Tag Archive for: Mays Cancer Center

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.

See More From the The Path to MPN Empowerment

Related Programs:

Diagnosed with an MPN? Why You Should Consider a Second Opinion

Could an MPN Clinical Trial Be Your Best Treatment Option?

Improving Life with MPNs: The Latest Research and How to Get Involved

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.

How Does Genetic Testing Impact Your MPN Treatment Options?

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How Does Genetic Testing Impact Your MPN Treatment Options? from Patient Empowerment Network on Vimeo.

How can genetic testing results impact your treatment and treatment response? Dr. Ruben Mesa provides an overview of common mutations associated with essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF) and how identification of these mutations are moving research forward.

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.

See More From the The Path to MPN Empowerment

Related Programs:

Essential Lab Tests for MPNs

Could an MPN Clinical Trial Be Your Best Treatment Option?

An Expert Summary of Current MPN Treatment Options

Transcript:

Dr. Ruben Mesa:

We are learning much more about the genetics of Myeloproliferative Neoplasm, as we truly are about the genetics of many diseases. First, when I speak of genetics, these are not the genes we think of of inherited genes that are passed from mother and father, to son or daughter. These are the genes in ourselves that potentially can change over the course of our lives, and those changes or mutations can be associated with diseases.

So, what we have learned is that the genetic changes that are associated with myeloproliferative neoplasms are important, both in terms of predicting how the diseases might behave, and also, potentially in terms of therapies. The genetic changes fall into two different groups.

There’s a first group of the most common mutations that we think are important in driving the disease. The most common is the mutation in a protein called JAK2. That’s a mutation in about half of the patients with ET, half with PV – or half with myelofibrosis, and the majority with polycythemia vera. There is mutations in calreticulin. That’s about in a third of patients with ET, and a third with MF. And then, there’s mutations in MPL, which are present in a handful of patients with ET and with MF.

But in addition to those three mutations that tend to be mutually exclusive; patients tend to only have one of those, and there’s a small group of patients that do not have any of those three. But there’s another group of mutations that we have learned about.

That we are able to obtain on panels of sometimes anywhere from 40 to 100 genes that may or may not be changed or mutated in diseases like MPNs and the implications of what those pattern of changes in those mutations have for those patients. 

MPN Treatment Decisions: Which Path is Best for You?

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MPN Treatment Decisions: Which Path is Best for You? from Patient Empowerment Network on Vimeo.

Dr. Ruben Mesa provides an overview of available treatments and reviews important factors to consider when choosing a therapy for essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF).

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.

See More From the The Path to MPN Empowerment

Related Programs:

Essential Lab Tests for MPNs

Ready to Start an MPN Treatment? What You Need to Consider

An Expert Summary of Current MPN Treatment Options

Transcript:

Dr. Ruben Mesa:

The treatment landscape for myeloproliferative neoplasms is changing very rapidly. And in a good way, it’s increasingly having many more options for patients with Myeloproliferative Neoplasms. But I would separate it, really, into two groups. First, there are those individuals with essential thrombocythemia and polycythemia vera.

These individuals, we have newer therapies, such as interferons, we have, potentially, use of JAK inhibitors, we have some experimental therapies, as well as prior therapies we’ve used and become accustomed to, including hydroxyurea, phlebotomy, and aspirin.

But we’re learning much more about how to use these therapies; how to combine them; what constitutes success with these therapies; what should constitute a change in terms of therapy.

And there are new therapies being developed in the future that will impact this group of individuals with earlier MPNs: ET and PV.

For patients with myelofibrosis, the treatment is evolving. Patients with Myelofibrosis are affected in different ways. It is, in some ways, a more problematic disease.

There is evolution of our most impactful therapy, of stem cell transplantation. We have a better sense of in which patients we should consider that treatment, and how that can be applied in the safest way. We also have more medical treatments. We just saw in 2019 the approval of Fedratinib as the second specific JAK inhibitor approved for patients with myelofibrosis.

We, additionally, now have, truly dozens of clinical trials of new therapies in development that are in clinical trials right now that might be helpful for patients with myelofibrosis who have either had Ruxolitinib, or have a suboptimal response to Ruxolitinib, or sometimes even newly diagnosed patients. But I would say the future is very bright.

So, it is key with a treatment to first understand what is the treatment, what is the dose, and what is the goal? Each of the treatments have different goals. Some of the goals are to decrease the likelihood of blood clots or bleeding.

And frequently, we assess whether we’re protecting against the blood clots or bleeding by bringing down elevated counts. Is the plate account high, and we’re trying to bring it into the normal range? Is the hematocrit high, and we’re trying to bring that to under 45%? Is the white blood cell count high? Have we lowered each of those? First, it’s around controlling blood counts if that is the goal, as well as trying to decrease at risk of blood clots or bleeding.

 Second, if patients have symptoms associated with their MPN, sometimes itching, sometimes symptoms associated with high courts, sometimes enlargement of the spleen, or symptoms associated with the spleen, have we reduced or nullified those symptoms? Have we shrunk the spleen if the spleen was enlarged?

And then, finally, we assess our goal by trying to be sure that patients are not progressing or getting worse on the disease. So, depending upon the treatment, we first asses what is our goal? Is it to improve counts? Is it to improve symptoms? Is it to shrink the spleen? And have we accomplished one, two, or all three of those goals? Or was only one those our goals to begin with? 

Newly Diagnosed With an MPN? Start Here.

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Newly Diagnosed with an MPN? Start Here. from Patient Empowerment Network on Vimeo.

If you’ve been diagnosed with an MPN, such as essential thrombocythemia (ET), polycythemia vera (PV) or myelofibrosis (MF), Dr. Ruben Mesa outlines key steps you should take, including a visit with an MPN specialist.

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.

See More From the The Path to MPN Empowerment

Related Programs:

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

Ready to Start an MPN Treatment? What You Need to Consider

An Expert Summary of Current MPN Treatment Options

Transcript:

Dr. Ruben Mesa:

Patients who have a Myeloproliferative Neoplasm should consider seeing an MPN specialist at least at some frequency. The myeloproliferative neoplasms are not common illnesses. They’re not exceedingly rare, but they’re not common. And there is many nuances in terms of how we best diagnose the disease; the discussion we have with you regarding what are the treatment plans and goals, and then putting that plan into effect.

So, frequently, there’s a value in seeing someone who focuses on MPNs to help to establish that plan, and then frequently, there is a home physician, hematologist, or medical oncologist that works together along with the specialist in terms of managing the patient.

When patients first come for their visits related to an MPN, they have many questions. You know, they’re not common diseases, and people typically don’t have much experience with them. They’ve not had a family member that’s afflicted or someone at work. So, frequently, it comes on out of the blue. People will frequently, sometimes, go online and get a lot of information, but sometimes too much information; information that may or many not be appropriate for them.

So, there are many questions that are valuable, and I always advise patients to write down their questions ahead of time because sometimes in the heat of the moment, having a conversation, particularly with a new physician or provider, those questions may not, necessarily, be top of mind for them. So, we can go through those questions clearly.

I think key questions, I wouldn’t limit it to one key question, but I’d say I would put them in categories. 1.) Truly understanding the diagnosis; what’s the actual diagnosis that that patient has. 2.) What does the physician think are the risks that patient has? With each of the diseases, there are different risk classifications, and that will also help to give patients a frame of reference if they read other information about their disease online from highly reputable sources, or other educational sort of materials.

To understand, what is the recommended treatment plan. The plan may or may not included medications and understand what those medications are intended to do, and what their side effects may be, or what to anticipate.

It may or may not include aspirin, it may or may not include phlebotomy, or it may or may not include other therapies. So, understanding that diagnosis, understanding the risk, and understanding, what is the recommendation in terms of treatment.