MPN News and Updates From ASCO 2017

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Topics include: Treatments

As part of our coverage from the 2017 American Society of Clinical Oncology annual meeting, Dr. Srdan Verstovsek from The University of Texas MD Anderson Cancer Center provides a hopeful update for patients living with myeloproliferative neoplasms (MPNs). Dr. Verstovsek shares news on JAK inhibitors in development, the hope of immune checkpoint inhibitors and other antibody treatments, as well as promising therapies in development for chronic anemia.  Tune in to learn more.

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Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

Andrew Schorr:

Hello and welcome to Patient Power in Chicago at the America Society of Clinical Oncology meeting, the ASCO meeting.  I'm standing with someone that's well known to anybody with an MPN, that's Dr. Srdan Verstovsek from MD Anderson.  Thanks for being with us once again. 

Dr. Verstovsek:

My pleasure.  Thank you for having me. 

Andrew Schorr:

Okay. So I know you have hematology meetings around the world.  Hematology is just a smaller part of this, but is there something that's being talked about of interest to people with MPNs? 

Dr. Verstovsek:

Yes, there is.  In fact, tomorrow morning we're going to have an oral session of most important findings in myeloproliferative neoplasms along with all the other findings in leukemia entrancement.  And two presentations were selected from MPN field, and these are the results of the Phase III studies.  These are randomized studies that are done in patients with myelofibrosis. 

The first one was comparison of a medication called momelotinib and new JAK inhibitors compared to ruxolitinib (Jakafi), approved therapy for myelofibrosis.  And the second study was momelotinib compared to best available therapy after ruxolitinib in the second-line setting, so?called.  These are Phase III studies that were performed to possibly approve momelotinib, but, unfortunately, results it's kind of a mixed bag.  It was good for some improvements that we like, symptoms or a spleen or anemia, but not all line up very well.  So unfortunately, we will see whether this will lead to any further development of the drug or a need for another study. 

Andrew Schorr:

Okay. Now, you have other studies going on now with another one, pacritinib, that we've been hearing about for a while.  It had been stopped for some time, but now I understand it's sort of back in the game.  So does that give us the hope of another JAK2 inhibitor?  

Dr. Verstovsek:

Absolutely. We need to develop other JAK inhibitors.  It is a very unusual situation.  If you look back over last 10 years we tested in the clinic about 11 different JAK inhibitors, one approved.  There must be room for another one.  So we have two actually that are still in the running.  The pacritinib was tested in the Phase III randomized studies, two of them so far.  Again, mixed bag of results, need further refinement.  So a study will open throughout the United States very soon that will test different doses in patients that previously treated with ruxolitinib, so myelofibrosis patients open-label Phase II study to define the proper dose and schedule. 

The next one is NS-018—doesn't have a full name.  N stands for Nippon Shinyaku.  It's a Japanese company.  Again, the study was done in the second-line setting after ruxolitinib, so people who are not doing well.  It is active drug.  We are looking forward to more studies with that, too.  So we have two going JAK2 inhibitors in further development.  

Andrew Schorr:

Okay. So for any of us who are on a JAK2 inhibitor, the one approved one, ruxolitinib, would we at some point need another?  Or if that isn't working for you, are there other options, and it sounds like at least the science is moving forward trying to sort that out.  All right.  Let's move on to other approaches.  

Dr. Verstovsek:

Obviously, the JAK/STAT pathway, hyperactivity, that is the signaling pathway inside the cells is the driving force behind the diseases.  That's why we use the JAK inhibitors, to inhibit it, but we need different approaches.  There are several different approaches in clinical studies at the moment. 

The hottest one is so-called immuno-checkpoint inhibitors.  What this is all about is allowing your immune system, a patient's immune system, to recognize the malignancy and fight it along the medications that we give to the patients.  So, for example, in Houston we have a study with medication called nivolumab (Opdivo), which is allowing again the immune system to contribute and possibly help manage the disease.  That medication is already approved for six different other malignancies, and there are two other studies in Northeastern United States as well.  

Another approach is use of antibodies.  Antibodies are medications that are given to patients, usually IV, that go around and attach themselves to specific proteins on the surface of malignant cells.  So antibodies for the bodies, kind of funny thing, but it really is kind of targeting specific populations of cells that are of interest. 

And one in the clinical studies for myeloproliferative neoplasms is one called SL-401.  It is—again, don't have a full name.  It's antibodies for protein which we call CD-123, which is a protein on the surface of myelo cells, leukemic cells that are possibly a cause for a disease.  So it's really stepping up the field to trying to eradicate with a targeted agent the cells that would be causing the disease on its own.  And it's being tested in acute leukemias, myelodysplastic syndrome and myeloproliferative neoplasms. 

So new approaches are necessary, and these two are kind of out of the usual, not really pills, IVs or injections, but trying to harness the other knowledge that we have about the disease so far.  

Andrew Schorr:

Okay. So when you take this together, obviously some of us have myelofibrosis.  There are people with PV and ET who worry about their illness, about how it may progress.  Are you encouraged, Serge? 

Dr. Verstovsek:

I am much encouraged.  And there are other approaches.  I'd like to touch upon two other ones.  The anemia is a major problem in myelofibrosis.  So far we have been preoccupied with the JAK inhibitors and spleen and symptoms.  Hardly ever, with some exceptions, do they improve the bone marrow, but that doesn't happen.  Sotatercept, luspatercept, funny names for two medications that are being developed, that they're given as injection under the skin every three weeks for patients with myelodysplastic syndrome and for patients with myelofibrosis that are anemic.  

Some encouraging results were reported six months ago at the American Society of Hematology meetings for patients that are anemic and have a myelofibrosis.  So sotatercept, luspatercept, two similar medications in development for chronic myelo diseases.  

And now that you mention ET and PV, essential polycythemia and polycythemia vera, we all know about interferon, ropeginterferon.  Preliminary results were presented at American Society of Hematology meeting in PV, active drug, very well tolerated, injectable under the skin every other week, possibly in the United States to be developed for patients with ET and PV.  We are looking forward to these medications to improve our ability to control the disease and possibly with interferon perhaps even eliminate disease in ET and PV. 

Andrew Schorr:

Okay. So a lot for all of us.  Now, we have a whole—whole bunch of programs coming up for people living with MPNs, so please make sure you're registered with Patient Power, tell others about it, talk to your doctor about what you learn, and make sure you take advantage of all this.  But again, with Dr. Srdan Verstovsek from MD Anderson.  He's a wonderful partner in this as we work hard to educate you in the latest information and to give you hope, as it does for me, living with fibrosis.  Serge, thank you so much for being with us once again. 

Dr. Verstovsek:

It's a great pleasure.  Thank you again.  

Andrew Schorr:

On location in Chicago at the American Society of Clinical Oncology meeting in Chicago, I'm Andrew Schorr.  Remember, knowledge can be the best medicine of all. 

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you. 

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Page last updated on June 19, 2017