Wednesday, July 16, 2008

Really Cool Research

Good morning! First I want to thank everyone for their help with perusing the handbook. No offense, but you are a truly easy group to impress. Either that, or you are just so thankful to have this resource available that you can't possibly think of anything negative to say. Well, I feel the same way. Regardless, I am still open to constructive criticism if any would like to throw some my way.

I am busily getting ready for the conference. Over the last few years it has become increasingly more evident that while everyone appreciates the CNCF no one seems to truly understand what we do, what our priorities are, or how we do what we do. I think part of that is our fault. We are so busy doing that we often don't spend adequate time telling everyone what it is that we have been up to. So, in a nutshell, I have been tasked with that duty.

This is no easy task. I am tremendously proud of all that CNCF has accomplished. This really should not come as a surprise. After all, I am a pretty active neuroblastoma guy and I have decided to dedicate my life to this organization. So, it must be important. They must do important things. But, what are they? Do you know? Of course not, you have not been told. And that, is my job.

The problem becomes, how do I boil all of this passion and information down into a few minutes of a presentation? How do I adequately cover all of the important things that we do? Thankfully, most people already know what we do for education. After all, we do put on this humongous conference for families. But, we also write educational articles, provide educational seminars, provide discussion forums and a home for the neuroblastoma handbook. In fact, when you get right down to it, I don't think there is one of us that you could not call at 3:00 AM and would not gladly try and talk you through anything that is neuroblastoma. Also, I can guarantee that if we did not know the answer we would call every expert necessary to find it out. Bottom-line, I think these are all examples of what we are relatively well known for. It is because we are out there in the forefront and anyone can see these efforts.

Ironically, it is just a sliver of what we actually do. My job is to bring the rest to light. What are the other things we do? Where do I start?

Well, if you have come to know me at all you must know that research is one of the things that drives me the most. In fact, secretly, this is probably the most important to me. Knowing that research is that important to me and that I have dedicated myself to this organization you must then begin to realize that it must have a key research component. And that it does. I am extremely proud of the research that the CNCF funds. I don't think people realize what we fund or why. Well, I know that people think we fund the NANT and MSKCC and I am pretty sure that they have an inkling that we have this thing we call a Junior Investigators program but, I don't think anyone truly understands the impact of what and how we fund. Frankly, if they did, it would not be rational for anyone to put their dollars anywhere else. I have to get this across. This is were you come in.

Now I can handle talking about our initiatives with the NANT and MSKCC. Those research investments are bulletproof. Furthermore, when I explain how we fund them, I think everyone will be blown away with what a critical funding piece we are. For example, did you know that the CNCF pays the patient research costs for more than half of the patients that are put on a NANT trial? Did you know that in some cases we have even committed to pay for the drugs utilized? Yes, NANT, the largest phase I and II consortium providing neuroblastoma clinical trials is able to put kids on trial because of dollars the CNCF provides. I bet you did not know that. Furthermore, did you know that the NANT has provided 14 different clinical trials over the last 8 years. It is an incredible efficient and light weight organization that, dollar for dollar, is simply the single best investment in research around. But, I digress. I already told you that that was the easy stuff to get across.

What is more difficult to explain is our Junior Investigator Program. With this, the CNCF funds many, many studies - and they aren't inexpensive either. For example, just this year we funded 4 more 2 year $100,000.00 grants. Think about that. And, I bet you don't even know what we spent that on. What research? You see the problem. That is what I need to tell you about. At any moment we may have as many as 10 of these different research projects going on and no one has any clue (a) that we are doing them or (b) how totally cool this research is. Now my trick is to pick a few of these and tell everyone about them in a way that will make sense to everyone. That is no easy feat. For the most part these are all complex studies chock full of medical jargon. So, once again, the price for reading my personal diary is that you are going to have to listen to me talk about a few of these studies so that I can practice. They are incredibly important and innovative studies but, unless I explain them correctly, you will never know how truly great they are. So, here I go. My first stab at one of our most recent studies.

B7H3, an Immunological Target for Neuroblastoma. This study is brought to us by Hong Xu from Sloan Kettering. This researcher is being mentored by Nai-Kong Chong, the father of the 3F8 antibody. So we have a pretty good indication that this research is already going to be exciting. Regardless, this is what you need to know about this incredibly cutting edge science.

Cancer is able to survive in people because it is able to evade our immune systems. After all, our immune system is the most effective weapon in the world, it is better than any drug. The question becomes: How does cancer avoid being ravenously murdered by our little white blood cells. Well, the common belief is that there are little proteins that sit on the surface of cancer cells. These proteins essentially paralyze our killer white cells. This is how cancer survives and grows. Think of the cancer cells spraying freeze spray at the white cells. The white cells are immediately frozen and can no longer kill the neuroblastoma cell.

Researchers at Sloan Kettering have had much success in treating neuroblastoma brain metastases with an antibody called 8H9. They have recently discovered that this antibody attaches to protein on the surface of a neuroblastoma cell called B7H3. The belief is that this protein is one of those that is so good at paralyzing white blood cells. Unfortunately, we don't understand exactly how these proteins work and how they turn off and paralyze the killer instinct of the white cells. We don't know how the white cells receive these signals. We believe that there must be some type of receptor on the white cell. This study will (a) identify what these receptors are and (b) identify antibodies that could be used to block these signals.

Now, this is incredibly innovative and incredibly important to the neuroblastoma world. Having a thorough understanding of exactly how cancer cells evade the immune system is very important. Furthermore, enhancing the activity of natural killer cells has implications for all different kinds of therapies in neuroblastoma. This very well, could be the key to unlocking many doors. This is important research that I am proud of. In fact, I would say this is a critical study to fund and I would stack it up against any other study in need of funding anywhere in the world. Wouldn't you agree? What is more important? Do you know of something?

Okay, so how did I do? That was just one example of many of our research projects that I plan to cover. Was it easy to understand? Do you see its relevance? Do you understand my excitement? Did you know we were doing this research? Are you blown away? Am I just nuts, (Don't answer that one?) or is the some of the coolest research you have ever heard about?

Once again, I would love your feedback.

I have purpose from the bottom of my feet to the tippy top of my head.


Raelene Soritau said...

I was blown away by the depth of information available and the easy read format,breaking everything down in small sections for the reader to digest. This is especially important for families first diagnosed since it is a time of extreme overload and anything to make life "easier" is very much appreciated!!!

Charon said...

I totally understand your description and the importance of this type of study. NB is so aggressive and can create so many tumors in so many parts of the body that I understand the importance of trying to figure out what these single NB cells do and how they operate. Having a NB handbook for current and future patients is great. Thanks to you and the CNCF for your continued dedication! Charon Edgington

themacdonnells said...

The handbook was great. I learned a few more things about NB that I didn't understand before. Nice work.

And, the new study sounds amazing and what you wrote was very easy for me to understand. Keep up the great work and fight for our kiddos!

Heather MacDonnell