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

[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