ASH 2014 CML Research Update

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Topics include: Treatments

Where do we stand with developing CML research? Dr. Michael Mauro, a leading expert, joined Andrew Schorr at the American Society of Hematology meeting to provide an update. Dr. Mauro shares his thoughts on emerging research and expresses his hope that CML could become a "functionally curable disease."

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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.  I'm Andrew Schorr.  We're on location at the American Society of Hematology meeting in San Francisco.  We're talking about CML with someone we've met with many times before, Dr. Michael Mauro. 

Dr. Mauro:

Thanks for having me. 

Andrew Schorr:

Welcome back to Patient Power. 

Dr. Mauro:

Thanks for having me, Andy. 

Andrew Schorr:

Okay.  So we've moved over the years, and you've been very involved in it, from it being really for many people a fatal condition to living from—not everybody, but for many people a longer, pretty normal life, not always without side effects, and with a number of medicines available.  Both from this meeting and what you know of is going on now, what do you want to update patients on? 

Dr. Mauro:

You know, this story is—it's not a sprint.  It's a marathon.  So now we're starting to answer some of the subtle questions that we probably needed to answer better earlier on, but we're, now that we have much more mature data from clinical trials and a little bit more deliberate agenda to sort out what exactly are the right milestones. What exactly is our best treatment algorithm, what are the subtle side effects that we're seeing or the somewhat concerning side effects we're seeing late in treatment, what risks do people have who have CML who may be in remission and may be quite safe but may still have things to be advised about. 

It's, you know, it's still a moving picture.  It's very good results from all of our therapies we have,  very good options for frontline.  We have good options for salvage and second and third and fourth treatment.  We're still lacking in treatments for patients in advanced phase.  That's really an area of unmending still.  So I think that requires some attention. 

And we probably still have room for therapeutics in the realm of immunotherapy and minimal residual disease and any other targets that we can sort out.  So there [are] still many unanswered questions. 

Andrew Schorr:

Let me ask about immunotherapy for a minute, because a buzz at this meeting has been immuno-oncology across blood cancers.  Does that apply to CML, where your immune system can be turned on to fight the cancer it didn't recognize the first time? 

Dr. Mauro:

Yeah.  Certainly we've seen exploitation in a fantastic way for checkpoint inhibitors and CAR T-cell therapy.  Now the boon there is you're—you're dealing with, you know, cells that naturally communicate the NT-cells, so the lymphoid cancers are a natural fit. 

But I think there's really hope that once we recognize what some of the epitopes and the things the immune system might recognize we might be able to engineer the immune system or cellular therapy to treat myeloid disease.  It hasn't moved as fast, but I think the better we understand what we see in the diseases we're treating so well now with immunotherapy we will.  So I'd stay tuned on that, but I'm hopeful. 

And, you know, CML has always been an immune-responsive disease.  It's one of the best diseases to treat with allogeneic transplant, because of the graft versus leukemia effect, the indolent nature of it, the responsiveness to transplant is—really probably leads us to look hard at this disease. 

Andrew Schorr:

All right.  Over the last couple years we've talked about can we knock back CML to such a degree that you can stop therapy, and I have friends who've done that.  So where are we now with that? 

Dr. Mauro:

So we've seen a number of trials happen, and I think we have a really good sense because there's an almost complete concordance between what we've seen that pretty much no matter how someone looks as long as they have had good therapy for a period of time, hopefully they didn't have high-risk disease, and they've defined themselves as being in a stable and deep remission—and I'm going—we can quantify that—it looks like a significant minority or maybe nearly half of those patients may not proliferate off treatment. 

But I think we're changing our definitions.  First off, in order to stop therapy a patient may not need to have an arbitrarily low level below, say, a complete molecular remission.  I'm not saying that that's ready for prime time.  All of this is still in the realm of clinical trials.  But, for example, a trial in Europe that's looking at discontinuation for patients who really probably have less than a complete molecular remission but have at least a 4-log reduction, they do as well as people who have a deeper remission.  So I think it's really about defining CML as being nonproliferative. 

And then we clearly need to look more at what's going on with the fraction that don't—you know, that failed the experiment.  We have a little more confidence in retreating people who failed the experiment, if you will, people who do proliferate, which is good, but not complete confidence.  So I think we're still in the realm of clinical trials, but we're learning a lot more, and I'm really hopeful that this will be a functionally curable disease. 

Again, we don't know exactly what that means.  This is a new world for treating someone into a remission that may still have disease that's detectable but it's not proliferative.  

Andrew Schorr:

Michael, one other question.  So we have imatinib (Gleevec®) that's been around a long time.  Now there's even a generic version 

Dr. Mauro:

Soon, yes. 

Andrew Schorr:

…in some places.  And then we have more powerful TKIs and range of different medicines.  How do you know what's right for which person and certainly in an age where cost is a factor? 

Dr. Mauro:

I don't know if there's a right answer yet.  I think we know what we know about advantages of using more therapy or more potent therapy at diagnosis, but the same issues remain with us.  We are concerned about what is the trade-off, what is the—what is the downside that be maybe has not allowed us to see an advantage in overall survival, that has more patients maybe not staying on treatment for the long haul. 

I mean, success on therapy for CML needs to be on treatment, tolerating therapy well enough, in remission and gaining relevant landmarks, and that may be slightly different for different drugs.  So I think there's still room to explore how good we can use—how well, rather, we can use imatinib.  Maybe we can use it at somewhat higher doses.  I think that's a natural question people will be asking in places where it's available at lower cost. 

And we still need to look carefully at the data we generated with second-generation drugs.  They are more powerful, and they are putting patients in remissions deeper and faster, but how much quality that brings to patients is still a point of discussion.  I'm not—I don't want to diminish the results we've seen, but I also want to caution us to not think we're done and just assume that—that the book is closed on that. 

Andrew Schorr:

One last thing, and that is people feel well, often, many people do.  And they've been taking the pills, and they say, hmm, doesn't matter if I miss a dose or two or three or whatever.  What do you want to say about the importance of staying on therapy? 

Dr. Mauro:

I try to be open and honest with everybody that I see that asks because we're all human, and it's very natural to drift into a state of confidence and somewhat of laxity, but we have to still remember it's leukemia.  If it does require, you know, intensive therapy or consistent therapy, especially early—some people see the paradox.  Why should I be so worried about taking therapy religiously every day if I hear that there's a trial where I could just stop it or my friend who also I see in the clinic has just stopped therapy. 

But again, that's not ready for prime time yet, and it's really getting—gaining a high-quality remission, which is probably tied into adherence to treatment and us as physicians and people who are being treated for CML to talk, so we can manage any of the pitfalls that come.  A lot of times adherence is due to side effects, and we're concerned about side effects.  And so that's our job.  We don't want to miss the ball there.  But patients, we have to work together to make sure we get to—get to the finish line, and we can hopefully see a brighter time. 

Andrew Schorr:

And I think work together is really the name of the game.  You've got to communicate with your physician.  If you're concerned about side effects, if you're concerned about costs, if you're concerned about any of the issues related to whether the drug therapy is right for you, whether you can just stop…

Dr. Mauro:

Talk.  

Andrew Schorr:

…talk, talk, talk.  Okay?

Dr. Mauro:

Absolutely. 

Andrew Schorr:

Michael Mauro, thank you so much for being with us…

Dr. Mauro:

Always a pleasure. 

Andrew Schorr:

…once again on Patient Power. 

Dr. Mauro:

Thank you. 

Andrew Schorr:

Andrew Schorr on location in San Francisco.  Busy place.  News continues to come out.  Hopefully, if you're living with CML, you do well.  It's a long?term, hopefully very, very long journey, and the medicines will do well for you.  And if you get to stop, that would be great, too. 

Thanks for joining us.  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 December 18, 2015