5:15 AM 7/27/2008

Good Morning! I am back from my trip to Houston and, other than being in the doghouse, the trip went superbly. Lynley is not a big fan of the traveling, even if I am back by 6:00 PM the same day. I don't know that I learned a lot yesterday but it certainly was a good refresher course. Dr. Russell gave a basic science discussion and described two of the ongoing research projects that are being worked on in the lab at Texas Children's. It wasn't by any means an overview of all of the work that they were doing with neuroblastoma but it certainly was a glimpse into some of their most promising projects. The first was an interesting new approach to solving the N-MYC problem. Just about everybody in neuroblastoma knows about N-MYC in neuroblastoma. It is an indicator of how aggressive a particular child's neuroblastoma is. For example, Sydney was N-MYC amplified. That is not a particularly good sign. In fact, it is a bad indicator. But, what it means is that the genetic material (the DNA) of her neuroblastoma cells included extra copies of the N-MYC gene. So, what? Well, N-MYC is believed to control cell growth. The more N-MYC you have the more aggressive and faster growing your neuroblastoma is. So, what the researcher at Texas Children's did was look at N-MYC and see what else seems to be out of whack when N-MYC is amplified. They found that when N-MYC was amplified MDM-2 was increased as well. When they looked closer at MDM-2 they discovered that it suppresses p53. p53 is a well known to regulate cancer cell death. So, if you loose p53, your cells are resistant to dying i.e. they lose their ability to die. So, there you have it, basic science. Increased N-MYC, increases MDM-2 which decreases p53. The less p53 you have the harder it is to kill neuroblastoma. So, Texas Children's has looked for a drug to suppress MDM-2. The good news is that they have found it in a drug from Roche called Nutlin. The bad news is that the drug is only available for research (not for treating patients) and it does not look like that will change. The other hint of good news is that there is a new version of Nutlin coming that will hopefully be better and there are other pharmaceutical companies that are working on other MDM-2 inhibitors. Now, to make this apply to our kids they have shown that in combination with certain chemotherapies Nutlin makes them up to 10 times more effective. This would mean that we would likely be able to dramatically decrease the amount of chemotherapy that we give to our children while still increasing the effectiveness. This is important science but unfortunately still quite a ways away from being in our children.

The other research presentation she made was interesting as well. I have spoken about it briefly before but they have discovered another surface protein on neuroblastoma. However, I think I have made this entry technical enough for today and I think I will end it here and save that discovery for another day. The great news is that all of the rugrats are fine and they were happy to see their Daddy back home yesterday evening.

It was another good day in purposeville.