dose lowered? If it’s stopped, how long do we stop it? – usually until that side effect has recovered. And then, do we restart the drug? And, if so, do we restart it at the same dose or at a dose reduction? So, a clinical trial is guided by something that is called a protocol, which is kind of the long recipe book for exactly how that trial will work and will detail all of these things so that it can be done in a thoughtful way, but also in a consistent way, across institutions.

Katherine:                  

Mm-hmm. Well, that leads me to the next question. I’m curious to know what protocols are in place to protect patients?

Dr. Mesa:                   

So, it depends very much by each clinical trial.

There are specific protocols in that any clinical trial that is developed needs to be reviewed and approved at multiple levels through an institutional review board, which is in ethics or specifically focus on clinical trials for an institution or sometimes for a broader group. There are times that there’s additional regulatory oversight from the FDA, from the National Cancer Institute, cooperative groups, and others.

So, there’s really an entire network of things put in place. Mandatory training for physicians, nurses, and staff in terms of good clinical practices in the conduct of the study. There are specific safeguards in terms of the handling of the drugs. The pharmacist, and other safeguards in terms of you receive the drug that you’re intended to receive at the right dose, made in the right way.

Everything is heavily focused in medical practice anyway on patient safety, but you can imagine that in the conduct of a clinical trial that’s taken really to the next level in terms of trying to provide every safeguard for the patient.

Katherine:                  

Dr. Mesa, let’s move on to participation. How can someone find out about what trials are available to them?

Dr. Mesa:                   

So, first and foremost, it begins with a conversation with your physician. And overall, clinical trials – the majority of clinical trials are in situations where things are not going perfectly. You know, if you’re doing well, you’re feeling well, you’re doctors a hundred percent happy with how you’re doing, then a clinical trial may or may not be an option. They are usually in a situation where things are not going as well as we would like. You have residual symptoms; you’ve only had a partial response.

If the current medicines for the disease don’t agree with you, you had side effects, or others. Now additional research for learning about these trials include many different organizations. There are disease-specific ones, like the MPN Research Foundation, MPN advocacy & Education International, MPN Hub, amongst many others. There is the broader, clinicaltrials.gov. Now, that’s a very broad site.

It is searchable. Sometimes it gives you more information than is helpful, but most things are listed on there. The Leukemia and Lymphoma Society, at LLS.org, has a specific kind of navigation function that they have for learning more about clinical trials and getting matched up with them.

But it truly starts with you and your doctor. If things aren’t working well, what are the options that I have? Is it a different option in terms of therapy? Or, if not, asking about clinical trials because clinical trials, again, will have their own upsides and downsides you and doctor will go through depending upon your situation.

Katherine:                  

What are the barriers to accessing clinical trials? Are there any?

Dr. Mesa:                   

So, first, clinical trials have, kind of, the broader logistics barriers. Frequently, you need to enroll and participate at a particular site and sometimes that site is not locally. Your doctor may or may not be participating in that trial. Some trials are only done at a single institution. So, for many, there can be a hassle factor.

You know, it’s impractical for me to be there, or be there for the frequency of visits or other pieces. So, that is one potential barrier. Overall, we hope that insurance or other coverage is not a barrier. In general, clinical trials are structured in a way to hopefully have them be financially neutral for patients.

It’s not less expensive to get your care if you’re on a trial, but it shouldn’t be any more expensive because the standard of care items are billed to your insurance as they would be normally. But if there are things that are experimental, they are included as an expense of running the trial and you’re not charged for those. Now the other barrier is, specifically, trials tend to have a specific set of eligibility for participation that are medical. It may be in a subset of patients based on any number of factors.

And there may be other limiters in terms of prior health conditions, sometimes in terms of age, sometimes in terms of how well the heart, the liver, the kidneys, or other things work. There’s both kind of a logistical piece, but then there is very specific eligibility. As a researcher, when a patient is a candidate for a clinical trial, I will have to go point by point, and sometimes there might be 50 points of disease, blood tests, and organ function – other pieces that need to be correct for participation in that trial.

It’s not to say that drug may not conceivably help that individual. It’s to say that for that specific trial, that’s what’s needed to participate in that very specific clinical trial.

So, sometimes that can lead to a bit of frustration, but it’s critical so that that trial is comparing the right group of patients so that the safety is really as great as the safety can be in the conduct of that study.

Katherine:                  

Right. What sort of questions should patients be asking their healthcare team about participating in a clinical trial?

Dr. Mesa:                   

Well, I think this discussion acts as a nice framework. So first, why should I participate in this clinical trial? Meaning, what is it about my disease that makes a different treatment option a consideration? So, why to begin with? And, if so, why this trial? What drug is it? Why does it help? If it was successful, what can I expect?

Then, what is entailed with me to participate? How frequently do I need to come? What’s involved? Is there more expenses that I can anticipate?

Again, in general, I can hopefully say no. But, of course, if you’re having to fly once a month, that, in some trials, may be covered as an expense of the trial and you’re reimbursed, but it may not. So, again, I think it starts with, medically, why does it make sense? What is involved for me? And then, really, what are those other next steps? And then, what are the alternatives? Sometimes there’s more than one clinical trial as an alternative. Sometimes there’s other options that are not a clinical trial that are an alternative to consider as well.

Katherine:                  

Before we end the program, Dr. Mesa, I’d like to get your thoughts. What message would you like to leave the audience with related to clinical trial participation?

Dr. Mesa:                   

Clinical trials are essential.

They are really the only way that we make progress in terms of developing new treatments. In the United States, less than 10 percent of patients with diseases like MPNs and cancers participate in clinical trials. And, to be honest, this really slows our ability to develop new therapies that would benefit folks. These are a very important resource.

I’ll flip it around another way – in children, where, again, we want to do everything that we can – about 80 to 90 percent of children are treated in the conduct of a clinical trial, where, again, they’re constantly pushing the envelope to try to develop better therapies.

And because of that, I think our progress comparatively, in childhood cancers, has been much faster in developing therapies than it has been in adults. So, it’s critical. It’s an opportunity.

Again, it’s very much a personal decision, but it’s something that I would strongly encourage you to consider. Again, one can begin and you are not obligated to remain on if that clinical does not, in the end, end up having the benefit that you had hoped, or if it ends up having a side effect that you prefer to not experience.

Katherine:                  

Dr. Mesa, thank you so much for joining us today. It’s been a pleasure.

Dr. Mesa:                   

Wonderful. Thank you so much for including me.

Katherine:                  

And thank you to all of our partners. To learn more about MPNs and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell. Thanks for being with us.