The Changing CML Treatment Landscape

Andrew Schorr:

Hello and welcome to Patient Power.  I'm Andrew Schorr on location at the American Society of Hematology meeting in New Orleans.  This program is sponsored by Northwestern Memorial Hospital. 

And with us is Dr. Jessica Altman from the Lurie Comprehensive Cancer Center, Northwestern University.  Thank you so much for being with us. 

Dr. Altman:

Thank you.  It's a pleasure to be here.  Good morning. 

Andrew Schorr:

Okay.  Let's talk about CML.  You and I have talked about it before.  Last year, there was so much excitement, then this last—during this last year one drug actually has been stopped.  So for CML patients, how do you feel about the news now? 

Dr. Altman:

Okay.  So we have, as you're aware, a plethora of drugs to treat chronic myeloid leukemia, and over the last number of years there have been an increasing number of drugs from what we started with, which was imatinib (Gleevec).  As you mentioned, one of the drugs, ponatinib (Iclusig), was withdrawn from the marketplace, and that was a decision that the company made in conversations with the FDA.  In Europe, that drug has not been removed from the marketplace, so this is something that is affecting patients in the United States, though certainly patients elsewhere are aware of the problems as well. 

The decision to withdraw the drug was based on the risks of cardiovascular, thrombotic events, and I think this brings to light to patients and their caregivers the risks with all tyrosine kinase inhibitors or drugs in this class to treat chronic myeloid leukemia and Philadelphia chromosome?positive ALL, the other disease we use to treat—the other disease we use that gets exposed to tyrosine kinase inhibitors. 

We still have a number of drugs available for patients, from patients who are initially starting treatment to those who need to switch therapy either due to lack of optimal response or a lack of tolerability of the drugs that they start with. 

Andrew Schorr:

So what's the headline now?  We're at sort of news central for discussions of treatments for patients with CML?  Should they still be feeling positive? 

Dr. Altman:

Absolutely they should still be feeling positive, and I'd like to stress that ponatinib, the drug that was—the decision was made to withdraw from the marketplace, is available if the drug is needed.  One's physician just needs to apply for a single?patient IND or a drug application to be able to obtain that drug and utilize it in the clinic.  Clearly, patients should be counseled about the risks associated with the drug, but if that is the best option for them that drug is available for them. 

Andrew Schorr:

Okay.  So with CML then, for many patients there is still the prospect of a long life as long as they work with their doctor to stay on their medicine and the dose that's right for them. 

Dr. Altman:

Absolutely.  So there are a lot of interesting things in CML.  The vast majority of patients will stay on tyrosine kinase inhibitors, have excellent life quality, very good tolerance of therapy, and should they be experiencing symptoms from the medications, those are certainly things they should discuss with their doctor.  Perhaps there are supportive measures that they can utilize to be able to continue to tolerate or tolerate better the drug that they are taking, or sometimes they need to be switched due to intolerance. 

Likewise, their disease should be exceptionally well monitored in the beginning with at least every three-month molecular studies to assess disease control, and that allows the ability to recognize loss of response or lack of attainment of appropriate response early.  In fact, one of the things that's most interesting that has come out over the last couple of years is the recognition that if there's an appropriate disease control at three months, 10 percent or less percentage of copy number BCR?ABL, then the chance of long?term disease control is outstanding for those patients.  

Andrew Schorr:

Okay.  But the importance is if the medicine is working, then working with your doctor you need to remember to take your medicine. 

Dr. Altman:

Take the medication daily, and certainly it becomes a challenge when we look at other chronic conditions.  Hypertension, diabetes, there is natural, a reluctance for individuals to continue to take their medicine daily, and CML, the current mandate is that patients need to take their medications daily.  Some of the medications require the drug to be taken in a certain manner.  For instance, nilotinib, or Tasigna, needs to be taken twice daily on an empty stomach, but it is also a very well-tolerated drug. 

And there is an interest in looking at the ability to stop therapy in patients.  That can only be done in the context of a clinical trial, but there are clinical trials that are ongoing looking at how to stop some of the medications. 

There are—as you have a sense, because one needs to take a medication daily the drug results in a functional—can result in a functional cure but doesn't cure the disease because for the majority of patients if they were to stop taking their drug the disease would come back.  So investigators are looking at the addition of other medications to see if they will eliminate the leukemic stem cell that is causing chronic myeloid leukemia and try to result in cure.  Likewise, there are some additional agents that are being investigated to try to target some of the resistant populations that—some of those were patients that would have previously been on ponatinib, and those are all drugs that are very early in development. 

Andrew Schorr:

So it continues to be a positive time in CML. 

Dr. Altman:

Absolutely. 

Andrew Schorr:

Okay.  Very good news for patients diagnosed with CML.  Now, remember, it's a rare condition, so it's important for you to have a consultation with a specialist such as Dr. Jessica Altman at Northwestern, so you're on the treatment plan long?term that's right for you.  Thanks for being with us once again. 

Dr. Altman:

Thank you very much.  It's a pleasure.  

Andrew Schorr:

As always, on location in New Orleans at the American Society of Hematology, 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 Northwestern Memorial Hospital, its medical staff 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.