Wisconsin Immunotherapy Grant Summary

CKc Co-Founder Melissa Wiggins with Dr. Mario Otto, M.D., Ph.D., 

CKc Co-Founder Melissa Wiggins with Dr. Mario Otto, M.D., Ph.D., 

Summary of Madison, Wisconsin Trip: $75,000 Immunotherapy Grant

"We have a a two-year-old with relapsed Rhabdomyosarcoma - the hope is they will be placed on this trial as soon as possible."

Our response:

"Let's make it possible."

CKc left making it possible. We left confirming we would give an initial grant of $75,000.

First... THANK YOU!!!!! This is only possible because of YOU!!! All of you... donors, sponsors, volunteers, our Board of Directors and Scientific Advisor. CKc is a grass roots foundation and this is happening because of you.

Why Your Help Is So Important

1. Pediatric cancer is the number one killer by disease

2. Incidence rate is increasing, not decreasing

3. National Cancer Institute (government funding) is decreasing and is only 3.8 percent of the total budget. 3.8 percent divided by 12 primary pediatric cancers and 100 subtypes.

The Trial

It's a complicated trial but here is my understanding of it. I'm not a scientist but I have learned so much over the last three years in the pediatric cancer world so here is my attempt at a layman's version of this trial:

CKc believes in immunotherapy. This trial is an immunotherapy trial but it also uses an extremely complex, novel approach by removing and modifying cells as part of a stem cell transplant with cells from a mom, dad or adult sibling and then doing the immunotherapy.

This trial is designed for children who have relapsed or have refractory (non-responsive) HIGH RISK solid tumors, specifically rhabdomysarcoma, Ewing sarcoma and neuroblastoma, as well as some leukemias and lymphomas. The approach is innovative. One child has been treated so far and three children are waiting to start the trial.

Immunotherapies work best when there is minimal disease in the body. Let's take Cannon, for example. He was not placed on immunotherapy with ch.14.18 and IL2 until he had minimal disease. In other words, his up front treatment was designed to get as much cancer to go away as possible. Cannon endured 6 rounds of high-dose, adult chemotherapy, 108 hours of straight high-dose chemotherapy followed by infusion of his own stem cells, 12 rounds of proton radiation, a 13 hour surgery to resect (remove) his tumors, and then and only then was he able to do the immunotherapy.

This trial uses a novel, groundbreaking immunotherapy approach by doing a stem cell transplant for kids that have failed prior, harsh therapy, and where the cancer still has come back or doesn’t go away. The important immune cells actually come from mom, dad or an adult sibling and are given to the patient together with the stem cells. They choose a donor whose immune cells (particularly so-called natural killer cells and gamma-delta T cells) are predicted to detect and remove the patient’s cancer cells best. The way it was described to us is, think of a bouncer in the night club and he has to check identification. If the cells can’t show they are healthy, normal cells, they are removed by the killer cell (the bouncer!) immediately. Therefore the doctors manipulate and change the composition of the cells from mom or dad before they are placed in the child's body. They manipulate them so that the cells that fight the disease and also infections, work better, and the cells that would attack the healthy cells of the patient are removed. Then, patients will also get a medication that continues to stimulate the new immune system to fight remaining cancer cells in the child’s body – so mom’s or dad’s own immune cells can help fight their child’s cancer!

This trial is a novel approach of a translational type trial – meaning a trial that is bench-to-bedside. These are the trials CKc believes in. To us, there is nothing else. What is the point of science staying in the lab? Let's help kids live – TODAY!

The trial uses a donor's stem cells by manipulating the cells and reducing complications of transplant. This in turn requires less chemotherapy than traditional transplants and leads to a faster immune recovery. You can imagine that this is MUSIC to my little Scottish ears. This all means less side effects and quicker recovery, and with active immune cells transplanted, immediate anti-cancer immunotherapy to fight remaining cancer cells. So again, MUSIC to my ears.

The immunotherapy drug they will use after the transplant stimulates Natural Killer (NK) and gammadelta (γδ) T cells – both of these are known to be good anti-tumor responders.

Standard transplants are paid for by insurance. Where CKc comes in is by paying for the trial immunotherapy drug and monitoring the immune cells and recovery after transplant. In other words, we are dedicated and remain dedicated to filling the gap and ensuring kids are treated as soon as possible and not waiting for insurance fights to be sorted.

The Team Behind The Trial

Dr. Mario Otto is the principal investigator on the trial. He is supported by an incredible team including the inspiring legend, Dr. Paul Sondel. Dr. Sondel is Dr. Otto’s mentor and has supported and researched immunotherapy since 1969. Dr. Otto has won many, many awards including Young Investigator. Dr. Otto completed his MD and PhD training at the University of Tübingen, Germany, and his Fellowship training at St. Jude's. The entire team spent many hours presenting to us, showing us their research, their labs and explaining their work. It was just incredible to be surrounded by researchers who are trying to save our kids from the number one killer by disease – pediatric cancer. I was truly humbled and honored. A few times I held back tears just thinking of the kids that their research could help. The kids fighting cancers are my heroes but so are the nurses, the oncologists and these researchers who have dedicated decades to this extremely hard field to work and stay in.

About The Institution

"Small but mighty" is how I would describe this institution. It may only have 87 children’s beds but it is one, yes one, of only 15 institutions that have a MIBG full lead room for the very specialized MIBG therapy. It is also one of only 7 member institutions of the Stand-Up-To-Cancer (SU2C) St. Baldrick’s Pediatric Cancer Dream Team, and a National Cancer Institute (NCI) designated comprehensive cancer center, one of only 45 in the country.

Quote From Dr. Mario Otto

“We are very grateful and humbled for the support of our clinical study by CKc. Cancer in children that doesn’t respond to conventional therapies or has come back is often hard to cure – many children therefore will die from their cancer. So, we have to find more innovative ways to cure childhood cancer. What most people don’t know is that clinical trials that try to test new drugs or treatments are extremely costly and seldom covered by health insurance. Most clinical cancer trials are designed only for adults and are sponsored by the pharmaceutical industry, which rarely shows interest to support studies in kids as they see no financial incentive to do so. That often means that even the most cutting edge promising treatments we have developed in the lab will never be tested in the clinic to find out if they will benefit kids with cancer.

And this is why CKc is such an amazing foundation. They understand that the lack of sufficient funding for pediatric clinical cancer trials slows us so much down in finding new and better, less toxic treatments for kids with cancer – and they have decided to tackle this terrible situation. They are passionate advocates for the children affected by cancer, their families, and pediatric cancer researchers alike. They can relay the importance of clinical trials in kids with cancer to their generous donors and are passionate lobbyists for our cause with policy makers in DC and elsewhere. CKc, supporters and donors: We are thankful for all what you do – it will allow us and other cancer doctors and researchers to find new ways to treat and cure kids with cancer.”

How We Decide Where To Fund

Please know that hundreds of hours go towards finding trials: deciding if they are right for CKc, have applications reviewed, Board approval, Scientific Advisor calls and emails, honoring the child whom the trial is named after, etc. There are so many factors that go into this process. I say all of this because I want donors, community supporters, volunteers, sponsors and everyone involved with CKc to know that we work really hard to make sure the money that all of you work hard for is spent in a way we believe will best serve children fighting so hard against cancer.

How Much Funded To-Date

By the end of July, we will have funded almost $300,000 in the 18 months since we received our 501(c)(3) nonprofit status in January 2015.

If you have any questions or need more explanation, please feel to email me direct. I am happy to help in any way I can: melissa.wiggins@cannonballkidscancer.org