Tag Archive for: MPN clinical trials

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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La historia de Yolanda: mi camino hacia un diagnóstico de neoplasia mieloproliferativa

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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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How Can MPN Experts Help Inform Patients About Clinical Trials?

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How Can MPN Experts Help Inform Patients About Clinical Trials? from Patient Empowerment Network on Vimeo.

What are optimal ways for myeloproliferative neoplasm (MPN) experts to inform patients about clinical trials? Expert Natasha Johnson shares how she prepares herself, her approach to informing patients, and how she reacts if she senses patient hesitancy.

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“…go on clinicaltrials.gov, search up MPN trials, and that will first clue you into could you be eligible and where are they being done. And then you can contact that center or ask about it where you’re being seen locally and just get information. You’re not committing to it completely. Minds can be changed at any time.”

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Transcript:

Natasha Johnson:

As a provider of MPN patients, I first make sure that I am educated on what clinical trials are available and out there. And then when I see patients, I offer these options. I’m a strong, strong advocate for clinical trials because if it wasn’t for them, we wouldn’t have the medications that we have today. And just in the last several years, we’ve had many medications approved for MPNs that we didn’t have 15 years ago, and all that was done by clinical trials. I have on firsthand seen benefits of clinical trials.

So I strongly encourage patients to be a part of that. If there’s hesitancy from the patient or caregiver, I would take time to dig into that and help to answer or alleviate fears that come along with that. It’s important that the clinical trial coordinator who knows specific protocols of trials very, very well, they can come in the room or spend time on the phone with the patient to answer any questions, to alleviate fears and allow that opportunity if a patient is open and willing to do it.

So my activation tip would be first of all, go on clinicaltrials.gov, search up MPN trials, and that will first clue you into could you be eligible and where are they being done? And then you can contact that center or ask about it where you’re being seen locally and just get information. You’re not committing to it completely. Minds can be changed at any time. The clinical trials are greatly, greatly, greatly beneficial and as I said, we have what we have today because of patients who participated in clinical trials 10, 20 years ago and then have given these great opportunities to patients now. 

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How Can Underrepresented MPN Communities Access Support?

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How Can Underrepresented MPN Communities Access Support? from Patient Empowerment Network on Vimeo.

What are some ways for underrepresented myeloproliferative neoplasm (MPN) communities to access support? Expert Natasha Johnson shares ways that she’s helped inform patients about resources and clinical  trial information to help bridge gaps in care.

[ACT]IVATION TIP

 “…try to get into a large cancer center or academic center and see an MPN expert. Many times, this is just by self-referral. Charity is sometimes provided through these. Zoom visits can be done as consults or follow-up visits. So my encouragement would be search these out, find out who the experts are, and contact them directly and see if there is any possibility or a way that you can get in to see an MPN expert for a consult so you can get the best care possible.”

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Transcript:

Natasha Johnson:

From a nursing perspective, there are several gaps that affect our underrepresented communities. And unfortunately, these are patients who are very late diagnosed, maybe incorrectly diagnosed, and even underdiagnosed because there is difficulty with access to care and then getting treatment. And here’s where it’s important that as providers, as nurses, we’re educating the community. We’re educating the public health department on these diseases to help pick up for that. We’re making people and patients aware of these diseases to help pick up on that. And there are many resources that are available online for free.

There are also local support groups for patients with MPNs and their caregivers that you can join in-person or on Zoom that provide just education resources on new trials that are out there or new treatments that are out there. Patients themselves can research clinical trials by looking at clinicaltrials.gov and see what’s out there and contact the academic center that’s performing those trials. There’s free information online that provides recorded sessions from conferences or speakers or speaking done by the MPN experts that you can just look to and get to easily to help understand the disease, knowing the symptoms, and then guiding treatment.

If you belong to an underrepresented community, my activation tip would be to try to get into a large cancer center or academic center and see an MPN expert. Many times, this is just by self-referral. Charity is sometimes provided through these. Zoom visits can be done as consults or follow-up visits. So my encouragement would be search these out, find out who the experts are, and contact them directly and see if there is any possibility or a way that you can get in to see an MPN expert for a consult so you can get the best care possible. 

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Emerging MPN Therapies in the Research Pipeline

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Emerging MPN Therapies in the Research Pipeline from Patient Empowerment Network on Vimeo.

What emerging myeloproliferative neoplasm (MPN) therapies are in the research pipeline? Expert Dr. Idoroenyi Amanam from City of Hope discusses MPN treatments that are under study, what the therapies target in MPN patients, and the outlook for the future of MPN care.

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Transcript:

Lisa Hatfield:

Dr. Amanam, can you speak to any exciting new developments in MPN care or trials that you see moving forward with great progress?

Dr. Indoroenyi Amanam:

Yeah. I think for MPNs and namely really the classic BCR-able or Philadelphia chromosome-negative MPNs, which include essential thrombocythemia, polycythemia vera, and myelofibrosis. I think we have a lot of exciting therapies that are going to be possibly FDA-approved in the next couple of years. So currently, for essential thrombocythemia, really the dogma therapy is related to keeping the counts under control and giving a therapy to reduce the risk of having a blood clot or stroke. We actually are in a space where we have therapies that are going to be targeting the underlying clone or basically the cells that are driving the proliferation of these platelets that lead to high platelet counts. And so I think that’s exciting.

So we do know that, in MPN there is an overexpression of Bcl-xL, and there’s a drug that targets Bcl-xL. And we’ve seen really great responses in essential thrombocythemia. And as a segue, this drug also targets the same cells and polycythemia vera and myelofibrosis, and we’ve seen really great responses in those patients. We also have had difficulty in managing patients who have myelofibrosis, but have very low counts. And typically the FDA-approved drugs that we’ve been using actually make the counts worse.

And so there are multiple drugs that are in the pipeline that are helping patients with low blood counts. And what they do is they help increase your red blood cells and reduce your requirements for red blood cell transfusions.

And one of the drugs helps stimulate erythropoiesis, and it’s an injection. And we’ve seen really good results in reducing the risk of…or reducing the amount of transfusions that patients receive. And then another one of these drugs targets ACVR1, which we understand that in myelofibrosis, you have overproduction of hepcidin, which leads to worsening anemia. And so by targeting ACVR1, it helps control this hepcidin. And by doing that these patients have improved red blood cell counts. And so that’s another drug that likely will be coming…that will be FDA-approved very soon, and I think will help patients in this space.

We also are interested in immunotherapy. And I think in other cancers, immunotherapy has been very successful in eradicating those cancer cells and curing some patients. And so there are clinical trials looking at a vaccine which targets certain mutations that are relevant to MPN patients. And also we are interested in actually using other types of immunotherapy namely, CAR T, which really helps connect your own immune cells to these cancer cells to help clear them out. And so I think over in the next five to 10 years, there’re going to be a lot of drugs and a lot of therapies that are going to really help patients who have MPNs.

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How Can MPN Clinical Trials Be Diversified?

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How Can MPN Clinical Trials Be Diversified? from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) clinical trials participants become more diversified? Expert Dr. Idoroenyi Amanam from City of Hope explains how MPN clinicians, institutions, pharma companies, and others can help expand the participant pool for all population groups.

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Transcript:

Lisa Hatfield:

Dr. Amanam, how can we better encourage more diverse participation in MPN or any cancer clinical trials?

Dr. Indoroenyi Amanam: 

This is a great question. I think that going back to the idea that we want to practice the best science, we want to be able to publish the best data. The responsibility is on the clinicians, the scientists, the clinical trialists, the drug companies, the institutions to really be able to structure clinical trials that are relevant to our real world experience. And so how can we better encourage that? I think from a government perspective, potentially incentivizing drug companies and institutions and the other major players that really are involved in pushing this field forward to practice better science. I think we put so much of responsibility on the clinical trial participants, and we’ve spent many years really analyzing or thinking about the reasons why the clinical trial participants are not or the lack of clinical trial participants is from specific groups.

But I think we have to put that look at us, the scientists, the physicians, the institutions, the companies. What are we not doing right here? And I think we have to put a lot of energy there. And once we’re clear that being able to have a diverse participant pool will give us the best results and therefore will lead to your drug being approved. I think we will have more participants from all groups.

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Using Telemedicine to Help MPN Clinical Trial Enrollment After COVID-19

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Using Telemedicine to Help MPN Clinical Trial Enrollment After COVID-19 from Patient Empowerment Network on Vimeo.

How can myeloproliferative neoplasm (MPN) clinical trial enrollment be aided by telemedicine? MPN expert Dr. Jamile Shammo shares ideas for how clinical trial protocols can be adjusted with telemedicine and other remote options for improved patient care. 

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Transcript:

Lisa Hatfield:  

With the nature of telemedicine being virtual and the fact that clinical trials, thus far, have only been conducted in-person, it may seem strange or impossible to try to move clinical trial participation to the virtual space. However, clinical trials are so important to research and getting the best care to patients, that researchers are beginning to figure out how to make this a reality. MPN expert Dr. Jamille Shammo reiterates the importance of clinical trials by saying:

Dr. Jamile Shammo:

There’s no doubt that COVID has certainly impacted our ability to enroll patients on clinical trials. There have been a lot of governing bodies that have created various rules and regulations around that to facilitate enrolling patients on clinical trials, and I think right now we are seeing that this has become feasible, such that we are able to enroll patients yet again on the clinical trial. So, now I think that we have the vaccine that is available, it has become a little bit more feasible and possible to do so. So, this should not stop us. I think we should continue to seek better treatments for MPN patients actually the only way to do so is by, you know, only patients on trials.

Lisa Hatfield:

So what ideas do researchers have, to start making clinical trials available via telemedicine? 

They believe in simpler clinical research protocols that not only allow telemedicine for nontreatment visits and consents, but also require fewer laboratory tests and imaging studies that may burden patients with extra visits and reduce the regulatory burden for research staff as well. It is important to specify that patient visits and/or consents can be conducted remotely in the trial protocol. 

A patient-centric approach toward clinical research needs to be explored in virtual trial assessments. A 2022 ESMO journal article by Sessa et al described new approaches to oncology clinical trials, including the use of electronic patient consent forms and medical updates, local laboratories for blood tests, home nursing visits for blood draws or infusions, and electronic patient-reported outcomes. It also stressed the importance of working together with local doctors, direct-to-patient drug delivery, and remote monitoring to increase patient convenience. 

While there is still more information to consider, this gives us hope on how trial access can be improved and approached in the future for better MPN care. 

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Will Advancing Technologies Allow MPN Clinical Trials in Patient Homes?

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Will Advancing Technologies Allow MPN Clinical Trials in Patient Homes? from Patient Empowerment Network on Vimeo.

Will myeloproliferative neoplasm (MPN) clinical trials at patient homes be enabled by advancing technologies? Experts Dr. Krisstina Gowin from University of Arizona Cancer Center and Dr. Ana Maria Lopez from Sidney Kimmel Cancer Center share their perspectives on clinical trials models that work well with remote models and an update about the hospital at home concept.

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Transcript:

Lisa Hatfield: 

Do you think that technology has progressed enough that clinical trials might allow a patient to be at home, maybe in a more remote area and monitored remotely? Whereas in the past, that same clinical trial required them to be at the facility? Do you think that we’ve progressed to that point in some clinical trials?

Dr. Krisstina Gowin: 

You bet. Yeah. I think COVID out of necessity has forced us to do that. And I have in my own clinical trials, even with pharmacologic clinical trials conducted telemedicine visits that were approved by the sponsor. So the paradigm is shifting, and particularly when it is oral therapeutics, I think that’s really accessible when they’re IV subcutaneous, I think that has different challenges. Obviously you can’t do that as remote, but when their oral therapeutics are non-pharmacologic intervention, such as our integrative interventions, I think it really lands well to a more remote experience.

Lisa Hatfield: 

Okay. And then would that require communication between the local oncologist and maybe someone like yourself, the investigator on that clinical trial to know what is going on with that particular patient? I assume that that communication would be ongoing?

Dr. Krisstina Gowin: 

Absolutely. Always.

Lisa Hatfield: 

Okay. Yeah. All right, great. Well, thank you for that information. So, Dr. Lopez, kind of a similar question for you. What are some examples of how technology is influencing cancer care right now?

Dr. Ana Maria Lopez: 

Yeah, let me just add on the clinical trial question.

Lisa Hatfield: 

Oh, Yes.

Dr. Ana Maria Lopez: 

That there’s also the opportunity. Again, there were so many things that we thought, “Oh no, we just can’t be done.” But because of the necessity, necessity is the mother of invention, we do remote consent, so that was a big deal in the past. We can also do a televisit ahead of the appointment, and screen for the cancer clinical trials, people travel large distances for studies and instead of traveling four or five hours, and then to be told, “Oh, actually you don’t meet the criteria.” To be able to do all of that at a distance, to get the records, to get the images, to review all that needs to be reviewed. And then to say yes, and not only yes, but we can also do your consent at a distance in some situations.

And then when you come, there’s actually the more substantive, perhaps even the treatment. There’s also a large movement around hospital at home and that these patients that are eligible for that would be able…with digital support, be able to get hospital level care in some cases at home. So some of that may involve infusion, some of that…again, but that visual connectivity and in the past you really had to kind of conceptualize it, and it was kind of space aging to talk about it. But we now, we’ve all done FaceTime, so I think we all really can understand what it entails, so tremendous shifts and, we want to try to keep that momentum going for our patients. So, I do think that, there’s so many ways that technology has impacted cancer care, even when we talk about the electronic record and patients accessing the electronic record and patients having the opportunity to go into a portal and to see their labs, to see their reports…

To be able to track their changes. All of that is really, really powerful. You know, patients with…the most common I think is patients with diabetes who track their blood sugar sometimes to the minute and they can say, “Oh, I ate that and now I see the impact.” So the opportunity for monitoring, the opportunity for also bringing in experts. So let’s say there’s a patient with a rare disease and the expert is elsewhere, there might be the opportunity to bring people together. We do tumor boards. That’s just part of what we do in cancer care. And also as many…there are health systems now so that it’s not one hospital, it’s multiple hospitals together where we can bring all of those folks together, bring in local expertise, regional expertise, national expertise, all for the patient’s benefit.

So there are so many ways that technology even something as simple as the note. Now this is something we experimented with and it’s still in experimentation phase, but there were these Google classes where you could interact with the patient and as I’m talking, the Google glass would record kind of the conversation and would come up with some sort of a structure for the note. So for what that encounter had been like. So there are lots of ways of how do you capture natural language in real time to really help the workflow, the documentation process. So I think there’s aspects to help the patient, to help the families, to help the clinical teams and to help everybody work together. 

Lisa Hatfield: 

Great. Thank you. And you talked about the patient portal, and I’m one of those patients at fault of seeing a lab result before my doctor saw it and calling him or sending a message via MyChart saying, “Hey, this is going up. What’s wrong with this?” So I’m sure you don’t have to mention any names. I’m sure you’ve seen the challenges of, digital health too, are having that patient portable or portal accessible to patients. So anyway, just wanted to throw that out there that I’m sure that brings challenges to you. Also few little challenges here and there.

But at the same time, that’s so good, right? It’s so good that patients are engaged. It’s so good that you’re engaged. And I think as long as we’re communicating that yes, you may see this before me, so you may have questions and then, we just get together and answer the questions. 

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Is Technology Accelerating Progress in Myeloproliferative Neoplasm Care?

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Is Technology Accelerating Progress in Myeloproliferative Neoplasm Care? from Patient Empowerment Network on Vimeo.

Is myeloproliferative neoplasm (MPN) care being advanced by technology progress? Expert Dr. Krisstina Gowin from University of Arizona Cancer Center discusses how MPN care has benefited from technology advances and explains two MPN technology tools and how they assist in MPN patient care.

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Transcript:

Lisa Hatfield: 

So, Dr. Gowin, a couple of questions for you. Is technology playing a role in accelerating progress in MPN care?

Dr. Krisstina Gowin: 

Oh, absolutely. And I think some of the ways that it really accelerates progress is pulling us together. So what we need to recognize is that myeloproliferative neoplasms truly is a rare disease, and we just celebrated Rare Disease Day. But there’s a lot of challenge in treating patients and progressing the field forward in rare diseases because you can’t do the big clinical trials. It’s hard to come together because everything’s siloed, and there’s just a couple patients here, a couple patients there in each practice. But with digital health and clinical trials that are offered on a digital platform, it pulls the nation together and even the world together. And we’ve seen that. I’ve done an international survey-based analysis and I had 858 MPN patients from 52 countries participate in that survey. And so that just shows how it pulls the world together. And for the web app that we just discussed, we had 93 patients say they were interested within three weeks, and within actually a week, we identified them all and then took three weeks to actually accrue them to the trial. So it really speaks to A, how MPN patients are digitally engaged and excited about these kinds of platforms. And then B, how effective it really can be to pull the groups together.

So yes, I think it’s…and that’s really how we’re going to get progress is through these kind of interventions with a rare disease. And I hope it’s okay if we jump back to something you said, Dr. Lopez, which is, I think telemedicine is so so important to bring everyone together. And in particular, I see that on the transplant ward. And so in myelofibrosis, that’s the only curative therapy. And so many myelofibrosis patients actually go through allogeneic stem cell transplantation. And my goodness, that is a socially isolating experience. Patients are in the hospital, not uncommonly for at least 30 days and then have to be near their transplant center for three months, which often is away from home. So to pull in their support system, both through the acuity of the transplant themselves and then the couple of months after is so crucial to a successful transplantation. And I think through FaceTime and also the MPN support groups, which is very robust, the patient advocacy and the way the MPN network sticks together on a digital platform, I think is really unique and offers unique support.

Lisa Hatfield: 

Thank you. And then what role does technology play in the disease symptom management, and in particular, in clinical trials too. What role does technology play with clinical trials?

Dr. Krisstina Gowin: 

Well, I think it helps us through different, clinical trial accrual patterns, we can see who’s eligible where, so it helps us identify patients. It helps us to, understand the different kind of precision-based medicine approaches so we can start to pool the data, say for, particular mutations… ASXL1 mutations. And so it helps us in the precision medicine aspect of clinical trials and now we’re looking at symptom management and how do we really integrate that. So large survivorship platforms like Carevive, if you’ve ever heard of Carevive, is now integrating our validated symptom assessment form into the Carevive platform. So now we can really collect that data and use that to mine it for potential kind of retrospective analysis. So it’s helpful for clinical trials as well as for our clinicians and clinics to really identify changes in symptom burden.

And just as Dr. Lopez was mentioning, that we can track these over time and it can flag and say, “Oh, your symptoms are changing, they’re increasing over time,” and maybe we need to be thinking about that. And so Carevive is really kind of an electronic medical record driven it’s really a healthcare driven platform, but now there’s patient ones too. And I just learned about this two weeks ago, I was at an MPN conference in Phoenix and learned about MPN Genie. And so MPN Genie apparently, is tracking…patients are putting their symptoms in and that’s shooting that information to the electronic medical record to their doctors. And so I think that’s fantastic, ’cause, we now get that information real time and we can change our clinical management, maybe bring that patient in sooner, maybe do a bone marrow earlier. We never would’ve identified that if it weren’t for those kind of digital engagements, so I think it’s a really exciting time. And I think we’re going to see more and more of these new platforms and ways for, different EMRs and smartphones to be communicating back and forth between patients and providers. 

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How Did COVID-19 Impact MPN Treatment?

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How Did COVID-19 Impact MPN Treatment? from Patient Empowerment Network on Vimeo. 

How was MPN treatment impacted by the COVID-19 pandemic? Watch as expert Dr. Jeanne Palmer discusses the positive and negative impacts of COVID-19 restrictions on care of MPN patients.

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Transcript:

Lisa Hatfield:

All right, so, Dr. Palmer, the COVID pandemic has resulted in significant changes to many aspects of daily living for all of us, but for patients like myself who are living with cancer, there are different realities that we have to deal with, so can you give a brief overview of the impact that COVID-19 has had on MPNs.

 Dr. Palmer:

So I think the impact of COVID-19, I think we just spoke about some of the favorable things that telemedicine became a real reality, some of the detrimental things, enrolling in clinical trials has been very, very difficult because of the fact that, number one, the public health emergency, some patients weren’t able to travel. And then number two is, I think there has been sort of an exodus of people working in healthcare, I think healthcare has become extremely stressful because of all the pressures associated with the COVID pandemic. So having the appropriate staffing for clinical trials has been difficult, but one of the things that I think is coming out of this that I think will be really positive is there are a number of studies that are being looked at now that are actually creating ability to have some of the visits done by a telemedicine. So taking what’s not as critical to be seen in person, and what labs we don’t need to necessarily get that need to go to a central processing area, but there are actually ways that we are working with home health care companies with different labs to be able to provide some of this ability to do telemedicine, especially on the clinical trials where there’s monthly visits.

I have had patients travel from multiple different areas of the country to be on clinical trials. I’m usually more in the Southwest or at least the West Coast, but I think that with some of these changes, it’s going to be a lot more of a reality for it. So I think some of the pressures of the COVID pandemic will…again, there will be sort of a silver lining of it, and that we may have this ability to do that, because even if I looked at…you look at the pre-COVID clinical trials, if there was a trial that needed monthly visits, which a great number of them do, I would say the majority of my studies that I have for patients with MPNs require monthly visits, at least the first six months. Being able to have that extended out is hugely important and will allow access for it, so if we can have a virtual visit, even every other visit, that can make a big difference in somebody’s ability to access new treatments.

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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! 

Key Questions to Ask When Considering an MPN Clinical Trial

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Key Questions to Ask When Considering an MPN Clinical Trial from Patient Empowerment Network on Vimeo.

MPN researcher Dr. Gabriela Hobbs shares advice for patients interested in joining clinical trials, including an explanation of eligibility criteria and key questions to ask their healthcare team about participation. 

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:

So, what should be considered when deciding whether to join a trial? 

Dr. Hobbs:

What a great question. Many things need to be considered when joining a trial. And I think some patients are really eager to join a trial, and they just need to be aware that they may be either too healthy, or they may have other things going on that may not make them eligible.  

And that’s okay. There are actually many ways of participating in research, even if it’s not a clinical trial that requires a medicine. For example, we often can send patients to what’s called a tissue bank where they have patients just give a sample of blood.  

So, patients can participate in research in many different ways. When considering whether or not a patient should enroll in an actual clinical trial with a new medicine, I think it’s really important for the patients to be informed and to not be afraid to ask questions. First, what is a clinical trial? Second, what will this trial involve? Is this a drug that has never been given to people before, or is this a drug that has already undergone many different clinical trials? And this trial that’s being offered is a Phase III trial where the purpose of the study is to get the drug to be approved.  

So, I think learning about the risk of the study, how it’s been utilized, and also the other more practical things. What is the time commitment of this clinical trial? How often are you going to have to be going to the office because of the clinical trial? Because there’s certainly a big investment in the part of the patients in terms of their time. Participating in a clinical trial most of the time requires more time than not participating in a clinical trial. That’s not always the case. There are some studies that definitely don’t require that many visits.  

But most clinical trials will require at least something extra from the patient. And I think it’s really important to ask about that, to read the consent that’s given to the patients. Oftentimes these consents are very long.  

And so, they can be overwhelming. I personally find them overwhelming. And I review a lot of those consents. And so, I think taking a minute to really ask those questions, speaking to the research staff, and getting the clarification on that is really important.  

Like you said, it is impossible to approve new therapies and improve the care that we offer our patients without patients participating in the clinical trial. But that doesn’t mean that absolutely every single patient needs to participate in a clinical trial if it just doesn’t make sense for them. 

Expert Perspective | Promising MPN Research

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Expert Perspective | Promising MPN Research from Patient Empowerment Network on Vimeo.

What’s the latest in promising MPN research news? Dr. Angela Fleischman shares an update about treatment research and discusses the importance of 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:

Dr. Fleischman:

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. 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 (Besremi) 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:

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.  

Health Equity: Accessing Quality MPN Care and Clinical Trials

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Health Equity: Accessing Quality MPN Care and Clinical Trials from Patient Empowerment Network on Vimeo.

How can health equity be addressed in MPN care? Dr. Angela Fleischman discusses the importance of clinical trial diversity and ways to help provide equitable MPN care for all patients.

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:

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.  

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:

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.  

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.  

The Risks and Benefits of Participating in an MPN Clinical Trial

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The Risks and Benefits of Participating in an MPN Clinical Trial from Patient Empowerment Network on Vimeo.

What are the risks and benefits of MPN clinical trial participation? Dr. Angela Fleischman discusses clinical trial risks, benefits, safety protocols, monitoring, and importance of 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:

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. 

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 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:

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.