Help me! Compassionate use exceptions needed for trial drugs NOW

[Carolyns]

Hi Joe,

Thank you so much for advocating for us. I was excluded from the most recent trial due to previous use of Tykerb and Xeloda. I am excited to learn about the TDM1 Pertuzumab trial. I hope that I can hang in there until it opens and that I am not excluded for some reason. I am going to investigate the Heat Shock protein trial also.

It is just that I lead such a full and active life. I am a single parent working full time and helping out with my parents. I so want to continue my QOL for as long as possible. The next chemo's will put a dent in my life for sure... TDM1 would allow me to keep up and has as good a chance as anything else of working for me. Heck almost every drug I have been on is off label ... it is frustrating.

I feel like the old lab chimps. I was good enough for trials over the past 20 years ... now only phase 1 trials or none at all. Some of us tired warriors respond pretty darn well to these treatments... we might surprise the drug companies. I think that they could learn much from us. We are a growing population and living longer and longer...

It doesn't seem fair.

That said, I really, really appreciate every single thing you and Christine do on our behalf.

Happy Fathers Day!

Carolyn

[Believe51]

Next week is a busy one for many of us. I will be sending out positive vibes and prayers for us and what we are fighting. Please know you will be in those thoughts, I hope you make some terrific headway Monday. Good luck and stay strong. Ed is in a real sticky situation himself and I can relate so deep with all of your feelings. Stay strong and go get 'em. Let us know where you are at with this all, I am right behind you!>>Believe51

[Believe51]

I have been following this post and was not going to ask this. Sounds like you are set on this trial but what do you feel about Rexin-G?? I do not have the information in front of me but I can get you the contact information if you desire. Cannot stop feeling your pain and frustration.>>Believe51

[Carolyns]

Hi Believe51,

Thank you for your warm heartfelt concern. I follow your struggles as well and you and your husband are in my thoughts and prayers.

With regard to other trials, there may be others that I can qualify for when the time comes (could be a week or two). Wednesday I am meeting with a doctor about the Heat Shock Protein trial. Today, I do not qualify for any trials that are recruiting (that I know of) and there may not be trials open in a week or two either. The key words here are "qualify and open". Practically speaking my doctor believes that the TDM1 trial is the next best chance for me. It has shown the greatest results with less side effects even compared with the remaining largely "off label" chemo's that are up next for me. The most recent TDM1 trial opened and closed so fast that more than 100 patients were left standing in line without rain checks. I hope that those folks were able to find something else.

To me the question I will ask was already asked at ASCO and pointed out to me by our dear Lani from an article posted, “Another issue to ponder, Dr. Peppercorn pointed out, is whether there is "a clear rationale for withholding an experimental therapy outside of a trial, when we are prepared to use it to treat a patient within a trial."

My answer is NO. There is no clear rational for withholding or closing incurable cancer patients out of trials. The side effect of this choice for many is death. Some may have drug treatments off label that have even worse known side effects than the trial drugs… side effects like congestive heart failure.

Incurable breast cancer patients are living long with breast cancer and our numbers are growing. There is little to no “evidence” in our treatment. In a sense everything we do is an experiment with risks both known and unknown taken every day by the patients, their medical teams, the drug companies and the government. In my opinion this is just a failed process with false choices. I beleive that it can be updated to be a win, win, win for all with the will to do so. I do not believe that allowing incurable breast cancer patients into these trials would hurt clinical trial participation or that it should hurt the data if the FDA would remove this inclusion requirement. This is a failed process and people are dying because of it. I believe that updating this process will allow many of us to live to see the cure and contribute to finding the cure faster.

Think back to the scene in the movie “Living Proof” where the early participants in the Herceptin trial were not allowed to continue. How did they feel when they were turned away? How did their doctor feel when he had to turn them away? Who benefited? Not the patient. These trials are our life support and we deserve admission in the sunlight.

I have faith in the universe and believe that the right door will open for me. I also have the strength right now to voice my serious concerns around this issue. It doesn’t have to be this way. Thank you for your support and concern and mine for you is coming right back at you.

Love, Hope, Peace,
Carolyn

[Rich66]

I'm not super up on the formalities of trials but...seems like other drugs have been given fast track, orphan status etc. Eg. http://www.medicalnewstoday.com/articles/58460.php
Is that not possible with a drug as promising as TDM-1?

That having been said are PARP inhibitor (BSI-201) trials nearby?
Also, how about:
http://www.mskcc.org/mskcc/html/2270.cfm?IRBNO=07-155

[StephN]

Hello all -
This discussion has been rattling around in my head the last fews days as I fight off a vicious cold.

I am wondering why there could NOT be a class of use that is not exacly a Clinical Trial in the usual sense, but after a promising phase 1, a drug could not be open to compassionate use? The answer most likely lies in the clammoring that would happen for that use. If you let in 20 patients, why not 40 or 80??

Anyway, we know now how to administer once the dose is defined in phase 1, and how to gauge efficacy of the drug. If the patient shows improvement, stay on it. This could run parallel to the "official" trials. This "use" would probably have to be in centers where the trial is taking place for access to the drug.

But then the other questions are: which drugs to allow such use? Where are the lines drawn in such a gray area? I am sure other concerns arise, but just as in the trials there would be Informed Consent, and the drug companies are off the hook if the patient wants the drug that badly.

WHO PAYS? That is probably the biggest question, if this would not fall under Compassionate Use.

[Carolyns]

Hi Rich,
Thank you for your thoughts and ideas. I am no expert on this issue either but I am trying to get up to speed fast. I have watched in horror as I saw other people trying to outline the issue in order to get treatments approved.

My doctor feels (as I do) that TDM1 shows the most promise for me and my particular situation. That is what matters most to me. I feel that I should not be excluded if my doctor feels that it is what is best and I agree.

If not TDM1 then (so far) it would be Doxil. I have had CAF and I am concerned about possible heart issues. Also, as a single mom with a full time job my ability to work while in treatment is important to me. I think that even if I didn't have a full time job I would still deserve the option to get into a clinical trial that is my next best option. Every choice in this setting is just a roll of the dice with our best educated guess. I don't think that just because I have been heavily pretreated that I should be closed out of or excluded from trials.

With regard to the Parp trials, so far my doctor has not felt that those are a next logical choice for me. I will ask again.

My scenario with TDM1 exclusions so far were:
1st attempt - not heavily pretreated enough to qualify.

2nd attempt - no previous use of Xeloda which was a requirement to enter trial.

3rd attempt - New trial requires no previous use of Xeloda (which I went on in part to meet a requirement of my second attempt to get into the trial).

I continue to hope that I can receive TDM1 without exclusion. That said, I am still searching for something else as a plan b. I am just amazed at the fact that this situation with trial exclusion for incurable patients still stands.

Love, Hope, Peace,
Carolyn

[Carolyns]

Hi StephN,

Thanks for staying in tune with this discussion. Take a look back at a couple of earlier posts by Lani about the discussion at ASCO and the "Dying patients access" link. There is also a post by Schoonder with Dr. Rugo's thoughts on this. There is momentum behind this argument as the science is moving faster than the "process" and too many people are dying because of this situation.

Love, Hope, Peace,
Carolyn

[Carolyns]

Hi All,

For those interested in reading and learning more about this issue the website below is a wealth of information. I plan to contact them this week to see if they have any ideas for me. Wow what an education I am getting. The more I read the more determined I become.

Happy Father's day to all of the Dads out there. I am getting ready to go see mine now.


Link to Abigail Alliance: http://abigail-alliance.org/

Love, Hope, Peace, Carolyn

[Mary Anne in TX]

Joe, thank you for your continued hard work. You and Christine invest so much in what you do for all of us. I surely do appreciate all that you do. ma

[IRENE FROM TAMPA]

Hi Carolyn

I just read your post and wanted to tell you what happened to me. I live in Tampa, Fl. and noticed you are close by. I have been battling breast cancer since 1996 and I really think I have tried every chemo out there. I have also had a stem cell transplant in 1999 and liver abalation and liver resection.

I was on the Tykerb/Herceptin trial a few years ago and that gave me 1 year of stability, but never really tumor free. I heard about the Herceptin/DM1 trial and started that in June/2008 and they can not seem to find any tumors at all (from a diagnosis of 16 tumors in June/08)
I am afraid to even say it out loud for fear of jinxing myself.

My oncologist in Tampa is keeping on top of all of the trials. I go to Florida Cancer Services in Tampa (formerly Bay Area Oncology)

My trial co-ordinator had a difficult time getting me into the trial - first because I had been so heavily medicated, then it was because I had a stem transplant in 1999. She fought very hard on my behalf and finally got me in.

I have now finished my 17th treatment and am going into what they call a continuance and it will be the same drugs as long as my counts stay stable and I do not have any progression. I have been having some low counts to my plateletts and my Bilirubin has risen and they are keeping an eye on that. I can't seem to keep my potassium levels stable but take pills for that. Other then that it is a very easy regimen to take.

Where do you live in Florida? It might be far for you but if you want I can give you my doctors information and maybe you can give a try. As I understand, they might be having a new trial coming to them soon. I do not know the numbers on that one yet but am keeping tucked in my bag of tricks against this horrific disease.

I wish you the best of luck. You can post or send to my email address.

[Carolyns]

Hi Irene,

Thank you for your reply. I have followed your story since I arrived on this site about 3.5 years ago. I am SO happy for you and your results on this trial.

My timing was really good with Tykerb. I remember others crying out when they could not get access. I couldn't believe that this could happen to people. I really prayed that it would never happen to me... Hopefully I will have good timing in this case as well. Joe has pointed out that a new trial with TDM1 will be coming out soon w/ Pertuzimab. My doctor will offer that trial. I hope that it comes out in time and that I am not excluded for some other reason. It is my strong feeling that bad timing should not stand in the way of access to clinical trial drugs for a Stage IV patient.

I am a single parent with a 10 year old boy. I work full time to keep our insurance and a roof over our heads. I don't have anyone to advocate on my behalf or cover for me if I am down. I can't afford to let things go too far because there is no one to fight for me (other than my doctor and friends). Thank you for sharing your story and I will email you if I think that I need your help. Your story is amazing and I pray that you are one of many wonderful people who are leading the way to a cure.

Love, Hope, Peace, Carolyn

[Darlene Denise]

Joe: Looks like you've gone to the dogs! He is a cutie, just not as cute as you! I think I will miss your mug. This is going to take some getting used to!

Has Christine put you in the doghouse?

Thanks for all that you do for us!!

Darlene

[schoonder]

Carolyn,

I believe that if Roche(Genentech) agrees to supply you with the T-DM1 product, your oncologist can file an application to the FDA (form available on the net) for an "individual" IND. Of course the big question here:
is Genentech willing to work with you and for that matter, potentially thousand others in similar situations?
I believe that the company by now is sitting on tons of "interim" data results from T-DM1 trials still actively recruiting and ones no longer accepting volunteers that show how well this compound is performing.

[Joe]

Her name is Belle.

Joe