6 replies to this topic

[BethG]

Hi all,

 

I see a lot of the same faces and a few new ones on the forum. Glad to see so many still fighting the good fight.

 

I was one of the early patients in the Phase I trial for ponatinib--I started at the 30mg dose almost six years ago. Been doing great, briefly flirted with "undetectable", but mostly been co-existing with a very low level of disease and doing great. Unfortunately, when the FDA did their shenanigans last year and we had to go to 15mg while they sorted stuff out, I lost my response. I've been back up to 30mg for the past year. When we did my BMB this year (as required by the trial protocol--thank goodness it was required), I was still showing a loss of response, but with an addition of trisomy 8 and isochromosome q17 in my leukemic cells. It looks like I'm headed for transplant. I don't have any additional mutations, but do still have the t315i mutation. The only good news in this situation is that whatever is going on was caught very early. Without the BMB, it is very possible we would have found out things were evolving much farther along in the process.

 

Thankfully I am in great hands--I'm being seen by Dr. Shah at UCSF and Dr. Druker has also been consulted.

 

We are trying a higher dose of ponatinib (45mg), which kicks my a** with side effects--mild pancreatitis, body rash, fatigue, etc. and will re-do my BMB at the end of March. I'm consulting with a transplant doctor at UCSF later this week and also heading to OSHU next Monday for a consult and to look at the trial for the new Novartis drug.

 

It is never a dull moment with this disease. I've had a sobering reminder that you can never take it for granted.

 

Hugs to all,

 

BethG

[mikefromillinois]

Thanks for the good info Beth.  I think your situation serves as an excellent reminder that even though the great majority of us here have had their disease controlled by our meds there are no guarantees.  Taking that idea a step further, I think that we should all have a "backup plan" in mind, so that if ever our situation begins a turn in the wrong direction we can quickly take steps.

 

Here's wishing you the best...

 

Mike

[Gail's]

I am so sorry to hear about the serious change due to bureaucratic upset. Curious as to why you couldn't have taken 2 15mg tabs when only 15 mg tabs available. Do you think it would have been able to maintain your nearly undetectable level? Hope you get hope and encouragement during your upcoming difficulties.

[BethG]

Gails: I've always taken 15mg tablets, probably because I'm in the original Phase I trial. Everyone in the trial was moved down to 15mg due to safety concerns, so I went from two pills a day to one a day. While we will never know for sure, I believe that if I had stayed at 30mg I would have probably maintained my response. For whatever reason, my disease wants to be a Bad Actor...opening the door, even a little bit with the 15mg dose, gave it the opening it needed. This is totally my opinion.

 

But, thankfully this time around I am in incredible hands with Dr. Shah and getting the right medical advice so I can make good decisions. It is pretty devastating, but one must keep marching ahead! 

[Melanie]

Beth, so sorry to hear the direction your journey is taking, but so happy you're in good hands and receiving good advice about your options. Your so right about not taking things for granted and am also grateful for the required BMB protocol while in clinical trials. If it's any consultation, I'm in a clinical trial and I've had the trisomy 8 show up in two of my BMB, but then disappeared within 6 months. I didn't have the other mutations you do though, which I know are really difficult to manage.

 

I'm curious about the new Novartis drug you speak of.  Is it possible that it might postpone the transplant for you? The back up plan to the back up plan.I hope you can keep us updated on your progress.

 

Praying for the best for you and that the 45 mg works quickly... that we hear soon that you're going back to 30 mg for maintenance and the other options remain "back up plans" for now. Take Care!  Melanie

[Trey]

Beth,

You should look into this issue:

http://www.scienceda...50209113044.htm

http://www.nature.co...ature14119.html

 

A drug called Axitinib used for other applications has been found to be effective at binding with and inhibiting T315i.  It is a TKI drug for kidney cancers, but a recent limited study showed promising results.  You should ask Dr Shah about it.

 

Good to hear from you after so long.  Hope you were able to get back in the saddle. 

Edited by Trey, 23 February 2015 - 10:50 AM.

[hannibellemo]

Beth,

 

It is so good to hear from you! Wish it were better news, but, as you say, you are being seen by the best so you are where you need to be right now. With a little luck maybe the 45mg. will knock out the 8 and q17 and get you back in lresponse.

 

Thinking of you and wishing the best for you!