Tag Archive for: MPN Research Foundation

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

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Dr. John Mascarenhas discusses the evolving landscape of myelofibrosis research, emphasizing the concerted effort among researchers, pharmaceutical companies, and advocacy organizations to advance care and treatment options for patients.

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

See More from Evolve Myelofibrosis

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Updates in Myelofibrosis Research From an Expert

Updates in Myelofibrosis Research From an Expert

 How Can You Learn More About Myelofibrosis Clinical Trials?

How Can You Learn More About Myelofibrosis Clinical Trials?

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Is Stem Cell Transplant the Only Curative Option for Myelofibrosis?

Transcript:

Katherine Banwell:

Is there anything you’d like to add about the evolution of myelofibrosis care? What are you excited about?  

Dr. John Mascarenhas:

I always make the comment they don’t feel rare to me because I see so many patients with myelofibrosis, and it’s what I do. But I recognize in the context of lung cancer, breast cancer, other more common cancers, these can be forgotten diseases. But what has been encouraging is, between the NIH funding, for example, our research consortium, which is really geared to translating the biology into clinical trials across the country.  

Pharmaceutical interest, which is essential to providing drugs and finance to run trials. Young investigators that are coming to the field that want to make a difference, institutions that continue to support the programs, and then foundations. Whether it’s MPN Research Foundation, Leukemia & Lymphoma Society, it really takes a village. And we’ve been working with the FDA to try to better understand how to develop trials that are meaningful that can get drugs approved and to the patients. 

So, it’s a concerted effort, and I’ve been enthusiastic. I remain optimistic that everyone is trying to do the same thing, achieve the same goal, and work together, and that’s the only way we’re going to be able to do it. 

Katherine Banwell:

Yeah. How long does it take for the FDA to approve a drug? 

Dr. John Mascarenhas:

Forever. It feels like forever. It’s a long process, and for patients, it can be quite frustrating, because there’s so many steps involved. From just the original, initial steps to allow you to introduce a drug into humans, there are IND-enabling studies, so there’s a lot that goes into this.  

And then the initial studies are safety studies. They often can take quite some time. There’s a lot of scrutiny on safety, obviously, because the FDA is really charging to making sure that we do no harm, that we maintain safety for patients, so that can take a long time. And then the ultimate testing and comparison to a control arm is essential to get a drug approved. 

So, we’re looking at a timeline that can easily be a decade from the time we have a great idea, and have a drug available to us, to the time that we can prove that through the different stages, and then demonstrate that to the FDA and negotiate what a label will look like. And that is both a time-consuming process, a very expensive process, and a laborious process, but obviously an important process. 

Katherine Banwell:

Yes, and as you say, so many new drugs and therapies have become available in the last 10 years or more that really have advanced myelofibrosis care enormously. 

How Can You Learn More About Myelofibrosis Clinical Trials?

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Dr. John Mascarenhas shares advice for patients looking to learn more about clinical trials starting with consulting a specialist. Dr. Mascarenhas also emphasizes key questions to ask, including a discussion of the benefits versus risks of participating in a potential trial. 

Dr. John Mascarenhas is Professor of Medicine at the Icahn School of Medicine at Mount Sinai (ISMMS) and the Director of the Adult Leukemia Program and Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai. Learn more about Dr. Mascarenhas.

See More from Evolve Myelofibrosis

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Myelofibrosis Clinical Trial Participation | How Does It Move Research Forward

Myelofibrosis Clinical Trial Participation | How Does It Move Research Forward?

 Considering a Myelofibrosis Clinical Trial? Questions You Should Ask

Considering a Myelofibrosis Clinical Trial? Questions You Should Ask

 Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

Expert Perspective | A Concerted Effort to Advance Myelofibrosis Care

Transcript:

Katherine Banwell:

What about clinical trials? How can patients learn more? 

Dr. John Mascarenhas:

Well, clinical trials is definitely a confusing area because there are many clinical trials, and some of them are relevant to some patients may be not relevant to other patients.  

So, I think two ways is, see someone who does this. So, not everyone has as their primary care provider hematologist an expert, or someone who’s dedicated to doing this. They may be in a practice where they’re in the community setting; it’s not reasonable for them to be seen at a tertiary care center. 

But if you can get there even for a consultation or an initial visit to get plugged in, I think it’s really critical to see someone who is really invested in this with research opportunities, and clinical trial availabilities. And then the other resource would be clinicaltrials.gov.  

You can go in there, you can put in your diagnosis myelofibrosis. You can even manipulate it for geography to understand what trials are in your area. 

But it is a good way of looking, just to understand what’s there. And then the MPN Research Foundation, which is very supportive of the patient community and engages them, is often a very good resource to go to, to either learn about trials or join webinars where physicians that do this will discuss, and I think that’s another excellent resource. 

Katherine Banwell:

What questions should patients ask their team about clinical trials?   

Dr. John Mascarenhas:

Well, are there clinical trials for what I have? Because again, they can be very particular to where you are in the disease process, what medications you are on, what your kidney function is, things that may really influence decision-making. So, are there clinical trials, what are the clinical trials evaluating, what should I expect from them, both potentially from a positive angle, but also from a negative. What are the known toxicities, what would be the time commitment? Trials are more involved, and they’re more involved for a reason. 

I actually personally think patients do better on clinical trials than standard of care. The reason why I think that is because they are more involved. There’s a lot more oversight and eyes on you. Not just a physician perspective, but maybe more importantly, from a research nurse and research coordinator perspective. There’s a lot of regulatory burden which translates, I think, to a lot of attention to safety and assessment for advocacy.  

So, I think understanding what that looks like at any given institution, and how it will affect the patient and their caregivers from a time perspective, and obviously toxicity. But also, what is this trial trying to achieve? Does it make sense for what I’m trying to achieve? 

Katherine Banwell:

I suppose another question might be where this trial is taking place? As a myelofibrosis patient and a caregiver, are we going to have to travel to get to this clinical trial? 

Dr. John Mascarenhas:

I think that’s a major obstacle for a lot of patients. We’re talking about a disease that typically affects people that are in there sixth, seventh, eighth decade of life. Patients don’t all live, as I’ve said, right around a cancer center or a tertiary care center, so travel, the logistics of it, the ability to have that support available. Sometimes it’s loved ones, and adult children having to take time off of work to be able to help in that process.  

It’s a lot, and I’m particularly sensitive to it, because I work in a metropolitan area, and I realize getting in and out is not easy. And a lot of times, these trials understand that, and they build into the trials stipends and support for transportation and/or lodging which helps. It’s not perfect, but it can help at least financially, and sometimes logistically, so I would definitely inquire about what those resources are.  

And sometimes foundations also help bridge the gaps for patients to help link them to trials and facilitate that. So, it’s a super important part of engaging in a trial. 

Katherine Banwell:

And who is on the health care team that might be able to answer questions like this? 

Dr. John Mascarenhas:

Well, for sure, the physician should be able to. But I think the most valuable resource often is the nurse and the nurse practitioner. There are usually research coordinators. These are often young people, but very bright young people, that are very invested in this that will sometimes show up at the clinic to talk to the patient or will be a phone resource that you could reach out to. So, it can really be, I think, three levels. The coordinator, the nurse or nurse practitioner, or physician assistant, and the physician. So, it really shouldn’t be one person, but a team of people that are available to you. 

Yolanda’s Story: My Path to a Myeloproliferative Neoplasm Diagnosis

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Yolanda’s Story: My Path to a Myeloproliferative Neoplasm Diagnosis from Patient Empowerment Network on Vimeo.

Latina essential thrombocythemia (ET) patient Yolanda had many symptoms before receiving her ultimate diagnosis. Watch as she shares the symptoms she experienced, her long path to diagnosis, and her lessons learned about patient empowerment.

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Transcript:

My name is Yolanda, and I was diagnosed in my mid-40s with essential thrombocythemia (ET), a myeloproliferative neoplasm. I’m a Latina woman, and my path to diagnosis took an extended time.

Thinking back on my journey, my symptoms began with severe headaches and dizziness that made it too difficult to finish my work. I also experienced debilitating fatigue that would either keep me in bed for a day, or I’d feel like my vision and thinking were in a fog. It all felt very strange, and I saw my doctor about the symptoms, but he prescribed antibiotics for an infection. Then later I felt numbness and tingling in my hands and feet and then pain in my abdomen. Finally, my doctor decided to run full blood work to see which levels might be abnormal, and that was followed with a bone marrow biopsy to further investigate.

When I finally received my diagnosis with essential thrombocythemia, I felt some relief but also a sinking feeling and dread of what might be ahead for me. I feel like one issue with getting diagnosed may have been that I looked healthy. Maybe my doctor would have ordered the blood work sooner if I didn’t look well. But I try to look forward rather than back. An MPN specialist was recommended to me, and he initially put me on low-dose aspirin.

Then I was prescribed hydroxyurea (Hydrea). I’ve been doing well and feel grateful to have treatment options. But if my disease progresses to a point where I need other options, I’ve already decided that I’ll consider participating in a clinical trial. I feel like I’ve been relatively lucky and want to share my cancer story to help others.

Some of the things I’ve learned on my MPN journey include:

  • Empower yourself by asking your doctors questions about your MPN and what to expect before, during, and after treatment.
  • Learn about clinical trial options. There may be programs that will help you with travel, lodging, and other uncovered expenses. And clinical trials may provide an option for your MPN if you’ve already used all other options.
  • You are the person in charge of your health. If you feel like something is wrong in your body, advocate for yourself. Ask for more testing to find out what is wrong.
  • Be careful about where you look for cancer information. Use credible sources like MPN Research Foundation, The Leukemia & Lymphoma Society, and Patient Empowerment Network.

These actions were key for staying on my path to empowerment.

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Are There Predictors That an MPN May Be Progressing?

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Are There Predictors That an MPN May Be Progressing? from Patient Empowerment Network on Vimeo.

Many people living with MPNs are worried about progression. Dr. Raajit Rampal talks about possible indicators and discusses ongoing research to learn more about disease progression. 

Dr. Raajit Rampal is a hematologist-oncologist specializing in the treatment of myeloproliferative neoplasms (MPNs) and leukemia at Memorial Sloan Kettering Cancer Center in New York City. Learn more about Dr. Rampal.
 
 

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MPN Essential Testing | How Results Impact Care & Treatment Options

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Understanding and Managing Common MPN Symptoms and Side Effects

Transcript:

Katherine Banwell:

How do you know when it’s time to change treatments? 

Dr. Raajit Rampal:

Well, I think really two things. One is if we aren’t meeting our goals like we just talked about. But the other aspect of that is if we are incurring toxicities that are just not tolerable to the patient and that’s a reason to change therapy always. 

Katherine Banwell:

Many patients, of course, worry about disease progression. Are there key predictors or tests for progression that patients should know about? 

Dr. Raajit Rampal:

This is a key area of investigation currently. I think one of the things that patients say to us so often when we meet them is what’s going to happen to me. And right now, we don’t have great prediction tools. We can say on a population level well, there is X percent of chance of progression at 15 years. That’s useful if you’re talking about a population. That’s not really useful if you’re talking to an individual. Because if I say to somebody there’s a 20 percent chance of your disease progressing to leukemia, it doesn’t really make a difference. That’s a meaningless statement because if you’re in the 20 percent who progress, it’s not a relevant statistic anymore. 

It’s sort of a binary thing. We’ve got to do better at developing this. This is something that the MPN Research Foundation is really heavily invested in in trying to identify predictive biomarkers. 

If we can do that, then perhaps what we can do is say to a patient this is really what we think your actual risk is. And then, the next step is asking the question if we intervene early, can we prevent that progression from occurring. So, that’s where I think we need to go. We aren’t there yet. 

Katherine Banwell:

What signs or symptoms do you look for that may indicate that the disease is progressing? 

Dr. Raajit Rampal:

The blood counts are often the canary in the coal mine regardless of the disease. They can tell us if ET or PV is progressing into MF or whether MF is progressing to more of a leukemic phase. Changes in symptoms sometimes can be a harbinger of disease progression. So, Patient 2, for example, is doing really well and now, he’s having drenching sweats and losing weight. So, those types of symptoms are a sign that physical findings is the size of the spleen if it’s increasing. All of those things together give us a hint about progression.  

Katherine Banwell:

Well, is there any way to prevent progression? 

Dr. Raajit Rampal:

That is the million dollar question. Again, that’s where we ultimately need to be. We want to be able to intervene to a point where patients don’t get that sick. It would be amazing if we’d come to the point where we can intervene early and nobody progresses to late stage MF. Nobody gets leukemia. And I think that’s a worthy goal. That’s not something that we should think is too lofty of a goal. That should be our ultimate goal here. And a number of groups are investigating this exact question. It’s complicated and it’s going to take time. But I think that’s a worthwhile investment. 

Resources for Accessing MPN Clinical Trials

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Resources for Accessing MPN Clinical Trials from Patient Empowerment Network on Vimeo.

What are credible resources for accessing MPN clinical trials? Dr. Angela Fleischman shares credible resources for MPN patients and advice for inquire about clinical trial participation.

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:

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.  

Is There MPN Research Underway to Help Understand Progression?

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Is There MPN Research Underway to Help Understand Progression? from Patient Empowerment Network on Vimeo.

How and why do MPNs progress? MPN specialist Dr. Joseph Scandura shares an update on research being done to better understand–and possibly prevent–disease progression.

Dr. Joseph Scandura is an Associate Professor of Medicine and Scientific Director of the Silver MPN Center at Weill Cornell Medicine. Learn more about Dr. Scandura.

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Transcript:

Katherine Banwell:

Is there research being done on MPN progression to understand how it happens or even prevent or slow progression? 

Dr. Scandura:

Yeah. There’s a lot. I think there is a – from both the sort of basic laboratory using animal models to try to understand what are the kind of systems that are involved in how these diseases change. What genes are involved? How do they talk to each other? You know, these are not cells that live in a vacuum, right? They live in a special microenvironment. What are the signals that crosstalk between the MPN cells, the MPN stem cells, and their microenvironment?  

And so, there’s a lot of research on that and the basic side of things. In humans, there’s a lot that has been done over the years in terms of trying to understand what are some of the genetic features of progression. And I think we’re beginning to get a little bit of a better understand of what are the non-genetic things that are associated with progression.  

I was part of an effort from the MPN Research Foundation and still am.  

They have what they call the Progression Network, where they tried to put together a number of investigators from really across the world to share ideas about the nature of progression and how we might look at studying this and understanding ways to prevent progression.  

I think we do have some drugs now that show some promise in terms of being able to prevent progression. I think interferons have shown this in polycythemia vera in terms of a promise for improved long-term outcomes and delayed risk progression. I think that the gold standard randomized trials are maturing and are sort of bearing out some of the same findings that have been observed retrospectively, so sort of kind of looking back in time.  

But the difficulty is that it can take a long time for patients to progress. And you say, “Oh, that’s great.” And that is great. But, from a research – from a statistical side, it means things are really slow. If you have to wait 15 years to assess whether or not people progressed less in one treatment versus another, it’s really slow going. And so, we have to do a compromise of what’s – you know, what do animal studies say? What does retrospective analysis, when we might have people who started treatment 30 years ago, and now we’re just seeing how did it all work out? It’s not a perfect study, because biases can creep in, but it’s what we have now. And so, there’s a lot. And I think, increasingly, progression is being recognized as a goal of therapy, to prevent progression.   

Personally, it is one of my major goals, because I think we do a pretty good job at preventing clots with available treatments. But I don’t think we do a very good job at preventing progression, mostly, because we don’t exactly understand what’s driving that. And so, I think until we develop that deeper understanding and really invest the time and effort in terms of learning which approaches can help prevent progression, we’re going to continue to have these questions.  

Are Mobile-Optimized Tools Impacting MPN Care?

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Are Mobile-Optimized Tools Impacting MPN Care? from Patient Empowerment Network on Vimeo.

How is myeloproliferative neoplasm (MPN) impacted by mobile-optimized tools? Dr. Kristen Pettit from Rogel Cancer Center shares digital education resources and patient education websites to aid in MPN patient support and education.

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Transcript:

Dr. Kristen Pettit:

There are a wealth of resources online in the MPN world, there are a number of different digital education resources that have expanded even with COVID, a lot of patient education materials that are out there on various different websites, from the Patient Empowerment Network to the MPN Research Foundation and MPN Advocacy & Education International.

There are also symptom trackers that can be very helpful to where you can enter your individual symptoms as often as you want to and get a more objective number to follow how you’re feeling over time and see how things may be progressing over time.