Could an MPN Clinical Trial Be Right for You?

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Could an MPN Clinical Trial Be Right for You? from Patient Empowerment Network on Vimeo.

Is a clinical trial your best MPN treatment option? Dr. Ruben Mesa explains the clinical trial process and how patients may benefit from participating.

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

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Transcript:

Dr. Ruben Mesa:

There is much exciting research in myeloproliferative neoplasms. First, research trying to understand, why do people develop MPNs, and why do they progress. This is crucial research, and that this basic research to better understand the diseases will help us asses whether our treatments are having an impact slowing down the progression of the disease, and help us better design therapies that, hopefully, can cure these diseases.

Be reassured  that our goal as a scientific community is to cure the MPNs. Now, until we’re able to do that, we want to be able to best control them as best we can. So, the next level of research is really in new therapies; primarily drug-based therapies, but future therapies using the immune system; potentially using vaccine therapy to try to better control the disease to make the disease as neutral in your life as possible.

Our goal, short of curing the disease is to make the disease as invisible in your life as possible. Hopefully, minimal side effects, minimal symptoms, protected against risk of blood clots or bleeding, ideally, decreasing the risk of progression, and hopefully without any significant side effects from the medication your receiving.

So, that really is our goal.

 Clinical trials are a crucial way for us to improve the treatments that we have for any diseases. And in particular, in areas like myeloproliferative neoplasms where we have therapies, but we don’t have cures, clinical trials are crucial. Clinical trials are a structured way for you to be able to receive a new treatment. That treatment is closely monitored, and starts with a strong belief that that treatment is going to be beneficial for you.

Being on a clinical trial has many steps, but you are in the driver seat in each of them. So, you’re able to enroll in a study, and you’re able to decide at any point whether or not you’d like to continue on in that study. You are made clearly aware of what you’re receiving; what dose; what to expect at each and every step of that therapy.

It’s a treatment just like any other, but we use them because we are hoping that it will be better than the treatments that we have, and we do it on a clinical trial so that we can learn from that experience. If that drug is better, then we should probably expand its use and give it to other people, and have it be approved and used around the world. Or for whatever reason that therapy is not as helpful as we would like, then we learn from that, as well.

Why was it not helpful? Was it the wrong therapy? Was it targeting the wrong aspect of the disease? Were there side effects that made the therapy not beneficial? So, we learn a lot about it in either direction. Hopefully, individuals who participate in clinical trials will have a direct benefit themselves by being able to experience a new therapy that is, hopefully, better. But also, they do have the ability to help other patients now and in the future that will be facing the same disease they have.

Could an MPN Clinical Trial Be Your Best Treatment Option?

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Could an MPN Clinical Trial Be Your Best Treatment Option? from Patient Empowerment Network on Vimeo.

Lindsey Lyle discusses the role of clinical trials as an MPN treatment option and how research is advancing the field.

Lindsey Lyle is a physician assistant at the University of Colorado Cancer Center, specializing in hematological malignancies with a subspecialty in myeloproliferative neoplasms (MPNs). More about this expert here.

See More From the The Path to MPN Empowerment

Related Programs:

Diagnosed With an MPN? Why You Should Consider a Second Opinion.

Essential Lab Tests for MPNs

Could an MPN Clinical Trial Be Your Best Treatment Option?

Transcript:

Lindsey:

When considering treatment, remembering that clinical trials are an option – and often, a very good choice – is something that I really try to communicate to my patients. Generally, there’s a stigma around clinical trials as patients feeling like a lab rat or some sort of a study subject, and there is a perception that they’re not receiving as good of care as they would if they were not on a clinical trial. However, in my patients, I really try to dismiss this thinking because at this point in time, we do have really fairly good options for treatments with MPNs.

However, we do not have a cure for MPNs outside of a transplant, and our treatments are not perfect, and so, enrolling in a clinical trial really should be considered by patients and their providers as a very viable option.

It’s generally introducing perhaps a new way of approaching the disease treatment. Oftentimes, clinical trials are using a combination of agents, which is not necessarily available outside of the clinical trial.

And so, with clinical trials, we’re always trying to make something better. We’re trying to learn something, we’re trying to, No. 1, help the patient – so, my No. 1 goal in enrolling one of my patients in a clinical trial is to, first of all, help them, help control their disease, help them to feel better, and to live a long and good life. No. 2, we learn as we go along. Clinical trials are critical for drug development and for the future of treatment in patients with MPNs.

So, not only are our patients helping themselves, but hopefully, they are helping the future MPN patients who may come along and need a treatment. So, I always like to keep this really in the conversation when discussing treatments, and it may be up front, and it also may be after a treatment has stopped working that we consider a clinical trial.

So, there are also a lot of things that kind of go into clinical trial management and different requirements, so if a patient lives very far away, it may be challenging for them to come back to the academic center on a regular basis for routine clinical trial monitoring that’s required by the study, but if they live close by, I generally do recommend this. They are also associated with clinical research coordinators or clinical trial nurses.

And, these patients are monitored really very closely, and it’s kind of nice to have that extra person in it with you in the clinical trial, just another point person to discuss, perhaps, how you’re feeling or different questions or concerns as the clinical trial proceeds. So, when talking about treatments, in my opinion, especially in MPNs, clinical trials really should be one of the options that is first discussed when thinking about starting treatment, and especially if a treatment has stopped working.

So, there are very many exciting possibilities in MPN research right now. We have a lot of combination therapies, which I think I am most excited about, because we have a decent backbone of therapy at this point, but building on that and trying to maybe enhance the way that the backbone therapy works, and also to perhaps change the microenvironment of the bone marrow – basically, trying to reverse fibrosis.

So, there is currently a drug in clinical trial that is looking at this, and we are proceeding with this trial, and really hoping for the best, but I think that to combination therapies where we can put two things together that we think work really well together to help produce good outcomes – I think I’m most excited about that at this point.