Thursday, February 7, 2008

5:46 AM 2/7/2008

Good Morning! Well another morning has slipped by. I would have figured that I would have achieved more by this point but, well, you can't win them all. This morning I am going to talk about one of the presentations that I heard over the weekend. This may eventually turn into an article so bear with me. You may see it twice. I mentioned that over the last weekend we had a board meeting and that at this meeting we had the opportunity to talk to each one of the members of our medical board to gain insight into the work that they were doing. There were many things that I was impressed with but, one of the ideas was so innovative I just could not get it out of my mind. A year or so ago, Dr. Sue Cohn left a prominent children's hospital in Chicago and joined the University of Chicago. To be honest, these moves scare me because I always fear that, when someone leaves a pediatric hospital to join an institution whose focus is not predominantly pediatrics, they will be lured away from pediatrics. I feel this way for many reasons. For one, they become a little fish in a bigger cancer pond. Secondly, I fear that they will be lured into adult studies because of their experience and successes. Ironically, there are many pediatric investigators, especially in neuroblastoma, that are wooed by adult oncology because of there expertise and successes. Yes, we as parents think that progress does not move fast enough but somehow pediatric oncology achieves more successes more quickly with less resources and manpower than many adult cancers. I digress. The point is I don't like people taking MY neuroblastoma investigators and clouding their minds with stuff other than neuroblastoma.

So, when Dr. Cohn left, I was worried. That has been complicated by the fact that I know of 2 other extremely prominent neuroblastoma investigators that are also on the move and they are moving into mixed (adult and pediatric) environments as well. This made me very nervous. Sure, they told me that they would never leave pediatrics, that they would never leave us. But, still, I worried. How was this going to help kids with neuroblastoma? Well, it was talking to Dr. Cohn that I found out.

One of the problems with pediatrics is getting new drugs into kids. It takes too damn long. First, a new drug has to be developed. The bulk of them are tried on adult cancer lines (this is where the money is) If there is activity then they do all of the preclinical testing - first in Petri dishes and then in mice and rats. At this point they can write the trial. It goes through a review process and all kinds of things that can take an inordinate amount of time and then, finally, it can go into an adult. This entire process has to occur with pediatrics as well. It is double duty. In fact, it is even more than double duty because we have to be even that more careful with kids. There are all kinds of stopping and starting rules and it takes forever to get anything done. This process can take years sometimes decades to get a new drug into children.

This is where Dr. Cohn comes in. By the way, there is actually an article on this in the January 10, 2008 edition of the Journal of Clinical Oncology. The article talks about a new trial design called rolling 6. It gives a great methodology for speeding trials by avoiding most of the stopping rules. In essence, it has the possibility of make the drugs available to more kids faster in a trial setting. This is all fine and dandy but not what really excited me. What really floored me was how she was getting drugs into kids in the first place. She does not really get into it until the last paragraph of the article but it is nothing short of brilliant and probably one of the best strategies to get drugs into kids since time began. What they have done is this. When they are testing the drugs preclinically for adults they are also looking at some pediatric (like neuroblastoma) cell lines. When there is activity they combine this data into the adult trial. They then open the adult trial. Once they achieve the maximum tolerated dose in the adult trial they then expand the age requirements to include pediatrics. First they start with kids age 14 - 17, and then younger kids, and so on. By doing this they are effectively cutting years out of the drug delivery process. They are saving dollars, man power, time, and most importantly they are probably sparing populations of children that could be potentially under-dosed. The key is that they are getting drugs into pediatrics much earlier than was previously possible. Furthermore, we get a glimpse into a drugs activity long before we would have normally. This is a huge step forward.

Now, many may say that most of the kids are too young to benefit from this strategy. For the most part I agree. However, the young children will still be likely to receive the drugs much earlier than normal and with a better indication and more refined dosing. It stands to benefit them as well. Secondly, it is also important to note that many of the kids that relapse are much older and could benefit directly from this strategy. Finally for the group of older patients that we have right now like Erik, Anna, Joe, and Nick. These are great options for them, right now. In this sense, they will be eligible for so much more.

Now is this system perfect? No. Is there potential for problems? Yes. But, I can tell you for sure, this will speed quality drugs into children with neuroblastoma and is doing so now. Kudos, Dr Cohn! Thank you for staying with us.

Purpose comes in the strangest ways.

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