Prioritizing Neuroblastoma Research Part 2

On Wednesday I began talking about the issues with the slow pace of research. This seemingly slow pace is present through all of cancer research. To be honest, if you compare neuroblastoma to many other cancers you will find that neuroblastoma is moving forward much more quickly than most. In fact, for some it has been held up as a model of success. I know, as parents, it certainly does not seem that way but, in reality, it is the case. Still the problem remains, it isn't fast enough for our children and probably not for the ones that will be diagnosed tomorrow.

For neuroblastoma the problem is compounded by two factors - money and subjects (yes, our kids). There is only so much research that can be completed in any one year.

Our basic science research is limited by funding. There are only so many drugs we can test in petrie dishes and mice and there are only so many things we can do to understand neuroblastoma cells and how they work. That is a fact of life. We have plenty of researchers but only so much money to do the research. We have to be smart about which drugs we study and that we are only looking at the most promising basic science factors.

On the clinical trial side we have two factors that come into play. One is dollars. We could use more money for clinical trials. There are many promising trials that aren't available to our children because they are cost prohibitive. The other factor though is the one that probably slows us down the most. It is the fact that we have limited patient populations. In any given year there are only so many kids that we can put on trial. Period. That means that we can only have a limited amount of trials. If we have too many trials it takes years longer to complete any one clinical trial. This makes research take longer. Secondly, if we aren't providing the clinical trials with the absolute most promise then another clinical trial is holding up that promising research from moving forward and for those drugs to get into our kids.

It is for these reasons that we must prioritize research and clinical trials. We must ensure that only the most promising research is carried forward and only the best of those ideas make it to trial and our kids.

How do we do that? How do we prioritize research?

This is where researchers get a bad rap from us parents. Researchers keep doing all of this preclinical research. They keep testing and testing and testing. We just want to get it into our kids already?!?

You see, preclinical research is used as a predictive model for what will happen in our children. It is far less expensive to do preclinical research and the goal is for the researchers to to find out as much as they possibly can about a drug before they put it into our kids. This is part of the prioritization process. They want to insure that whatever idea is moved forward has the most robust data and holds the most promise. They want to make sure that it is better than all of the other options and they want to make sure that they are delivering the therapy optimally. This is one of the ways we speed up research. It makes sense.

Now, you may think that all of this talk is theoretical. It is not. I see examples of this everyday in the neuroblastoma world. Today, we have clinical trials that are open and accepting patients that are clearly not the best options out there. They never went through thorough preclinical research process. They were never prioritized. I can think of at least one trial right now that will cost us nearly $1,000,000.00 that is clearly not going to move research forward. Over the next 2 or 3 years it will absorb over 80 patients. That research is wasted - dollars, patients and years. Those resources could be used on something far more important- far more hopeful. Instead, for the next three years, we will be wasting valuable dollars and patients on research that is less promising than much of what is sitting in labs today.

That frustrates me. What a waste.

There are also very real examples of drugs and therapies that have been moved to patients well before we knew enough about how the drugs worked. I can think of one in particular that parents were clamoring for. It was a drug which had shown good preclinical activity but could not be replicated. Researchers were called every name in the book - publicly. The researchers would not open it up in trial and blocked most efforts to do so so that it could be studied further. They succeeded. After about six months they discovered that the drug needed to be administered at much lower doses to be effective. The drug is now in trial at those lower doses and is effective. However, had the researchers listened, not done more preclinical research, and just moved the drug to trial; it would have been administered at higher doses which would not have provided the activity level needed. It would not have worked. We would have wasted money, patients, and years.

I am not saying that prioritization is the only answer. There is more that has been done and it isn't a simple answer. It is a complicated question.

But we are out there fighting.

Everyday.

With purpose!