Evolving CML Research: Assessing the Need for Ongoing Treatment in Clinical Trials

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Topics include: Living Well , Treatment and Understanding

Is there such thing as treatment-free remission for a CML patient?  Dr. Jessica Altman of Robert H. Lurie Comprehensive Cancer Center of Northwestern University shares data about novel treatments coming from current clinical trials. The most exciting news is the major advance in treatment-free remission for patients in molecular remission. Dr. Altman expands on this notion and discusses the new therapy, ABL001, currently being studied.  

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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. 

Mary Windishar:

Welcome to ASH 2016 here in San Diego.  I'm Mary Windishar, and we are with Jessica Altman, MD.  She is the director of the leukemia—the acute leukemia program at Northwestern University.  So tell me if I got it right and what else we have to say about your work. 

Dr. Altman:

Hi.  Thank you so much.  It's a pleasure to be here.  I—you're correct.  I'm the director of the acute leukemia program at Northwestern University and the Robert H. Lurie Comprehensive Cancer Center, where I am responsible for and really enjoy caring for a large number of patients with acute myeloid leukemia, acute lymphoblastic leukemia and other chronic myeloid leukemia and other chronic leukemias.  I run a large number of clinical trials in those diseases as well. 

Mary Windishar:

So what kind of news are you hearing now at the conference? 

Dr. Altman:

So ASH has been really exciting identifying novel treatments for the range of hematologic malignancies.  I think in specific the questions were what is new in chronic myeloid leukemia?  So CML in general is a disease where we have excellent outcomes.  CML is treated with one of multiple pills to target the abnormality that leads to the development of CML.  The kind of evolving questions in CML are how to address toxicities of therapy and minimize those toxicities.  

Mary Windishar:

What is the toxicity of therapy? 

Dr. Altman:

Toxicity is just a side effect of treatment, whether that's something as simple as fatigue or diarrhea to something a bit more complicated such as coronary artery disease or vascular disease.  And we as a medical community and research community have been interested in gathering information about toxicities and trying to learn how to minimize those toxicities. 

In CML, it is remarkable that we're in an era where we can focus on toxicities.  And while response is critically important, because the outcomes are generally very good we are at a point in CML therapy where we are as interested in side effects in folks. 

Mary Windishar:

And are there new advancements, or is it just looking at how well things have gone? 

Dr. Altman:

No, there are a couple of major advances that have been highlighted at this meeting.  One of those advances is looking at individuals whose disease is in exquisite control and trying to figure out if they can come off treatment, either for a period of time or thinking about more indefinitely. 

That is something that we do not recommend at all in just kind of someone to do outside of a clinical trial.  It must be done only in the context of a clinical trial right now, but we're starting to have some outputs from folks having come off treatment in the context of a clinical trial and being followed extremely closely to ensure that their disease continues to be controlled.  

Mary Windishar:

You have certain feeling about this, though.  When we spoke earlier, you looked pretty happy about the opportunity to do that. 

Dr. Altman:

I think for individuals who know that if their disease reaches a molecular remission, that means no detectable evidence of disease, to know that there might be a possibility in the future that they'll be able to come off treatment provides a lot of hope.  Some individuals have some chronic side effects from treatment, and to know that they might be able to have a period of time—we don't know how long that will last for everyone—a period of time when they may be able to be free from treatment that adds hope for some individuals. 

Now, this is limited only to the individuals who have had extraordinary response to treatment, and must, at least to date be done in the context of very close monitoring in a clinical trial.  

Mary Windishar:

How long before the clinical trial is done, and you'll know more about people being able to do this just on a regular basis? 

Dr. Altman:

So the data that's being presented at ASH from a couple of venues, or a couple of—venues isn't the right word, but a couple of different protocols is starting to be more mature. And I will look forward to just a little bit more maturity of the data and making sure that I've seen that data published and rigorously reviewed before I offer it to my patients. 

Mary Windishar:

But you have to tell me that in mere mortal language.  So is that years or is that...

Dr. Altman:

So I don't think it will be years.  I think that we may have some of this data over the next year that—unfortunately, I don't know exactly when that data will be published and available, but the sense from looking at the abstracts and hearing the conversations is I think that may be what happens. 

So we mentioned treatment?free remissions is one thing that is very exciting.  The other area that is really exciting is there are a couple of new approaches to treatment of CML.  For a while we thought that we were left with a number of tyrosine kinase inhibitors, so we have imatinib (Gleevec), nilotinib (Tasigna), dasatinib (Sprycel), bosutinib (Bosulif) and ponatinib (Iclusig), which for one disease is quite a large of number of therapies. 

There is a new drug that's under development called ABL001 which binds and inhibits the abnormality in CML at a slightly different position than the other tyrosine kinase inhibitors and is being studied for individuals who have not tolerated the drugs or whose disease hasn't responded.  And the early outcomes look like the drug is tolerable, so that's the first step.  And some individuals appear to be responding as well, so I really look forward to further development of that compound.  

Mary Windishar:

How long does it take for a compound to be—to go from the development to the bottle itself that we take the pill in? 

Dr. Altman:

So many drugs that are studied in early?phase clinical trials never make their way to the market, but in CML we have almost an embarrassment of riches of the number of drugs that have come to market.  I think that drugs are getting to patients sooner than they have previously, but specifically the drugs in CML have come to patients relatively quickly. 

Mary Windishar:

And you talk about clinical trials.  How does a patient get involved with something like that? 

Dr. Altman:

So the first step is for them to talk to their physician and to say, hey, do you know anything about clinical trials?  Do you think that there's any benefit for me to be thinking about a clinical trial?  There are different clinical trials in different settings.  So when one has newly diagnosed disease, whether that's CML or any other type of cancer or other medical condition in general, one should feel empowered to ask their physician is—what are the options other than what you're telling me is the standard of care?  And is—are clinical trials an option? 

And if one is in the context of CML not tolerating treatment or not reaching the appropriate milestones or have met their milestones but the disease is progressing, that's another opportunity to talk to your physician and say, hey, is there a clinical trial that would be appropriate? 

Clinical trials are all listed on clinicaltrials.gov, which is a website available for anyone to access, but it can be challenging to muddle through a lot of information when you're not familiar with it.  Many academic centers have coordinators that are available to help patients get through understanding some of the clinical trials and see if there are clinical trials available at those individual centers.  But the best resource is frequently starting with your physician. 

Mary Windishar:

And are you challenging your physician by saying, hey, what about a clinical trial?  Are you saying, I don't think what you're doing is good enough? 

Dr. Altman:

That's a great question.  I don't think that that is challenging in a bad way.  It might be challenging, but patients need to be their own advocate.  It may be challenging, but it's not offensive.  I like my patients to be engaged with their medical care.  I like them to approach me.  It's possible that I have missed something, or it's possible that I know about it but I haven't thought about it for that individual patient, and so having the dialogue helps. 

And knowing that my patient is as engaged with their medical care and concerned is a wonderful feeling as their physician.  

Mary Windishar:

I think things have changed.  I think that doctors—well, maybe we've changed, the patients have changed, but it is different now, isn't it, that patients get so much more involved instead of just doing what they're told? 

Dr. Altman:

I think patients are involved.  I think media helps patients be involved.  There are—it's easy to access information now, and I think all of that helps.  There also are support networks for patients, and all of that helps empower a patient to be able to have a conversation with their physician. 

Mary Windishar:

So in general CML seems like—a lot of hope. 

Dr. Altman:

I agree.  I agree 

Mary Windishar:

Thank you for the work that you've done to put it there.  

Dr. Altman:

Oh, thank you so much.  

Mary Windishar:

And thank you for joining us.  I'm Mary Windishar. 

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 30, 2016