Skip to Navigation Skip to Search Skip to Content
Search All Centers

An MPN Researcher Discusses Calreticulin and Progress in the Field

Read Transcript Download/Print Transcript
View next

Published on September 29, 2015

At an MPN Town Meeting, Dr. Kim-Hien Dao from OHSU Knight Cancer Institute, joined Patient Power to discuss her research related to MPNs. Dr. Dao shares information about the latest discoveries, including the calreticulin (CALR) mutation and what it means for patients. She shares her excitement about progress being made in the field and new agents that will be approved in the near future.

Featuring

Partners

Stanford Cancer Institute

Transcript | An MPN Researcher Discusses Calreticulin and Progress in the Field

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.  I'm Andrew Schorr, and we're sitting here with a leading researcher and clinician in MPNs, that's Dr. Kim?Hien Dao from OHSU Knight Cancer Institute in Portland, or Oregon Health & Science University I like to think.  Thank you so much for being with us.  

Dr. Dao:

Thank you very much.  

Andrew Schorr:

Dr. Dao, so tell us about your role there, your work you're doing in science to help all of us all with MPNs and your help with patients day?to?day in the clinic.

Dr. Dao:

Yes.  I'm an assistant professor in—at the Knight Cancer Institute in the department of hematology and medical oncology.  My clinical specialty is on myelodysplasia and myeloproliferative neoplasm. 

My research, I have a laboratory, a research component to my program where I am currently investigating the role of calreticulin in—and its mechanisms of JAK/STAT activation.  And then my clinical program is translating some of the work that we do at the OHSU Knight Cancer Institute to clinical trials in MPN. 

Andrew Schorr:

All right.  Let's talk about calreticulin for a second.  So that was another gene found active related to some MPNs.  And so is your work to say, well, how—what understanding do we have of it, could there be medicines that help patients where this is identified?  

Dr. Dao:

Yes.  So calreticulin is a ubiquitous protein, and it's not clear how it leads to the same disease in those patients that have the JAK2V617F mutation.  And so we know that they have very similar JAK/STAT activation and cytokine profile.  The question is really how does calreticulin and this mutation lead to JAK/STAT signaling specifically because we don't see a direct connection at the present moment. 

Andrew Schorr:

So if someone is tested and calreticulin pops up, what you're saying is you're trying to figure out what to do about it. 

Dr. Dao:

Yes.  And I think phenotypically these patients are different from the JAK2V617F-positive patients, so we're seeing clinical differences. And there's a lot of work out there from Dr. Tefferi and his group, and so we see that the different—the different clinical phenotype, and now the question is can we also target that disease in a different way… 

Andrew Schorr:

All right. 

Dr. Dao:

…since the mechanism is different in how it activates JAK/STAT signaling. 

Andrew Schorr:

All right.  You're a scientist, what are you excited about?  I mean, we've seen changes going on just in the last few years, hopefully you have a long career, and a lot of progress will be made.  What are you excited about? 

Dr. Dao:

Well, I'm very excited about the progress we made in 10 years from the discovery of the JAK2V617F mutation in 2005.  You know, the first five years we were focused on the JAK/STAT signaling and controlling symptoms and spleen, and now the second five years we're seeing a big growth in novel agents in other pathways that target fibrosis and target telomerase, and so we're really seeing a growth in targeting this disease from multiple pathways as opposed to just JAK/STAT signaling. 

In The New England Journal in 2013, they showed that the median number of mutations in myelofibrosis is 13. And so, as you can guess, you know, it's not just one pathway that's abnormal in these diseases.  And I think to really improve quality of life and symptoms and the natural history of these diseases, we need to think of these diseases as multiple pathways being abnormal and target it in combinations. 

Andrew Schorr:

Right.  So like other cancers now, many other cancers, sounds like combination therapy as you figure out the combinations, that will be much more typical. 

Dr. Dao:

Yes.  And I'm very excited about all the studies that are out there in terms of looking at this now methodically.  

Andrew Schorr:

Okay.  So I know my goal as an MPN patient, myelofibrosis, is hopefully to see a long life, a better quality of life, and, boy, wouldn't it be great if there was a cure?  What's your goal as someone on the research side?  Same?  

Dr. Dao:

Yes.  Yes, for sure.  I mean, I think this is a chronic disease, and we need to, you know, work on delaying the natural history of these disorders towards, you know, end?stage myelofibrosis and acute myeloid leukemia.  And so I think treatment options that actually improve symptoms and quality of life and delaying that natural progression is very important.

And I think all of us in the MPN research community are very focused on those target areas, you know, in terms of improving of lives of patients with these disorders. 

Andrew Schorr:

Okay.  So you're in the lab.  You get to see things at the earliest stages.  Are you hopeful?

Dr. Dao:

Yes, I'm very hopeful.  I think there's—you know, with what we've accomplished in the last 10 years it's been very exciting to see the growth in the research and also the different agents that are coming to patients for clinical research.  The patients are very involved, so there's a huge efficacy in myeloproliferative disorders, and I think that's been very helpful in us studying these new agents in patients. 

So I'm very excited about the progress.  I'm also excited that we'll have more than one FDA?approved JAK inhibitor and also other agents for these patients.  

Andrew Schorr:

All right.  We're on the same team.  

Dr. Dao:

Yes. 

Andrew Schorr:

Okay?  People living and affected by the condition and you, maybe with the treatments of tomorrow.  Thank you for all you do. 

Dr. Dao:

Yes.  Thank you.  

Andrew Schorr:

Dr. Dao from the OHSU Knight Cancer Institute in Portland joining us here. 

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.

View next