TARGETing Neuroblastoma: ARRA Funds Supporting Childhood Cancer Research

[ Music ] [ Background Music ] [Hayley] Neuroblastoma. I don't know what it is. [ Music ] [Dr. Maris] We have one child who has a very benign variety, who can be cured with no therapy at all, not even surgery, and her disease just goes away. But there's another child, another-- probably 50 percent of patients where the disease is very, very aggressive. [Hayley] There was a sick bug in my belly. I always thought it was that. They were trying to treat me as a 3-year-old. By telling me it was a sick bug. But I could be treated like a 5-year-old. [Karen Kudro] Cancer is like, the furthest thing from your mind. You never-- even think that that's what it's going to be. I mean, she was checked in July-- no, August-- for school to start kindergarten, and that-- there is nothing, no mass. They could feel it in September. In August, there was nothing. Not a cancer that you can tell. It's got very mild symptoms, and it's not genetic. It's-- they don't know what causes it. So there's-- you can't really prevent it. [Dr. Maris] So it's about a year and a half of very intensive therapy, that cures the disease in maybe half of patients. [Hayley] One girl, she asked me twice why I have short hair, and I told her why, 'cause I'm having chemo. [Karen Kudro] Six months of chemotherapy, a tumor resection, which is her surgery, a bone marrow transplant, radiation, and then the second part of the study, which we're on now, is the antibody treatment. [Dr. Maris] Just about any side effect that you can get from cancer therapy, children with-- who survived, neuroblastoma, are saddled with. And these are growing children. These are young children. So it can stunt their growth, stunt their physical and/or cognitive development, it can certainly stunt their emotional development, it can cause deafness, blindness, significant heart or lung problems, most shockingly, it can actually cause cancer. So, having a second cancer arise from the treatment is an all too common problem. So all of these together just simply means that we have a standard approach that works about half the time and is pretty darn toxic. And so, we need to come up with better treatments. [ Background Music ] [Dr. Maris] The target project is designed to be a comprehensive survey of all of the genetic abnormalities in neuroblastoma cells in order to identify which of those we can design a drug against. Traditional chemotherapy gets cancer cells, but also gets normal cells. And that's why you have side effects. One of those types of targeted therapies that we've developed over the years before the initiation of the target project was an antibody against a molecule that's on the surface of neuroblastoma cells, immunotherapy. And that is targeting specifically the neuroblastoma cells compared to the child's own nerve cells, and that is the first time in neuroblastoma, we've been able to really exploit a target, and show that it improves cure rate. [Karen Kudro] I just don't want to have to worry about it. It's something that we're gonna have to worry about probably for the rest of our lives, but with this treatment, it should ease it a little bit, knowing that she shouldn't relapse. [Dr. Maris] The ARRA Funds came in at a time when we had done the initial sort of broad survey at a low resolution level, and we were able to sequence about 1 percent of the genome if that, and the ARRA funds came at a time that allowed us to now move with the technology and stay ahead of the technology and really do a very, very comprehensive sequencing of a lot of bad neuroblastoma tumor samples. [Karen Kudro] My dream outcome would be that they could use it right off the bat, get rid of the cancer, and that it would never return. [Hayley] Next summer, I'll be better. [ Background Music ] [Dr. Maris] The hope of the target project is that in our lifetime, and hopefully, not that distant future, we'll be able to see a new patient with neuroblastoma, we'll be able to take a piece of their tumor tissue at diagnosis, analyze it completely genetically and say that this child requires X, Y, and Z type of therapy, compared to this child whose genetic analysis is different, and will require A, B, and C type of therapy. [ Music ] [ Silence ]