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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.
Thank you. It's a
pleasure to be here. Good morning.
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?
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(Iclusig), was withdrawn from the marketplace, and that was a decision that the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.
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
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.
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
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.
Okay. But the importance
is if the medicine is working, then working with your doctor you need to
remember to take your medicine.
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.
So it continues to be a positive time in CML.
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.
Thank you very much.
It's a pleasure.
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.