A unique trip to the NANT

It is good to be home. I always miss my family tremendously when I travel. What can I say, I am a rather sappy advocate. However, I must be honest and say I wish this one could have continued on for a few more days. I had so much going on peripherally at this meeting that I missed several of the presentations that have traditionally been some of my favorites. Not to worry though, for the most part I had seen them and heard them all before. Additionally, the activities I was engaged in were far more important in my eyes. This was my fourth trip to the NANT and although structured as all of the others was quite possibly the strangest and most intriguiing of all of them.

Going into this meeting, one of my goals was to get a firm understanding on the support for a drug combination including ABT-751 and Fenretinide. I know that to many this would sound very simple. Here we have a very interesting (remarkeable) combination of drugs. Individually they have activity in children. Together the preclinical research predicts there to be substantially more. In fact, this additive effect has even been duplicated with other drugs that share this same mechanism of action. You weigh this exciting data against the fact that the protocol has been written and ready in the NANT for about 2 years and you would think this would be in kids today. What is the problem? Ultimately the problem has been that the pharmaceutical company would not provide the drug. This is further compounded by the fact that the preclinical work has been well received by well respected peer review groups. It was even awarded as one of the poster presentations at both the ANR and ASCO conventions, 2 of the top respected research conventions in the cancer world. Both the combination and the drugs individually have also received kudos by the NCI as being great examples of translation. If all of this was not enough I will tell you from my "laymen, father of a child with cancer who is nosey, neuroblastoma researching, purpose-devoted" perspective that there are several characteristics of this preclinical research that are so promising that I have never seen them before in any research that I have read. So, given all of these points, I still wonder what exactly is holding up the drug. It isn't the quality of research. There is something else at play here.

Before I get into this I don't want to be an alarmist. This combination is not the cure for cancer. I do believe it is promising. I do believe that it will work well for some kids. I think it is important and I believe it should be in kids right now. But, let's just keep it in perspective.

So, now, let me tell you what I know. I can tell you that there is general support from the medical community. In fact, I talked to many researchers in private one-on-one conversations on this topic. Furthermore, I can also tell you that I did not talk to one single investigator that was not in support of the promise of this combination or in moving it forward.

In fact, there is so much support for this particular combination that it will be discussed in the next scientific review committee meeting within the NANT to identify what the next best move is. I will be honest and say that I went in with the intention of coming out with a signed letter of support. However, in light of several new happenings, it is clear that discussion needs to take place before the combination is moved forward. These are recent findings that need to be carefully weighed and considered before it is decided whether this combination will move forward as it is currently planned or if some changes will need to be made. I can also tell you that these are not the issues that have held up the trial previously. These are simply new and emergent issues that need to be addressed and one of the reasons that made this particular meeting so unique.

So, to make a long story short, it will take a bit longer to get to the bottom of this issue. But, coming from somebody that wants to see this move forward as quickly as anyone else, I can also tell you that this is a necessary step.

Wow, I finally made it through that and that was just one of many of these types of issues. I will spare you the other for the time being. I guess the only thing that I should probably address right away is the rest of the meeting. People often wonder what we hear in these close door meetings. Do we know how patients respond? Do we know what works?

In short, no and no. And frankly, even if we did, we sign confidentially agreements which preclude us from discussing much of the meeting. With that being said there are things that can't be "legislated." This is what I think is the most important point. It is from this perspective that I can tell you that I am completely impressed with the work of the NANT and the decisions that are made. The trials currently available in the NANT and the ones that are likely to come give me significant hope and appreciation for all of the investigators and researchers that work so hard on our behalf. I am routinely blown away by their thoughtful consideration of our children and their commitment to move forward in a thoughtful and meaningful way. I can tell you from my perspective that every single one of their trials is not only meaningful from a scientific perspective but also for our children that need options now. They face difficult challenges and extremely hard decisions.

Yet, they always remain extremely committed to our children. In fact, it is that commitment that is the reason that the five parents of children with neuroblastoma participate in these meetings. They want to be informed of our needs and concerns and they want to move research forward that is meaningful to us.

Finally, what makes this group such an important group for our children is their commitment to get the prioritization right. You have heard me harp on that subject to the point of exhaustion but it truly is the key and the NANT has it right.

There will be more to come throughout the weeks.

I have never felt purpose so strong.