Our Biggest Swing At Pediatric cancer Yet And We're Just Getting Started!

Please share the exciting news below... Awareness = Funding = Research = Cure!


I know some, but not all, people want to know about the science behind the trial we just began funding, so for those who are interested, here goes:

I am not science-educated, but I do understand some of what we are trying to achieve. Below is my understanding of the trial, the science and details on the doctor, the hospital and how we made this decision. If you have questions, please write them in the comment bar and I will respond as best as I can. 

Let me start by wowing you. The trial we are funding is the first of its kind in the world. Fact. 

Want to learn more? Keep reading, it gets more interesting, I promise. 

A Little Background

Immunotherapy has been a way of treating neuroblastoma under the Children’s Oncology Group (COG) for some seven years. Only in the last few months, has the immunotherapy CH14-18 been implemented into the protocol. Memorial Sloan Kettering Cancer Center has been working with 3F8, a version of antibody immunotherapy, for over 20 years, but COG only followed suit in the last 7 years. 

For brain cancer, however, there are no approved immunotherapy drugs. The trial we are funding is specifically a brain cancer immunotherapy trial in children. This is a very exciting time in pediatric cancer and scientists are really motivated to move towards this way of treating kids. 


The Science Behind The Trial

The immune system is naturally able to identify and destroy cancer cells. All of us have cancer cells in our body but most of the time we are able to kill those cells naturally. For decades, doctors and scientists have believed that our bodies couldn’t see the tumors. According to this idea, Cannon’s immune system did not see the tumors in his body and that is why they did not try and kill them and they grew until treatment. 

A new wave of scientists is disagreeing with this and saying, "What if the body is seeing the cancer cells, yet the cancer is outsmarting the immune system so that it cannot destroy the tumor?" In order for cancer to grow, it must evade or disable the immune system. The body has checkpoint pathways, one of which is called IDO. Scientists believe the tumors (cancer cells) use the IDO enzyme in the body to escape the immune system’s ability to naturally kill the cancer. Dr. Johnson and the team at Georgia Regents University Cancer Center have developed a drug called indoximod which blocks the IDO, allowing the immune system to do its job – fight cancer cells. This drug is now being developed by NewLink Genetics Corporation in multiple clinical trials for various types of cancer. 

The power of the immune system is truly quite fascinating – news to me. It has a memory, so if we educate the immune system to target the cancer, it can develop a memory to do so in order to potentially stave off cancer forever – perhaps preventing a future relapse. We all know I dislike the "R word" – but the truth is, it is the main reason for death in kids' cancer – relapse of the same or a different cancer.  

Indoximod is the IDO inhibitor drug that is the furthest along in development for clinical use in adults. In the pediatric world, it is the ONLY IDO inhibitor. For the last five years, Dr. Johnson has been working on the pre-clinical data and is now approved to move into the Phase One Clinical Trial that we are funding. The trial is planned to open in September of this year. 


The Really Exciting News

Dr. Johnson got an individualized approval for the drug for a seven year old boy with relapsed brain cancer. There were no other options for him. He went to see Dr. Johnson at GRU Cancer Center and, on March 25th of this year, the first ever IDO-blocking drug was given to a child. Right there, at that moment when we saw the boy's picture (with his mum's approval), right there – THAT IS WHAT THIS IS ALL ABOUT!!! This could have been Oscar – maybe this could have saved him. But maybe, just maybe, this child with no options will beat cancer and we are believing he will. To think that our foundation could help save children...I get teary-eyed just thinking about the reality of that. This is why we will never ever STOP!!! Kids like Oscar deserved to live, but there were no options. We must develop options that work. I cannot imagine a better one than trying to retrain our OWN bodies, our OWN immune systems to achieve that. 


How Is This Different From A Vaccine?

Many hospitals, such as Memorial Sloan Kettering are beginning an era of vaccines. I am thankful that many different approaches exist for curing cancer. It's like rolling a die, none of us know which one will be the winning number. Dr. Johnson's approach cannot be called a true vaccine, because a vaccine in its true sense is given before we get something – like the measles vaccine is given to a child before they actually have the measles. Dr. Johnson’s trial is trying to target the patient's immune system and change it. Now if we compare that to the treatment Cannon had, Cannon’s treatment was immunotherapy, but an antibody was inserted into Cannon and it was designed to actually attach to the cancer cells. In simple terms, to eat it. This trial is different in that it is not attacking the cancer, it is attempting to change the immune system so that it can do its job. 


How Do We Do That? 

Traditionally, chemotherapy is not used with immunotherapy, but Dr. Johnson and his team found if they combined indoximod with chemo they could get the effect they needed. Preclinical research also showed that this effect may be even more beneficial when also combined with radiation therapy. High dose chemo is probably not required as only some of the cancer cells need to die off to see beneficial immune effects after indoximod. Similarly, even if the child has used the same chemotherapy before and it failed to kill all the cancer, combining it with indoximod may still have the desired effect. The indoximod by itself likely does very little – truly a real case of hit and miss, but with the chemo or radiation/chemo it can amplify the inflammation. Think of a bug bite and how it rises and gets red before we put antiseptic on it. That is our immune system reacting to it. In order for the indoximod to be most effective we need some inflammation, and chemo can serve this very purpose. IDO appears to be a key pathway that the immune system uses to decide whether to tolerate or attack tumor cells. 


Adult Trial

In order for a trial to open for children there must be a trial for adults. In 2014, GRU, Dr. Johnson and his colleagues began a trial for adult brain cancer patients with dismal prognosis. The biggest concern toxicity-wise from the trial was brain pressure. There is no room in our heads for extra fluid, and that can cause big problems. That said, there was no toxicity found in the adult phase one trial, meaning there was nothing serious enough to delay, stop or change the trial itself. They are now enrolling at a phase two level. Phase one allowed them to look at dose level and phase two will use the highest dose-level of indoximod that was studied in the phase one trial. 


What Are The Concerns In A Trial For Children? 

The primary concern, aside of brain pressure, is the child would develop an auto-immune disorder – these can sometimes be fatal. Having experienced this with my husband, I know firsthand such disorders are no joke. The trial is a multimodal approach and fast becoming the standard in adult care. Childhood cancer has fallen far behind adult research and our foundation aims to CLOSE that gap. 

The Doctor

You know I always say that I don’t need my son’s doctors to be funny or personable, I need them to be smart and innovative. For me, Cannon’s care was my job; their job was the science and the approach. But on occasion, you meet doctors like Dr. Johnson – smart and brilliant, but also personable and able to explain things in layman's terms to someone like me who has little or no medical background. I am a lawyer by trade, what do I know about protein enzyme blockers? I have never met a doctor who said to me, "I want to change the way pediatric oncology patients around the world are treated." Maybe he will, maybe he won’t, but if he doesn’t try, he most certainly never will. I wanted to scream, "Where have you been?" Yes, Yes, Yes…let's change the way cancer in kids is treated. In the words of our CEO, Ashley VanDerMark – "He is our guy." And the simple truth is, he is. Some people may have told him he was crazy with this idea but that’s what people told the now 88 year old Dr. Freireich in 1955 when he started infusing pediatric leukemia patients with platelets to stop them bleeding out and later began the era of high dose chemotherapy, changing the six weeks diagnosis-to-death period to a 95 percent survival rate. In fact, he was told if he didn’t stop infusing platelets that he would be fired and for the first time the patients stopped bleeding out. Dr. Johnson is going to make a footprint in pediatric immunotherapy and we at CKc are going to help him help the kids around the world.


The Hospital 

Georgia Regents University Cancer Center is currently in the process of becoming a National Cancer Institute (NCI)-designated cancer center. There is currently no increase in NCI-designated cancer centers; in order for a new hospital to be designated, an existing one must lose its designation. You can imagine this is no small feat. The hospital itself was extremely impressive – I lost count of the number of times they told me, "This machine is one of six in the world." Their deal is immunotherapy and that was clear. 


How Did We Get Here?

Many of you reading this know how we got here, many do not – so bear with me. My husband/co-founder and I literally feel like we traveled the globe, and not on foundation dollars, but our own. I say that to ensure people know we try our absolute best to keep costs as low as possible so our dollars can stretch as far as possible towards research. We visited St. Jude's, met with their doctors and toured their institution. We met with CEO’s of top research foundations and we met with multiple hospitals. We wanted to ensure wherever our first grant went would be the best use of resources for our kids. Money that YOU helped raise – that is of primary importance to us. After a year of doing our research, under the guidance of our friends Jay and Liz Scott, founders of Alex's Lemonade Stand (ALS), who lost their beloved daughter to the same cancer Cannon, our son, has fought – stage 4 neuroblastoma, we were directed towards Dr. Johnson. Our guidelines were not complex; they didn’t require 20 scientific board members to develop them. Our guidelines were the research had to be in the brain cancer pediatric field, immunology oncology and cutting edge. Sounds simple right? Well, thanks to ALS, we ended up with Dr. Johnson and after vetting him, his institution and his trial we knew we had our guy and our project. Our team always says there is no room for egos in childhood cancer. Teams like ALS don’t need to help us but, the point is, they did and I know they will again because, like us, they just want the madness, that is childhood cancer, to stop. If you want to donate to research please consider Alex's Lemonade Stand, one of the leaders in the field. 


Lots Of Moving Parts

There are lots of moving parts that will make all of this work, from funding from us, funding from ALS to the pharmaceutical company that has poured millions into the drug development despite the fact they run a business and this pediatric study will not make them any money. I am in the process of writing a letter to that company to thank them for taking a chance on our kids. Not many companies do that and I am eternally grateful. Thank you NewLink Genetics for taking a chance on our babies. 

Some ask why don’t we fund locally? There is nothing saying we won’t in the future, if the right research needs our help. With that said, it is important to note that whether a trial is in Georgia or Germany – it is available worldwide and therefore available to all children. As a foundation, we must seek out the best researchers and fund them whether in Florida or across the pond. 


The Future

The truth remains – there are 120 adults diagnosed with cancer for every child diagnosed. How many of those 120 adults have children? How many of them would give a few of the hundreds of millions of dollars of research funding to their children?

The facts are simple. Kids' cancer kills more than ANY other disease. In fact, MORE of our kids are dying of cancer than many, many diseases COMBINED. THIS MUST STOP!

CKc has its Army, Navy, Air Force and Marines in place and we will keep fighting the war against cancer all our days on this earth. 

Our Army - By creating awareness that childhood cancer is not all balloons and smiles. By showing the truth about what cancer in kids really looks like.

Our Navy - Funding trials that would never leave the lab without private not-for-profit foundations like ours.

Our Air Force - Educating regarding the dire facts about kids cancer - how many are dying and how can we change it.

Our Marines - Lobbying and hitting them in Washington, D.C. Keeping pediatric cancer at the forefront of their minds when funds need to be allocated. 

Our approach is dynamic, but we believe this is how it needs to be done. 

This, my friends… is only the START.

Year one – one grant! Year two - three grants, and so on and so forth. We are not going anywhere.



I am 

I continue to be

Thankful For The Fight

Purchase our Book - "Thankful For The Fight" - all proceeds to research 


Make a donation to our pediatric cancer research projects 


Host a card club event


Written by Mumma-Bear

Cannonball Kids' cancer


Thankful For The Fight

Pray Hard

Pray Big 

1 year and 3 months No Evidence of Disease

No Relapse Ever