Wednesday, July 15, 2009

Neuroblastoma Research Conundrum

Good morning! This diary comes as a much needed break for me this morning. My eyes have begun to cross after reading mountains of research proposals. Don't get me wrong. I love reading neuroblastoma research. The problem is that I don't like reading all neuroblastoma research. Like any normal person I enjoy reading material that is interesting to me. Unfortunately, all neuroblastoma is not necessarily interesting.

My other issue is that with everything I read I am always reading it from a scientific perspective. Is it realistic, is it relevant, is it good science. I am great at punching holes in theory and assessing translational impact. However, with this group of scientific proposals, I am having to interpret it from the perspective as a patient advocate. I have to assume that the science is good and factual - frankly, I know in several cases it is not which is thoroughly irritating. Furthermore, in the scope of this review, it does not matter what I think. The perspective that I need to use is from the population of all patients and caregivers. What would they think? That is not easy for me. I am much better at what I think. Regardless, the point is that, I am having difficulty interpreting it from that perspective. I am not having nearly as much fun as I would ripping the research apart.

I know, sadistic, but true.

That is how I do it. I find promising research that I believe is meaningful to children with neuroblastoma and then rip it apart. I identify important questions and then evaluate the proposals ability to answer the question clearly. So, at the end of all of this, we actually have a meaningful answer. To me, that is good science. I then also assess the impact of potential new therapies on patient populations. I answer questions like. If this works, how meaningful would it be? Can it actually get into patients? How quickly? What is the cost (toxicity, etc.)? Will the drug be available? Would it be a good use of resources or would those dollars (and patients sadly) be better spent elsewhere?

As srtraight forward as that two step process seems to be it is more complicated. The answers aren't as clear as they seem. How do you factor in priority?

Ultimately, is the most important question: what has the most promise to make the most difference the most quickly? Or is it, what has the most potential to make the most difference in the long term?

There are drugs out there that could have an impact in the clinic right now. Potentially, they could save lives. However, they aren't the cure for neuroblastoma. They are just another tool in the tool belt. Do we prioritize those? Are those more meaningful? Or, do we prioritize research that does not have applicability in the clinic right now but holds significant promise in making a substantial difference in survival down the road?

It isn't an easy question. I know from personal experience that parents of children with relapsed neuroblastoma want treatments now - almost at any cost. Unfortunately, that does come at a cost. By pursuing that idea we are potentially losing out on another long term success.

Is it more important to find the genesis of neuroblastoma so we can learn how to stop it from the very beginning? Do we learn more about the stem cells and how to kill them? Or do we just focus on what we can kill right now?

I know what I think. The difficulty is in understanding what others would think?

Since I don't know the answer to that question it leaves me in the position of identifying something with a little of both - something that could have substantial impact and has a clear path to our children?

Seems simple, but you have no idea of how tall an order that is. Frankly, I don't know that I see that in this particular mountain of proposals.

Back to purpose.

No comments: