Neuroblastoma seems to come in spurts. I know that there is nothing statistically significant but I have always been surprised by diagnoses. Many hospitals will have only a few diagnoses one year and then have 10 the next - and all within a few months of each other. It always seems strange but it is consistent enough that I know it is just the way things seem to happen. This seems to be true with other things as well. I can tell you that people contact me about neuroblastoma in the same way. One week I will have one person email me and then the next week I will have 10 contact me within a few days of each other. I don't know that this has any relevance in the world but it is something that has always amazed me.
What can I say? I have a simple brain and shiny objects excite me.
This week I have had some extremely interesting questions and what I would consider a rash of parents contacting me about "non-responders" and/or refractory kiddos. These kiddos and families are generally the ones that I often grow the closest to and know the best. This is probably simply because I get to know them the longest. In fact, 3 of my 4 best friends in the neuroblastoma world are parents of refractory kiddos. So, in that sense, they are close to my heart.
Refractory kiddos are primarily those kiddos that have stage 4 high risk neuroblastoma that partially respond to therapy but despite all of our best efforts don't ever seem to achieve remission. Their disease seem to take root in their bones and, like the gum that Ainsley left on my favorite comforter, never seems to go away.
It seems to me that about 20% of the high risk neuroblastoma population falls into this category. They finish the 5 or 6 rounds of the high dose induction and they still have this stubborn disease lingering in the bones. Some end up having MIBG therapy, some go through transplant, some go straight to antibody therapy, and some don't do anything at all - all with differing amounts of success. Thankfully, some of these kids will eventually achieve remission and go on to live normal disease free lives. Many others will never achieve remission, yet, they will go on for years, moving from one low dose therapy to another with this smoldering stable disease. Some will find minor improvement with various low dose phase 1 trials but most will just continue on a stable path.
I know what you are thinking. What made the difference. Which therapies worked?
All of them? None of them? Some of them?
I wish I had an answer. It isn't that simple and I have not discovered one simple solution. With the exception of the 'do nothing' population, I have seen them all work and I have seen them all fail with no sort of rhyme or reason as to why.
A few years ago there was a group of researchers that actually looked at this group from the standpoint of survival and found this group to have some of the longest average survival. Ironically, it is not this slow responding disease that is the worst. It is often the most responsive disease to therapy that can be some of the scariest. I know that seems counter intuitive and not what many parents would like to hear but it is often true. I have always attributed this to the fact that aggressive disease was always growing more quickly and because much of the chemotherapy targeted the fastest dividing cells these were always the first to die. The more of the aggressive cells you have, the quicker the tumor dies. Unfortunately, this quick response can also be the sign of a more aggressive and often lethal disease.
From that perspective, one might be thinking that refractory disease might not be such a bad thing. Make no mistake, you still don't want to have disease left after therapy. You want it gone. However, somewhere between lightning responses and non responses is probably the best. Keep in mind though, refractory is not all bad (no matter what you hear), and I do know of many who have gone on to eventually achieve remission and go on to leave happy lives without therapy and doctors visits.
Don't give up hope. Refractory patients often get the benefit of time which many others do not.
Like purpose it just takes more effort and time.