Tomorrow I leave for a trip to Washington D.C. to finally discuss much of this neuroblastoma research that I have been reviewing. You may remember about a month ago when I was complaining about the quality of this year's latest round of research. Yes, that was depressing. However, this latest round has been far better. In fact, I would even say that some of it bordered on exciting. I am seeing some neuroblastoma research finally moving forward that I have been waiting on for years. Frankly, I am ecstatic to see the proposals out there competing for dollars. Whether they will be funded, is difficult to tell, but I could not be happier to see the ideas out there in written form. While it is not a guarantee that the ideas will be carried forward, it certainly means that someone is feeling strongly about them. Overall, I would say that the research that has been brought forward this year is better than the last, although I can still see room for improvement.
In general, one of my greatest frustrations continues to be the focus on these druggable targets in which there is no drug. You see, one of the great things that researchers do is exploit weaknesses. In fact, they are pretty good at finding these weak links in neuroblastoma cells. I can't tell you how many proposals I read where another investigator has defined a new theoretical way to kill a neuroblastoma cell. With new technologies now available to identify these new targets it seems everyone is interested in exploiting a newly found method of killing a neuroblastoma cell. The problem however is that even though the researchers believe that they have identified a new way to kill a cell, none of them seem to have a drug available that can actually do the job. That means they have to create the drug and that, unfortunately, means at least a decades of work - a long time to see if the original hypothesis will hold true in kids. And, that is only possible IF they are able to create the drug and IF the drug can actually be tolerated by humans and IF they can actually find a company to make a drug for a little known cancer called neuroblastoma. With this methodology you have to hit a home run on your first swing. Anything less is wasted effort - years of wasted effort. The only way the idea will make it to reality is if it is the cure for neuroblastoma or if it has applicability in another cancer.
Don't get me wrong. I know new discoveries are exciting. But our kids just don't have the time to wait. This type of research model just doesn't work for neuroblastoma and it never will. The only hope we have for any new drug development is that it works outside of neuroblastoma because unless it is the silver bullet (and it will probably still be a problem even if it were the case) it will never, ever make it to reality. Neuroblastoma is too small a market and we simply do not have the resources to create our own drug. Even if we do, it will not happen in any reasonable amount of time.
We have to be more creative. We have to either focus on targets that exist in a variety of different diseases or focus on druggable targets in neuroblastoma where there is actually a drug in existence. I wish it were different. It just isn't.
This is a case were pure purpose still isn't enough.