Wednesday, May 13, 2009

So I am in a tizzy, eh?

Good morning! So, yesterday I received a ton of email. It was very supportive. Well, that, or emails from people curious as to which trials had me all in a tizzy.

I am not going to mention them.

I really genuinely hesitate mentioning any of the trials. Yes, I personally believe some are worse than others. I believe from the standpoint of helping a child with neuroblastoma there are many options that at many times are completely inappropriate. To me, it is much less about the actual drug or trial itself and far more about the patient, the disease, and the timing. For example, everyone knows I believe in antibody therapy. It is no secret (and by the way it is now proven by a phase III trial.) However, there is a time and a place for antibody therapy. There are points in therapy where it works best. It works when you have minimal disease. It works when you have marrow disease. It works to a lesser extent on bony disease. However, given current medical technology, it is absolutely useless against solid tumor.

So, I would say that I think antibodies are probably a worthwhile consideration if you have have minimal residual disease. However, if you were going to use them with a big tumor in your child's belly and you were expecting great success I would be concerned, very concerned. I would tell you to talk to other parents of children that used antibodies with big tumors. I would tell you to review the research and I would tell you to talk to an expert. I would hope and pray that you would pick a different direction - at least initially. This is because, chances are, the antibodies would do nothing but cause pain and give the tumor some opportunity for progression.

So, that was a hypothetical situation but, believe it or not, over the years I have actually seen that decision made.

No, my concerns were with current trials. It was not so much that the trials were bad or the drugs were bad but more that the timing of the therapy was inappropriate either due to the treatment itself, the toxicity, or the dosing level of the trial. Finally, there are also a few trials out there that are very popular but with which the data simply does not support. There are a few trials out there that parents have made incredible claims that are flat out false. They have literally claimed that incredible responses that were never proven.

That bothers me.

That is why I always suggest that people look further. This is why I ask people to ask questions. In many of these cases these miraculous "responses" were actually quickly followed by death. In many cases these responses were not actually considered responses by the medical community. They sounded good but they were ultimately nothing. Remember, changes in HVA/VMA alone do not constitute response unless they are hugely significant. Second, solid tumor necrosis needs to be confirmed by biopsy. The list goes on.

Just be skeptical. Ask questions.

Step purposefully.

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