23 replies to this topic

[donnadavis]

When I went to my onc. today, after  three months I found my numbers have began to rise again. My onc. wants to do a bone marrow and if things are what he expects he wants

me to join a study with the new drug Ponatinib. I was on Gleevec for two years when it failed and I have been on Tasigna about eight months and  the same thing is happening.

My question is should I go for it ? I  feel  a little scarred , has anyone been in  one  of these studies?

[janner25]

Hi - I'm sorry that you are in the situation where the drugs don't seem to be working the way they are supposed to.  I think it's absolutely amazing that there seems to be new drugs coming out for trials / studies all the time.  I guess the question is - where are you as far as your counts - are you in blast or chronic?  Are you comfortable with what your doctor has advised you as far as the ponatinib?  I always said that if my doctor wanted me to be a guineau pig, that I would....but I'm one of the lucky ones so far in that the tasigna seems to have knocked everything down really quickly with no signs of it returning.

I think if you are comfortable with it - and your doctor is recommending changing meds because of your resistance, I would say go for it!  but again - easy to say - but maybe not to do.

Good luck and please keep us updated on your decision .

Janice

[GerryL]

Hi Donna,

Have a look at this thread http://community.lls...age/97607#97607

Gerry

[Trey]

Here is what I suggested several months ago -- still suggest it:

http://community.lls.org/thread/7984

[CallMeLucky]

I wouldn't do anything else until I got a mutation test done.  Why treat this blindly and why go to a trial drug when you haven't tried a very good drug that is already approved for use?  If you have a mutation that cannot be treated with Sprycel, then by all means, go to the trial, but if you do not have a difficult mutation, then I would think it makes more sense to try Sprycel first.  That is what I would do.

Best of luck

[gianfranko]

I agree that you should get a mutation test before deciding if you should join the trial.

One question: if she should come out negative for the mutation, is there any benefit of ponatinib over sprycel?

Also, if she decides to go for sprycel and she fails it, would the Ponatinib trial still be accessible?

[HPL]

I would agree with the comments above, make sure to have the mutation test and the biopsy, that may give you a good answer to what is best treatment.

As for the trial itself, I've been on Ponatinib for 18 months (can't believe it's been that long already) and nothing about the drug would scare me. Besides, you get monitored the same, if not better during a drug trial.

Regards,

Hans

Zavie's Club #1303

Weak Positive last 3 PCR's

T315I mutation found prior to joining study

[gianfranko]

HPL wrote:
I would agree with the comments above, make sure to have the mutation test and the biopsy, that may give you a good answer to what is best treatment.
As for the trial itself, I've been on Ponatinib for 18 months (can't believe it's been that long already) and nothing about the drug would scare me. Besides, you get monitored the same, if not better during a drug trial.
Regards,
Hans
Zavie's Club #1303
Weak Positive last 3 PCR's
T315I mutation found prior to joining study 

I don't want to hijack this thread, but I had some questions about drug trials:

1.  Who pays for the drugs?  I wonder this because these drugs are extremely expensive and my guess is that one's insurance is not going to cover experimental drugs.

2.  Who monitors you?  Is it specific doctors from the drug trial?  Is it your current oncologist?

3.  How is it that you join the trial?  Do you have to be in the same physical proximity of where the trial was setup?  I guess if it is their doctors who have to do the tests then this probably is the case.  But, if your current oncologist does the monitoring - can you be in another state?

[HPL]

1. During the study, the drug company pays for the drug. My insurance company is still responsible for 'standard treatment', things that I would normally have anyway such as BMB's, PCR's, etc. But overally I think my insurance company is coming out ahead with me on the study. There is a question what will happen once the drug is approved, from what I understand is that I can stay in the trial, but less frequent and they monitor for longer term effects. But I don't know who picks up the cost of the meds at that time.

2. The doctor at the trial site is now my primary oncologist, but I do know that they send all my records to my previous oncologist, as well as my primary care doctor (that one surprised me). I went in for a physical and he knew all about my results and progress. That was kind of a nice surprise.

3. How do you join ? Well, I would recommend to anyone that if one is remotely interested, first talk to your primary oncologist and get their opinion. But don't assume they know everything about trial and options out there. Then contact a trial site, typically the doctor is more than open to do a consultation. They look over your history, if you are eligable based on what they know at the time, as well as tell you about the drug protocol. Every drug trial has a complete protocol, that calls out what would be required of you, what you may expect, risks, etc. If you decide to proceed to effectively apply for the trial, then that doctor will have a bunch of screening tests done, to verify your eligibility.

You can partake in a trial anywhere in the country, if you're willing to travel. It's all about the requirements needed, time required, etc. One of my best friends here in Seattle was going to MD Anderson to participate in the Ponatinib trial for a few months, unfortuantely his CML was so aggressive that he passed away in January. There are no guarantee's with trials, and they carry risks, but in some cases they are the best options out there.

Regards,

Hans

[gianfranko]

Thanks for all the info.  I was just asking out of curiosity.  So far gleevec has been treating us very good.  But I think the info you gave us is really useful.

Also, sorry to hear about your friend; my condolences.

-Franco

[Zu-ko]

I have also been asked to join the same study of the drug Ponatinib. I was diagnosisd in June of 2010 and was on Gleevec and Sprycell. I have not made progress on either of these drugs. The chromosome test in the bone marrow did not change. All of the cells tested on the first and second bone marrow biopsies showed the presenence of the CML chromosome. I am going to see a Dr Michael Deininger who specializes in CML on Monday. I will keep you posted on how it goes. I am trying to keep a positive attitude about the thoughts of joing a trail drug.

[BethG]

You will be in good hands with Dr. Deininger!

I am also part of the ponatinib study--I will have been on this drug for 2 years as of June 2011. It is pretty powerful stuff and I have had great results. My last round of testing showed 0 on FISH and "faintly positive" on PCR. When I was deciding whether or not to enroll in this trial (I didn't have a huge choice--it was this or transplant as I have the T315i mutation), I figured that it is a TKI, I (kinda sorta) understand the mechanism of how they work, generally speaking TKI's seemed fairly "safe" and there was 10 years or so of experience with this class of drugs, so I went for it. I have had a few bumps in the road--but the big picture is that my CML is behaving itself.

Also, to answer one of the questions from above, according to my informed consent document, we get the drug for free until it is approved by the FDA and put on the market. After that we have to get it through normal channels through our insurance or out of pocket (I shudder to think what the cost will be!!)

Good luck! And, if you have any questions, please don't hesitate to ask.

BethG

[HPL]

I'll definately second what Beth said about Dr. Deininger. He was my trial doctor until the summer when left OHSU. Abselutely a wonderful doctor, and compasionate human being. I had a question this week about my Cytogenetics results, and I reached out to him and he replied within hours, even though he's not my doctor anymore.

Regards,

Hans

[bsbk13]

A special hello to Hans and BethG, both of whom have helped Bill and I so much during this crazy journey!  Hans, I am really happy to see you as I miss your blog!

Wanted to jump in here with a quick update.  Bill did well on Gleevec for about 9 months, then quickly went through Sprycel and Tasigna with little improvement.  He is 70 years old, therefore not a candidate for transplant, so was maintained....maybe I should say "maintained" on hydroxyurea for about 3 months before being accepted into the ponatinib trial March 15th.  He had to take so much hydroxyurea (up to 12  500 mg capsules....6 GRAMS...per day) that while he was alive, his quality of life was very grim.  Mouth sores were the bane of his existence and he lost over 25 lbs in 8 weeks.  Couldn't eat.  Couldn't sleep.  He was seeing his doctor 5 to 7 days a week for 2 1/2 months.  On Saturday/Sunday, he would go to an outpatient facility, have his CBC done, then get a transfusion if needed.  Basically like being hospitalized, but not.

He had been tentatively approved for the study in January, but had some ECG and pancreatic issues to sort out.  For weeks I thought he wasn't going to make it long enough to get the drug.....then suddenly everything fell into place and they called us to Tampa (Moffitt Cancer Center) immediately.

Anyway, we're 5 weeks into the study and while he was approved by Ariad to temporarily use hydroxyurea in conjunction with ponatinib, he has not needed it and the mouth sores are finally GONE.  Narcotic drugs didn't touch the pain....Magic Mouthwash barely affected them.  We finally discovered that over the counter topicals like Anbesol and a nifty little thing....Canker Patch? something like that...worked better than the prescription treatments (which cost up to $125 a bottle).

At 5 weeks out, he feels considerably better.  He has had to have platelets twice and red blood cells twice, but they're sticking with the 45 mg dose to try and get as good a response as they can, then they might adjust the dose downward.  We were told that initially he might need more tranfusions than he had had previously, so we weren't surprised.  They have backed him off now from going to Tampa every 2 weeks to going every 4 weeks, and his local oncologist has bumped him from 5 visits a week to 3 visits a week to 2 this week.

So far, we're VERY happy with this drug.  It was literally this or hospice.

Hope to see some good results at his first BMB May 7th.

What I don't know is whether this is going to be the next-step drug for all CML patients or whether its benefit is primarily for T315i patients like BethG, Hans, and Bill.  I'm guessing that while it is effective against T315i, it is also effective against all the Philadelphia + cells with or without the specific mutation?  Definitely worth talking to your oncologist about or contacting one of the cancer centers who are in the trial....

If you are in the Southeast, you could contact Moffitt Cancer Center.  Bill's doctor there is Dr. Pinilla-Ibarz.

BethS

[hannibellemo]

Beth S,

I'm thrilled that things are going so great for Bill! I hadn't heard from you (or missed your post) and I had been wondering!

Warm thoughts to you both,

Pat

[bsbk13]

Thanks, Pat!

It seems we've finally gotten a good break.  We are willing to accept that this might not be the drug for Bill (though it looks good at this point, fingers crossed), but what we weren't willing to accept is that he would never even get to TRY it.  That's the part that was really sticking in my craw.

It wasn't that the medical team at Moffitt was trying to keep him out on technicalities....they felt he was their #1 choice to be included....but there are guidelines that you have to meet.  As soon as he clicked in, they had him in Tampa.

:-)

BethS

[CallMeLucky]

Hi Beth,

Very happy to hear Bill is doing ok, I was worried when I first read your post and you said "He had to take so much hydroxyurea (up to 12  500 mg capsules....6 GRAMS...per day) that while he was alive, his quality of life was very grim." The past tense in your sentence made me very sad for a moment as I thought something happened to him.  I was relived when I read the rest of the post to hear he is doing alright.  I think it is so amazing that this drug is available and he has a chance to fight back with it.  I've heard good things about it, and yes, I have heard that it is good for anyone who has not done well on other drugs, not just individuals with T315i, but for anyone who is having resistance.  So it is good to know there is another weapon in the arsenal.

Speaking of additional weapons, something else I thought I would put out there.  There is another trial that is recruiting CML patients for a new drug that also targets T315i.  So in the event ponatinib did not work for someone, this is another avenue to pursue.

The drug is DCC-2036 made by Deciphera Pharmaceuticals LLC.

"DCC-2036 is a potent broad spectrum inhibitor of BCR-ABL kinase. Inhibition of BCR-ABL has been validated for effective treatment of chronic myelogenous leukemia and Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia. The emergence of mutant forms of BCR-ABL which resist inhibition by imatinib, dasatinib, and nilotinib is associated with loss of efficacy in treatment of the disease. DCC-2036 is a potent inhibitor of resistant mutants of BCR-ABL including the T315I mutation, and would therefore be expected to effectively treat patients who fail to respond to other BCR-ABL inhibitors."

Here is a link to the trial in case anyone needs it or just wants to check it out http://clinicaltrial...how/NCT00827138

Best of luck, I hope Bill's condition continues to improve and he does well on this treatment.

[bsbk13]

Oh Jeez, Lucky! Odd choice of words!  I meant it to mean "although", not "when", like "While I like Coke better, I'll take a Pepsi since that's all you have".  He's very much alive.

The next obstacle we need to deal with is the weight loss, much of which is muscle mass.  He's kind of in a place where he feels weak and doesn't want to exercise, but the less he exercises, the weaker he's going to feel.  He is experiencing muscle soreness down the backs of his legs (not the fronts!) which may be because of the ponatinib or may be because his poor little remaining muscles are being overworked.  I'm encouraging him to start slowly, but understand that there may be a certain amount of having to just suck it up and work through it.   We have a reclining exercise bike, and I think that would be a good place to start as it's not weight bearing, but can help build muscle and improve his circulation.

Any ideas for exercise?   I don't honestly know whether exercise is good for the leukemia-related fatigue in general, but am thinking you DO make more red blood cells with more muscle mass and aerobic exercise, and since he's chronically anemic, may help?

If anyone can point me to a discussion, article, or give their personal experience, that would be most appreciated!

On the previous drugs, he did experience some side effects, but seems to fall into the less-side-effects area in general.  The leg pain on Sprycel was probably the worst side-effect he had, but it's not like that now.  That was much sharper, but sitting down would relieve it. This is a duller pain, but sitting/laying down doesn't make it go away.

Thanks for your kind words!

Beth & Bill

[bsbk13]

Shoot!  Forgot to say, THANKS for the link to the other trial drug!  That's fantastic news.

As I believe I mentioned before, you can feel pretty hopeful when you start Gleevec and know you have other drugs to fall back on, but when you're all the way through Tasigna and transplant is not available for you, things look a lot more tenuous.  Having another potential option is HUGE!

Beth

[CallMeLucky]

Keeping in mind that Bill and I have a number of years between us, I can tell you from experience that exercise can help with the fatigue, but mustering up the strength to get started is hard.  It also seems like he has been through a lot and that is going to take a toll too.  If I were you, I would talk to the doctor about physical therapy.  While things like walking are always good and the bike you described also sounds like it could be good, I don't think he just try to do regular exercise without guidance.  A physical therapist can evaluate and prescribe various exercises that won't push him too hard, but will target the key areas he needs help with, like the legs.

As far as the link to the other trial, I think just knowing there is another option out there is always a big boost mentally.  I imagine it would be very difficult to be on that "last option".  Fortunately today, there are various trials with different drugs - the company that is working on DCC-2036, actually has another drug in pre-clinical development, DCC-2157, that also shows promise to work with T315i.  So there are more options coming!

Best of luck....