Summary of Michigan Trip - July 24-26, 2016: Relapsed Neuroblastoma Trial
15 Kids and 8 New Hospital Sites
We left approving an initial grant of $75,000. This initial funding will help at least 15 kids get on this trial and will assist in opening 8 new sites, meaning that 8 different hospitals will have this trial locally for their kids. This includes Arnold Palmer Hospital for Children. Everyone knows how much this excites me!! Pediatric Oncologist Dr. Don Eslin is actually on the Executive committee and the Scientific committee for the NMTRC (Neuroblastoma and Medulloblastoma Translational Research Consortium), the consortium running this trial. Dr. Eslin took care of Cannon many times and we respect him and his work immensely. Now, back to the 15 kids… 15!!! I love that number but I want to help more! I'm greedy, I know!!! The only time I will be greedy and not apologize for it is when it literally saves kids’ lives and that is what YOU and Team CKc are doing!
Layman's Summary Of The Trial
High risk Neuroblastoma remains a challenge in the pediatric oncology world and is responsible for 15 percent of ALL pediatric cancer deaths. That's more than any other form of pediatric cancer. For the 50 percent of kids where relapse occurs, oncologists used to state that this was incurable. The 5-year survival rate of these relapsed kids is 10 percent. What does that mean? It means 40 percent of the kids will have been buried by their parents within 5 years!! That is NOT okay with me! It is NOT okay with Team CKc! Dr. Giselle Sholler, the lead investigator and who started the trial says, “Thanks in part to DFMO, I do not believe that it is incurable. I’ve seen it cure kids and I believe we can cure more.”
So, here we are funding this trial to help those numbers change! It's all about ACTION and this is CKc in action!
This trial is the first of its kind - no trial has ever investigated DFMO with Bortezomib in children. The funding from CKc is assisting with Phase 2 of the trial. Meaning, Phase 1 is almost complete with 13 of 16 kids already treated. A Phase 1 trial means this is the first time the trial will be tested in humans. Every trial begins at Phase 1, this is a requirement by the FDA. The Phase 1 purpose is to:
1. Research the safety of the dose levels.
2. Find the highest/optimal dose level that can be used in kids safely.
3. Check toxicity levels. Toxicity is complex, but in simple terms, it can mean pain, side effects, long term effects, organ function, etc.
4. Look at survival in terms of long term and overall. In other words, efficacy.
This trial is a study of DFMO in combination with Bortezomib. It is designed to help children who have relapsed and refractory (non-responsive) Neuroblastoma. Neither one of these drugs have been FDA approved for children, but considering there have been three drugs in the last twenty years approved for kids – well, it's not surprising. Bortezomib has been approved for adults with various cancers, specifically myeloma and leukemia. DFMO has been shown to be effective in inhibiting tumor growth in adult colon cancer. DFMO has been used in five pediatric trials to-date and has shown significant increase in survival in Neuroblastoma.
Goal Of The Trial
It’s simple: save kids who have little or NO options! Every parent who hears the word “relapse” knows the chances of survival are tough and that picking the right trial is incredibly difficult!! The hope… the goal… is to save these kids who face relapse and help their parents have hope! Relapse doesn't need to mean DEATH anymore!!! Let's change the statistics!! The GOAL is that this trial which has been developed is effective in treating relapsed and refractory Neuroblastoma with little pain and side effects.
Why are we using a drug for adult colon cancer on kids with a hard tumor growth cancer like Neuroblastoma? This is complicated and I've read for hours to comprehend it, but the gist of it is this: aggressive forms of Neuroblastoma respond poorly to the regular chemotherapy approaches. Novel approaches are needed and required. Elevated levels of Polyamine are often found in Neuroblastoma tumors and as a result reducing Polyamine in cancer cells reduces tumor cell proliferation (rapid increase).
Why This Trial
When Cannon was diagnosed with stage 4 heavily metastasized cancer (meaning spread to other parts of the body), we were told he had a 50/50 chance of survival and that sadly the rate of relapse is around 50 percent. I asked what that meant and they told me "We are not discussing that right now - let's focus on beating it now, the first time." Thankfully that's been our journey but sadly it's not for many, many families we have journeyed with. That's NOT acceptable to me! It's NOT acceptable to our Board or our volunteers and certainly NOT okay to our donors who also contribute to change it.
So, after an application for funding was approved, hours of oncologist meetings, touring labs and Board approval - we are funding the original grant application for $75,000.
Dr. Giselle Sholler – Lead Investigator
Dr. Sholler is TRULY one of the most humble, incredibly gifted researchers I have come across in these three years. She and her team are changing the stats for stage 4 Neuroblastoma and CKc is privileged to play a small part in this work.
Dr. Sholler states that “Everyone at the NMTRC is very thankful to Cannonball Kids’ cancer for this incredible donation. Together we will bring this new therapy to children fighting Neuroblastoma, giving hope to families and their physicians. This would not be possible without the generosity of the team supporting CKc.”
The trial is in honor of our sweet Ava Crowley. Ava has been battling Neuroblastoma since she was 2 years old. She is currently 6 and has been dealing with relapsed Neuroblastoma on her aorta for the last 18 months. She is kicking butt. This trial is to honor her but also all the other fighters of this dreaded pediatric cancer. This past week, for the first time since joining this crazy pediatric cancer world, Cannon and Ava got to swim together and do “cannonballs” in our pool. It was an emotional but beautiful, beautiful time for her mother and father and myself. I will never forget that as long as I live!!
Huge Thank You
I would like to take give a huge shout out to Beat NB. They have allowed Dr. Sholler and NMTRC to work hard on the science by vastly and consistently funding her work! Without them, these projects wouldn't be as far along and as a mother of a child on the original DFMO trial, I thank you for your hard work and resilience!!! Thank you Patrick Lacey and Kyle Mathews whose foundation, Because of Ezra, combined recently with Beat NB. I’m super impressed by all they do!
If after reading this summary you want to help CKc do more for this trial or others, please email me. I would love to hear from you: Melissa.firstname.lastname@example.org