DM1 trial stopped and I may want to fight it - need advice!

[eric]

As I've written, Caryn was having great results on this drug before side effects caused her to miss 5 out of 6 treatments. After some persistance, Genentech agreed to lower the dosage from 2.4 to 2.0 and she had her first treamtent 3 weeks ago on the new dosage. She was not allowed treament 2 weeks ago due to high pancreatic functions but everything was good last week and she received treatment on Friday.
On monday she had her scheduled scans and the CAT showed growth in the liver from 3.4 to 4.9. Considering that her prevoius scan 4 weeks ago showed a "dramatic reduction" in the liver, Caryn and I are shocked that they've decided to pull her from the study when, it seems to us that, inactivity could explain the growth. The doctor at Sloan feels strongly that we have zero leverage in asking Genentech to reconsider but I'm thinking of calling anyway. Does anyone have the experience or knowledge to determine if my argument is sound and worthwhile? Obviously, I only want to fight to keep her on the drug if the possibility exists for it to still help.

[Jackie07]

Eric,

They evidently want fo keep everything stict for the purpose of their statistics - they want their study to be valid. The only way to fight is probably to get a lawyer. But even that might not help. See if your state has a Young lawyers Association through which you might find a not-yet corrupted lawyer to do a pro bono investigation into the situation.

[chrisy]

Hi Eric,

My experience with this trial is that the protocol is very strict, because as Jackie says they need to have a valid study, and because it is truly an investigational drug so they really need to be cautious. However the protocol is ALSO quite complicated. And there is some wiggle room sometimes where there are gray areas.

I think the key is to get the clearest understanding of 2 things:

1. What exactly does the protocol say about being discontinued from the trial due to progression? Is it progression of a certain "%" size and what is that. And how does Caryn's CAT scan result fit into that.

2. Is this drug working or not? You may have a valid question on whether this represents progression while ON the DM1 or progression due to NOT getting it. Can you get clarification from your doctor on this point - is the progression an indicator that the DM1 WAS working (see how it grew when she wasn't on it??) or that it WAS NOT working (see how it grew even when she got the DM1???). If you can reasonably say that Caryn was getting clinical benefit, that is where there may be some wiggle room for continuing, if her doctor supports this.

As Caryn has had significant adverse side effects throughout, and now possibly evidence of progression while on treatment, you may not be able to successfully argue for keeping her on the trial. On the other hand, if both of these things are the reality, perhaps this drug is not the right option for her despite all our hopes.

Remember, although Genentech certainly wants to have a valid trial and of course hopes the trial is "successful" in terms of demonstrating the safety and efficacy of the drug - they also (I believe this very strongly) want to do what's best for the patient. As does Caryn's doctor.

My thoughts are with you, praying for the right decisions to be made by all.

Much love
Chris

[Yorkiegirl]

Eric I don't have any information for you and Caryn, but I will certainly say prayer's that things will work out for her.

[Lani]

her met wasn't bigger and was subsequently shrunk by the reduced dose she did finally get.

Perhaps the best you can do is ask for a repeat scan to see if the tumor is continuing to grow or continuing to shrink (don't know how long it takes the drug to have its maximal effect), with a request to receive additional drug if there is not progressive enlargement

How to time that scan would be the question. Perhaps an onc who has had several patients on the trial could give their opinion.

In light of the serious opthalmologic side effects Caryn has had, I think they
want to be sure they don't seriously impact her quality of life (ie, visual dysfunction) and probably

There is such a thing in general as "compassionate use" allowance, but I doubt for such a new and unproven medication

It is sad to say but they are probably shying away from continuing Caryn as visual disturbance is something which might preclude FDA approval in the future--these drugs are considered something which prolongs life rather than cures, thus significant side effects showing up in the small early trials can be enough to incline the FDA not to approve the drug.

Nasty business, this. In the best of all possible worlds, it would be the patient who got to decide what risks they would and wouldn't agree to take.

My best wishes that you resolve this --I think there are some interesting trials eg. agamma secretase inhibitor against cancer stem cells, as well as trials of a heat shock protein inhibitor ,and perhaps a trial trying to combine Pertuzumab, herceptin and perhaps Iressa (not sure about the last), to look into proactively while you wait for things to sort themselves out

[eric]

Thank you each for your response. It is helpful. Once we got the information from Sloan, we put a call into Caryn's NJ Onc who, we felt, could offer us a different perspective. His reaction was very interesting. Not only did he validate our concern with the logic of pulling her from the trial so quickly (while understanding it from Genentech's perspective), he's questioning wether there has been real progression (as both Lani and Chrisy pointed out). He's insisted on seeing the films and blood results before determining what our next best step is. His feeling is that reading scans is not an exact science and the perceived increase from 3.4 to 4.9 could in fact be no increase at all. Our plan is to get him the info he's asked for by next week and meet with Sloan on Tuesday to discuss their new trial suggestion. If her NJ Onc feels that there's a reasonable argument to continue the trial (Lani, I love the idea of asking for a re-scan) then I will ask him to support me in contacting Genentech (as you suggested Chrisy) to discuss this directly rather than relying on Sloan to lead our argument. If he doesn't then we will look into our next course of action including the options suggested above.

Thank you!
Eric

[Faith in Him]

I don't have any answers for you but I admire your determination to stay on track with your wife's treatments. Please know that I will be praying for you both.

I also love the support you are getting from our sisters on this site. This is such a wonderful support group.

[Joan M]

Eric,

I'm praying that everything works out to you and Caryn's satisfaction.

Best, Joan

[Barbara H.]

Hi Eric,
I agree with Chrisy's and Lani's thoughts as well as the thinking of your oncologist from NJ. First of all, Caryn skipped so many treatments that it is not surprising that there was some progression. It makes since that she should be allowed to resume treatment to insure that the progression was from lack of treatment, not from her inability to benefit from the drug. I spoke to my research nurse for the trial today and she could understand my point. It is worth a try to persuade Caryn's oncologist at Sloan to allow her to have some more treatments to prove that she is truly not responding. Perhaps she will also get a resposne.

It has also taken by body a while to adapt to this drug. In the beginning I had to skip treatment every three or four weeks because my liver enzymes would be too high. They were really jumping around. Now they tend to inch up with each treatment, but they are currently much more stable.

Best wishes and good luck!!!

Barbara H.

[Jean]

Eric,
Kudos to you for not accepting the first response.
When they say it is a battle no one really understands except those who deal with all aspects of this disease.

Lani and Chrisy have both offered great advise (as usual) and it appears your NJ dr. is also questioning
the scans, which makes perfect sense. It just seems a bit early in the game to call it.

I just wanted to wish you the very best in this new challenge and will keep both of you in my prayers.

Please let us know how all goes.

Regards,
Jean