Tag Archive for: MPN Research

How Molecular Markers Affect MPN Treatment | Advances in Research

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How Molecular Markers Affect MPN Treatment | Advances in Research from Patient Empowerment Network on Vimeo.

Are there new molecular markers being discovered that could affect myeloproliferative neoplasm (MPN) care? Dr. Lucia Masarova explains common MPN driver mutations and what researchers are learning about recently discovered molecular markers, such as ASXL1.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.

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 Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

 Expert Perspective | Disease Modification in Polycythemia Vera

Expert Perspective | Disease Modification in Polycythemia Vera

Transcript:

Katherine Banwell:

Dr. Masarova, molecular testing is important for people diagnosed with MPNs and may help provide insight into effective treatment approaches. What are some new areas of research related to molecular markers? 

Dr. Lucia Masarova:

Molecular markers are very relevant in our designs or thinking about myeloproliferative neoplasms. Not only treatments, but also the disease qualification or prognostication wherever since the discovery of the so-called driver mutations, which are the mutations responsible for the overproduction of the blood counts and disease pathogenesis.  

Among them we have the most common, JAK2 mutation, then also calreticulin, MPL, or in some instances we don’t even understand and call it triple-negative. 

There we have learned, over the years, that the amount of the expression, or allele burden, does correlate with the disease behavior outcome. And then our ability to reverse that. So, a chief decrease of the burden is also relevant to the outcome of the patients. So, developing therapies or even putting these as an endpoint for clinical trials is important for our decision-making and moving towards eradication of the disease.  

Then there are additional molecular changes, which include non-drivers, which are additional mutations that we have learned and even implemented in the latest prognostic models, some of them are very unfavorable, such as ASXL1, Ezh2, IDH mutations, certain splicing factors.  

And those play additional roles, a lot of it we still do not understand, in how the disease is going to ultimately behave. What is their interplay, and how we can interfere with that?  

So, learning about the impact of these mutations and the drivers and the other effects that cause the disease evolution will probably become the landmark of this decade and in facing myeloproliferative neoplasms. 

And I’m hoping we will develop medications, or we will be able to focus our efforts and our decision-making based on molecular definition, as it’s currently very broadly seen across all cancers. We call it precision medicine where we really define, “How does this look like,” not how we box it in based on morphology. What is it driving? What is it not responding? And what can we do to improve that?  

So, I totally see here a big potent and powerful tool to allow us to make the most individualized and customized decisions for our patients to offer them the best outcomes.  

Expert Perspective | Disease Modification in Polycythemia Vera

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Expert Perspective | Disease Modification in Polycythemia Vera from Patient Empowerment Network on Vimeo.

Is it possible to change the course of disease in polycythemia vera patients? MPN specialist and researcher Dr. Lucia Masarova shares an overview of the research in disease modification, discussing her work as the coauthor in an article entitled Moving Towards Disease Modification in Polycythemia Vera, recently published in the journal Blood.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova. 

 

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 Myeloproliferative Neoplasm News and Research Updates

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 How Molecular Markers Affect MPN Treatment | Advances in Research

How Molecular Markers Affect MPN Treatment | Advances in Research

Transcript:

Katherine Banwell:

Dr. Masarova, you are a coauthor in an article entitled Moving Towards Disease Modification in Polycythemia Vera, which was recently published in the journal Blood. Can you share some of the highlights of the article and what it means for PV patients? 

Dr. Lucia Masarova:

Disease modification in polycythemia vera. I’m so excited finally being talking about this because we’ve been really, really, really so hungry for this term, although we still don’t know what it means.  

So, we group together with lots of experts in the myeloproliferative neoplasm field and try to brainstorm and put together, “What does it actually mean?” And to me, and to all of us, it was to offer our patients the normal or not-normal lifespan without the consequences of the disease that they face. Because we historically divided polycythemia vera into high-risk or low-risk disease based on the age or previous history of thrombosis or clotting complications.  

However, there is a huge area of patients that wouldn’t have either, and still suffer tremendously a bad quality of life, and ultimately also face the disease progression to myelofibrosis, which is the most actual complication of long-term polycythemia vera duration.  

So, the concept of disease modification would be to actually prevent the complications to even occur. To allow our patient to live free of having the fear of living with a thrombosis or clotting complication or ultimately progress into myelofibrosis. We have to learn how to get there. What are the relevant endpoints of tools for us to utilize to really understand? We have learned a lot from seeing what we call molecular remissions, or control of the JAK2 mutation with certain medications, for example, interferons or latest ruxolitinib (Jakafi), the JAK inhibition, where the decrease of the allele burden, which represents the disease, is correlated with better outcome.  

So, that is something that we have to be learning down the road with a longer follow-up. But that basically triggered us to focus on what can we do better? How do we prevent this from even happening rather than only controlling the historically main points of the disease which are presented by the blood counts symptoms and display? And where we are actually failing quite a lot of patients because despite them having a control count, they still don’t have a happy life, and lots of them suffer and complain.  

So, this is something to be learned, and this is opening the disease modification not only for polycythemia vera, but also for all patients with myeloproliferative neoplasms, which have a little bit of a different feeling in the whole myeloid malignancies field. Because it is a very long disease, and it could evolve and change, and only now we starting to understand what does actually happen there. Why some people could live for 30 years, and never face any consequences, and the others would progress very fast? 

So, disease modification would normally allow us to develop and learn more tools and better biomarkers, but also focus on drugs that are really needed to help with these long-term outcomes of our patients.  

Myeloproliferative Neoplasm News and Research Updates

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Myeloproliferative Neoplasm News and Research Updates from Patient Empowerment Network on Vimeo.

Dr. Lucia Masarova, a myeloproliferative neoplasm (MPN) specialist and researcher, discusses the latest updates from a recent MPN Congress. Some of the highlights include new learnings in hematopoiesis, JAK inhibitor comparisons, interferon therapy, and the potential for combination treatments in the future.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.

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 Advances in Research | Emerging MPN Therapies on the Horizon

Advances in Research | Emerging MPN Therapies on the Horizon 

Transcript:

Katherine Banwell:

Dr. Masarova, you were in New York recently for the MPN congress. Can you share some highlights from that meeting? 

Dr. Lucia Masarova:

Yeah. Sure. That was a very interesting and very loaded conference full of experts and great data. I really liked the overall excellent update of all the therapies that currently exist in the MPN space, including polycythemia vera, essential thrombocythemia, myelofibrosis. So, really a broad breadth of JAK inhibitors, their current sequencing, combinations, interferon update. I very much like also the focus of the novel therapies, which actually talked about, for example, the development of the antibody against mutant calreticulin, PIM inhibitors, and a couple others. They are very promising in the space. 

There were also very, very relevant clinical data. I think I really, really enjoyed the radiation in hematopoiesis topic. It spurred lots of discussions in the room. And also, fantastic talks about clonal hematopoiesis and its role in patients with myeloproliferative neoplasms and cancers.  

Overall, very great data on artificial intelligence because that will be a very needed tool, but also a very worrisome tool, at this point, until you learn how to use it to help our patients. But that showed a very promising effect and ability for us to, for example, predict thrombosis risk in polycythemia vera patients or to distinguish patients with ET versus prefibrotic myelofibrosis, which is still subject to lots of basically subjective analysis from hematopathologist.  

And also, the poster section was quite striking and really excellent. You could walk around and see so many interesting data. The match and direct comparisons of JAK inhibitors, particularly the latest approved, momelotinib (Ojjaara), as it compared to safety data which do currently exist in fedratinib (Inrebic) or pacritinib (Vonjo). 

Katherine Banwell:

When it comes to MPN research and emerging treatment options, what are you excited about specifically? 

Dr. Lucia Masarova:

There is a lot of excitement in the field currently, and it really depends how we put these patients in, as I would call, boxes, but I don’t like the term. We have these less aggressive diseases, such as polycythemia vera and essential thrombocythemia.  

Where I’m really excited about the role of interferon, with the approval of ropeginterferon (Besremi), or ropeginterferon in United States as well as Europe, we have opened a door for learning how can we do better.  

It is approved for polycythemia vera patients. There are currently clinical trials running in essential thrombocythemia patients, within patients with prefibrotic myelofibrosis. That’s an agent that has an ability to go after the disease clone and hopefully, hopefully eradicate or prevent it. Especially, especially exciting in the terms of preventing it for progression.  

Then iron metabolize modifier, hepcidin mimetics, other agents impacting this. It’s very important we finally learn how iron plays the role in these patients and how we can actually improve. Very important area in helping patients requiring phlebotomies and hopefully, hopefully altering the whole disease outcome in the long-term. 

For myelofibrosis we live in an era of JAK inhibitors. We are so excited to have four currently approved and we’re looking forward to the combinations where we have now safer and less cytopenic agents that have a role in anemia or thrombocytopenia and hopefully will be able to be combined with others. 

So, we could even move the field more into other hematologic malignancies, where in myeloma we use five, six, seven, eight drugs. For myelofibrosis, we still have one. So, I think we have still a lot to do. 

And then non-JAK inhibitor combination. Non-JAK inhibitor, a compounds or mechanism of action really tailored to the disease pathogenesis. Calreticulin, excellent topic, which I’m saying maybe in couple years we will be really classifying myeloproliferative neoplasm calreticulin-mutated, but also JAK2-mutated, and we will not be calling them one because hopefully we will find a tool to eradicate calreticulin and to really be able to offer ultimate – what I call ultimate cure.  

So, that’ll be something really exciting to come and all of these investigators in MPN fields are so eager to see what – whether the preclinical data we have seen are going to stand in our patients. And that would be really fantastic.   

Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask

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Choosing a Myelofibrosis Treatment Plan | Key Questions to Ask from Patient Empowerment Network on Vimeo.

When considering therapy for myelofibrosis, where do you start? Dr. Lucia Masarova shares advice and key questions to ask your provider when making myelofibrosis treatment decisions.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.

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 Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

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Transcript:

Katherine Banwell:

When considering treatment options, what key questions should patients ask about their proposed treatment plan? 

Dr. Lucia Masarova:

What’s the goal of my therapy? That is one of the most important things to know. Patients don’t even know how long they have to be on the medicines. What to do and how does it look when the medicine is still working? What do I need to be looking for in this medicine? And then what are we going to do if it fails? And what does it actually mean when it fails? What is the schedule? How burdensome the treatment is? How often do I have to come?  

How often and what do I have to pay? Because the financial burden we have to really, really face the truth. It is very, very, very significant and somebody living with this disease predicates. It’s something we cannot take lightly, and we really have to combine our efforts and help with that. There are fantastic patient support organizations, but is not well-known, and is still in the rare – in rarer field. So, there’s more effort that we do. 

When do I need more help? Where to be referred to more experts? What is the role of stem cell transplantations, if ever? So, those are really the key things.  

Where do I find reliable resources to learn about my treatment, to learn about the disease? How do I connect with people from the same community? It is a disease with a lower age in a lot of circumstances and really facing this disease in the 30s or 40s or 50s is a really challenging thing. Although we have more and more medications currently, we really do have now to start thinking about their durability, about the safety for long-term, about their assessments for not performing, and where do we place the ultimate cure for stem cell transplants?  

And how do we make it actually happen in more and more eligible patients? Because we have to face the truth. It is still not utilized to where it belongs. Patients are not being referred. 

Patients are not being transplanted. And they may change with novel therapies. But we have to really consider all of our tools to offer the longest life span and to prevent all the disease trouble that comes with living with MPNs.   

Katherine Banwell:

When it comes to clinical trials, where do they fit in in choosing treatment? 

Dr. Lucia Masarova:

For me, it’s number one., and always number one.  

That’s just the academic centers which are dedicated and focused on developing better and novel and up front and just tailored and customized drugs. But I know that the life is out there and it’s a little bit more challenging for everybody to deal with such a rare disease.  

I would definitely say any patient that does not respond to current therapy in terms of uncontrolled symptoms or spleen, or other concerns should be referred and evaluated for participation in clinical trials. It is the only way we could understand what is driving that this is not responding and how could we help the best?   

For patients with myelofibrosis, which is the most aggressive myeloproliferative neoplasm, I would definitely put it in. If they are not doing well on number  one, JAK inhibitor, whatever is being used, they should be highly encouraged to be referred to centers and evaluated for clinical trials. 

We have been developing as others and own strategies to potentiate the benefit and efficacy of the current treatments, as well as agents in what we call salvage or refractory setting.  

However, I cannot emphasize enough to really focus on the first track that providers choose for their patients and utilize it to the best ability to avoid frequent or quick switching. Because in a salvage or  refractory setting we cannot offer the same benefit we could offer upfront. We are pushing the disease, maybe being less responsive, maybe more refractory, if we don’t handle the medication we have currently on the table to the best ability.  

Those are excellent medications, fantastic drugs, but there are shortcomings in each and every one of them. And we could do better to really start thinking about what has happened with the medication, why is it failing the patient, and what else could we do? And that’s only possible in the clinical trial setting, especially in such a rare disease as myeloproliferative neoplasms are.   

Katherine Banwell:

Why is it important for patients to feel like they have a voice in their treatment options? 

Dr. Lucia Masarova:

Because it’s about the patients. I would say, as I always say to my patients, “Nobody’s a better advocate for you than you.” I really, really, really like working with patients. They are educated. They understand where to find resources. They’re not afraid to ask. That challenges all of my team and everybody to really be engaged. They know when to notify me. Not to be quiet when they need something. And really raise their voice when something doesn’t work.  

Patients know their bodies more than anybody can. And no data, no boxes, no books can ever tell me how it actually is. It’s not by chance we have two ears to listen and one mouth to talk.  

So, we have to really listen what the patient has to say and take all the abilities, the resources, the knowledge, the capabilities to really make the best thing for the patients, because it is ultimately and only about that.  

Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

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Is Stem Cell Transplant the Only Curative Option for Myelofibrosis? from Patient Empowerment Network on Vimeo.

Is there a cure for myelofibrosis? Dr. Lucia Masarova explains the role of stem cell transplant for the treatment of myelofibrosis and reviews additional therapies for patients who do not qualify for the procedure, such as JAK inhibitor therapy.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.

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Transcript:

Katherine Banwell:

Dr. Masarova, stem cell transplant is sometimes recommended for people with myelofibrosis. Is this still the closest option to cure for those patients? 

Dr. Lucia Masarova:

I would say so, as much as we don’t like it. We would like to develop novel conservative, less aggressive, that we call procedures or drugs. Stem cell transplants still represent a long-term cure for patients that are eligible. 

Katherine Banwell:

What about for patients who don’t qualify for stem cell transplant? What are effective long-term treatments for them? 

Dr. Lucia Masarova:

That’s a very, very important question and topic. The key point here is the long-term because long-term is a little difficult term in conservative management of myeloproliferative neoplasm, particularly when it comes to myelofibrosis.  

With the development of JAK inhibitors, the longest experiences we have with the first one called ruxolitinib or Jakafi, we have seen prolonged outcomes in survival so patients could live longer than expected before.  

However, it’s not forever. So, that’s why we are trying to develop novel strategies where I see a lot of roles of combinations of JAK inhibitors and other correlative compounds, such as bromodomains inhibitors or hypomethylating agents or others that would affect the pathways that we are missing currently to cover with the JAK inhibition. And that ultimately leads to medication failures and patients being refractory and then having a shortened lifespan.  

So, I’m hoping we will develop something for long-term. Particularly promising a very, very interesting concept is with the calreticulin where we are developing monoclonal antibodies or vaccines because we have seen and discovered calreticulin driver to be a targetable thing that causes immunogenicity. 

But I do really hope that we will move forward with these discoveries and the JAK mutate or other drivers causing myeloproliferative neoplasms to offer long-term management.  

Myelofibrosis Therapies in Clinical Trials | BET Inhibitors

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Myelofibrosis Therapies in Clinical Trials | BET Inhibitors from Patient Empowerment Network on Vimeo.

What are BET inhibitors? Dr. Lucia Masarova, an MPN specialist and researcher, explains what BET inhibitors are and discusses the role these therapies may play in the treatment of myelofibrosis.

Dr. Lucia Masarova is an MPN Specialist and Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Masarova.

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 How Molecular Markers Affect MPN Treatment | Advances in Research

How Molecular Markers Affect MPN Treatment | Advances in Research

Transcript:

Katherine Banwell:

We’re starting to hear more about BET inhibitors. Could you explain what they are and how they work to treat myelofibrosis? 

Dr. Lucia Masarova:

BET inhibitors are abbreviations for bromodomain inhibition, which is a very relevant regulator of transcription factors that play a significant role for making the blood cells.  

So, just differentiation of red cells or platelets, as well as very significant role in cytokines regulation. We know that myelofibrosis is a disease that is defined by overactive JAK-STAT Pathway that ultimately leads to increased cytokines.  

However, there are other pathways that play a significant role, and one of the very major ones is NF-kB, where the BET inhibitor come in play because they target it and help us to decrease the cytokine load as well as alter the differentiational block that happens in the red cells or megakaryocytes or platelets in these patients. 

So, the combination of bromodomain inhibition, or even using it as a single agent on or after refractory I think is a very promising tool that excludes the only JAK inhibition that we’ve been developing for diseases and opens the door for combination strategies that we were so many years thinking through and trying to find out. 

This is really the most promising compound or way of altering the disease background that we can see.  

Myeloproliferative Neoplasm Financial and Care Resources

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Myeloproliferative Neoplasm Financial and Care Resources from Patient Empowerment Network on Vimeo.

Some myeloproliferative neoplasm (MPN) patients may run into care barriers, so what can be done to help them? Expert Natasha Johnson from Moffitt Cancer Center shares her perspective on how she assists MPN patients with resources and how healthcare providers can help in overcoming care barriers.

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“…Let the healthcare team know, especially the great players here that are helpful are the nurses involved in the care team and the pharmacist who know of these outside resources to provide financial assistance so patients can get medication.”

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Transcript:

Natasha Johnson: 

When I’m speaking to MPN patients or caregivers about barriers, I really try to provide resources. All it takes is a Google search to look up the MPN Foundation or many other organizations that provide education. They provide direction to reach MPN experts. There are ways to get ahold of them and see them. Visits can be done through Zoom. Some large cancer centers even do charity care, so patients can get in who do not have insurance, but I would provide those resources whether I’m seeing that patient or I’m in the community teaching or at a supportive care group. I think it’s very, very important that patients need to know that they can access care, and it’s by researching, doing some research and being active in that care.

Secondly, when I’m speaking to patients about issues with medication costs, which can be a great barrier, my activation tip to them again, would be to let the healthcare team know, especially the great players here that are helpful are the nurses involved in the care team and the pharmacist who know of these outside resources to provide financial assistance so patients can get medication.

It’s very important, don’t let it be a barrier or stop anything or stop treatment, but pursue that by making us aware and then we can help you. 

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What Do MPN Patients Need to Know About Clinical Trials?

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What Do MPN Patients Need to Know About Clinical Trials? from Patient Empowerment Network on Vimeo.

What is a clinical trial and how does patient participation work? This animated video provides an overview of clinical trials, the process, and details key steps for engaging in your care.

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Transcript:

Brian: 

Hi, I’m Brian and this is my doctor, Dr. Liu. 

I have myelofibrosis, which is a type of myeloproliferative neoplasm, or MPN as it’s commonly referred to. 

And when discussing therapy with my doctor. She suggested a clinical trial might be my best treatment option. Dr. Liu, can you please explain what a clinical trial is? 

Dr. Liu: 

Absolutely. In basic terms, a clinical trial is a research study that involves human volunteers. The goal of cancer clinical trials is to discover new and improved approaches to prevent or treat the disease.  

There are typically four phases of a trial. Each phase gathers valuable information about the treatment and helps measure its effectiveness1.  

But, Brian you had some hesitations at first, right? 

Brian:  

That’s right.  Although I trusted your advice, I definitely had questions about the process.  

One thing I worried about was safety. I asked Dr. Liu how I would be kept safe if I participated in a clinical trial. 

Dr. Liu: 

I hear this concern a lot from patients. But every trial has a strict safety protocol, with set guidelines in place to protect patients.  

Additionally, researchers must follow patient safety rules, which are regulated by the U.S Food & Drug Administration (the FDA). And an institutional review board (or IRB) regularly reviews a study at each study location. Plus, many trials are overseen by a group called a data and safety monitoring committee2. 

Finally, there is also a clinical trial team that can include healthcare team members like cancer researchers and oncology nurses. This team regularly observes the health of each participant, through each stage of the process. Because of this, trials often involve more testing and visits, which can be a bigger commitment for a patient. 

Brian: 

When Dr. Liu explained the safety measures, it helped me feel more confident in the process and comfortable about participating. So, then I asked which trial she would recommend for my individual disease.  

Dr. Liu: 

It’s important to note that every trial has “eligibility requirements” that include things like a patient’s age, health, and prior treatments they have received. These requirements determine whether a patient qualifies for a trial. 

We talked about which trials may be a good fit for Brian and discussed the pros and cons of each one. We also reviewed the logistical and financial impact the trials could have on his life.

Brian: 

That’s right. Dr. Liu also pointed out that patients can leave a trial at any time–for any reason– once they have enrolled.

Dr. Liu: 

That’s right—participation is completely voluntary. 

Brian: 

One of the main reasons that I joined a trial was because I wanted to find out if a treatment in development would be more effective in treating my cancer. AND I wanted to help advance research forward, so I could help the MPN community.  

Dr. Liu: 

Right, Brian. Patients may have different reasons for participating, but trials are the only way to develop and study new and improved treatments for MPNs. 

Now that you understand more about trials, how can you find out more? 

  • Start by asking your doctor if there are any trials that you are eligible for.  
  • If there is a trial that your team recommends, ask to discuss: 
  • The treatment approach used in the study and the purpose of the trial. 
  • The risks and benefits of participation. 
  • The financial costs, if any, and if there are assistance programs to help if you need it. 
  • The location of the trial and whether it can be coordinated with your local institution if it isn’t conveniently located—or if transportation is available. 
  • And, how often you will need to visit the trial site and how long the trial will last. 
  • Finally, continue to educate yourself, using resources like clinicaltrials.gov. 

Brian: 

We hope you found this information useful! Visit powerfulpatients.org/MPN to learn more about clinical trials and MPN research. 

Dr. Liu: 

Thanks for joining us! 

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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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.