Off to the NANT

Good morning! Well, it is official. I know have a full house streptococcal twerps and one streptococcal wife. In fact, I am the only one that has not received an official diagnosis. Of course, I am also the only one that has not visited the doctor. Not to worry, I found a left over bottle of Augmentin from one of our dogs past infections. So, I am good to go, and well on my way to recovery. Although, I do have a strange compulsion to howl at the moon. You may laugh but, hey, it is the same antibiotic and dosing that Lynley is receiving.

Tomorrow I am off to Los Angeles for the NANT meeting. I have spent a considerable amount of time thinking about what I will say in my presentation. I have decided to concentrate on 3 areas and I will be pushing for change. I should probably also mention that we (several other parents of children with neuroblastoma) will be meeting with a few of the investigators beforehand to discuss these topics. Our goal is not to dictate change but rather to learn all sides of these issues. Often, what may benefit a single child or single group of children may, in fact, hurt research or the speed at which it may be accomplished. So, what we try to do is gain a thorough understanding of the issues involved so that we may pose suggestions or propose solutions that can benefit all involved.

The first topic I intend to cover is "informed consent." One of the things that the NANT does is blind response data. In other words, no one knows whether prior patients have responded to a clinical trial. Ironically, they do provide toxicity data. So, they tell us what toxicities other children on the trial have experienced. Frankly, they have to do that. However, my argument is that response data should be no different. If they are planning on us (or our children) to assume risks both known and unknown then we must, therefore, also have a concept of the potential benefits. With out that information we cannot provide informed consent. Secondly, due to the fact that we have no way to gauge our prospects of direct benefit this puts human rights protections into play which slows down trials, increases subtherpeutic dosing, and, likely, reduces the chances for children to have a response. At least, these are some of the arguments I intend to make. Over the years, this is one of the only improvements that the NANT has been unwilling to budge on. Frankly, from a scientific point of view and from a data integrity point of view I can understand their reluctance. However, there are other well respected institutions and consortia that provide this data so there must be an answer or a compromise to be made somewhere. I just have to figure out what that is. I know what we want. I just have to figure out how to get it.

The second topic I will be discussing is the issue of human rights protections. The irony is that we are advocating for the opposite of what advocates normally advocate for. We are advocating for greater risks for our relapsed and refractory populations. Yes, you heard me. We are arguiing against the age old medical adage "first do no harm." In fact, I think that if you polled the NANT investigators off the record, some might agree on the need to measure risk relative to the risk of dying from relapse. The irony is, believe it or not, the NANT does not have much control over this. There are federally mandated bioethical rules which dictate the level of risk that an IRB (Institutional Review Board) is allowed to let through. Believe it or not, for the most part, the government dictates that we are prohibited from exposing kids to anything that represents anything more than a "minor increase above minimal risk" AND that the standard for risk is measured against HEALTHY children. This is a ridiculous concept considering the fact that many, if not most kids, are eventually faced with the choice of death or a phase 1 trial. Their risk should be measured against the risk of death not that of a healthy child. Worries about potential side effects are real and we appreciate the concerns but when parents weigh the known effects of untreated NB (death) vs. the unknown side effects of a novel treatment I believe most parents of relapsed/refractory and especially progressive patients would gladly take the novel treatment. These changes in perception could potentially reduce the number of kids who receive subtherapeutic dosing in phase 1 trials, increase trial availability, and hopefully, speed the process of getting beneficial therapies into kids.

Now, I should point out that I am not advocating willy nilly changes in the rules for all kids with neuroblastoma. I am looking for a compromise which takes into consideration the reality of what relapsed and refractory children face. Finally, I want to remind everyone that the NANT is not our problem here. They are on our side. Perhaps together we can create some minor changes that will create substantial change for our kids.

Finally, many parents are clamoring for customized (or personalized) medicine and have asked that I advocate on their behalf. I have to be honest and say that while I think this has potential to be of value in the future, I don't believe it can be put into practice now. Although we have the technology to identify genetic tumor characteristics at will, we still have no idea (or proof) of how we can attack those mechanisms in reality. I think it is an important direction for the future. It is something that we should be considering, tracking, watching, and researching but, it just isn't ready for practice. Frankly, with the exception of one child (who passed due to toxicity of the customized medicine), I am yet to see it work for a single patient in a meaningful and measurable way. I know, I know, I am going against the grain of the childhood cancer conspiracy theorists. I know there are parents that are very passionate about this subject and believe it could help. It looks like it could. It just doesn't - yet - and the cost is significant. I can tell you already that the NANT collects much of this data. I can also tell you that they are hindered by a lack of financial resources to do this at the level that we would all like to see. None the less, I think this is an important discussion and one that I will be putting forth to identify ways that we can potentially move forward more quickly with this idea in a way that is meaningful for kids.

So, probably much more than you probably wanted to hear, but that is the direction that I am going. We discussed many topics that were suggested by other families and we found that these were the most important to get across in our limited time. I am always open to suggestions though.

Well, I had best be off. There is much purpose to accomplish and I have 2 sick kiddos at home again today.