Thursday, August 27, 2009

TPI-287 on my radar

Good morning! Aaargh! I am way behind. I sit behind a mountain of email and I am drowning. Email is a good thing but my problem is that I like to really answer every one in detail. I am not a copy and paster. I try and answer each email individually. This means it can take a significant amount of time to get through them. Earlier this week I poked my head out of my secret little world to do some advertising for the neuroblastoma seminar that the Neuroblastoma Foundation had yesterday and that seemed to open the flood gates of questions. Don't get me wrong. I love answering them. I am just at least a day or 2 behind. If you have an email sitting in my inbox please know that I will get to it.

The good news is that, aside from a few small technical difficulties on my part, the seminar went very well. I hope to have a video up by the end of the week. The TPI-287 study was one that I knew about but I did not have the level of knowledge that I felt comfortable sharing with other people. So, for this reason, it was a learning experience for me as well. I was extremely pleased to learn more about it. I can tell you that I certainly have more confidence in it as a potentially therapeutic option. There I went and did it. I used the term therapeutic in reference to a phase 1 trial. I am sure the human rights protectionists and bioethicists now have me on their hit list. Well, I don't care. Frankly, if you have a phase 1 trial that has already shown some significant activity in its first and a "half" cohorts of patients it is worth considering from a therapeutic standpoint - just don't forget to look at the potential side effects.

That was the point that stayed with me. I was skeptical going into this (as I am with all trials) especially considering the fact that this was a taxane, a class of drug with questionable activity in neuroblastoma. However, this one is special. It addresses many of the issues related to neuroblastoma resistance to this class of drug. That did catch my attention and the preclinical activity did seem pretty impressive. Usually I can tear the preclinical work down. IE. they weren't using good enough cell lines, the oxygen tension was too high, the drugs were tested well above potential therapeutic levels, etc. Bottom line, I was prepared to delve further into these questions.

However, once I saw the responses in patients, I must admit that I threw much of that by the wayside. You see, my opinion is this. If you have activity in kids with high risk relapsed and progressing neuroblastoma, does the preclinical work in petri dishes and lab rats really matter? In my mind, only if the drug cracked the petri dish or killed the rat and in neither of these cases did it do that. It is for this reason that I found myself chalking this trial up to a "consider." It should be on people's radar. I will let the seminar speak for itself and I will be sure to post a link once it gets through editing - yes, I have to do that too.

Purpose abounds!

1 comment:

Anonymous said...

Hi Mark! You have a great blog - wishing you and your family all the best!
I just had a question for you in regards to ZD6474 (MD Anderson). Have you looked at this trial? What's your opinion?