CKc Awards $680,000 in Pediatric cancer Grants
For Immediate Release, June 6, 2019
Contact: Karen Revels, Executive Director
CANNONBALL KIDS’ CANCER FOUNDATION AWARDS $680,000 IN PEDIATRIC CANCER GRANTS
ORLANDO, Fla. – Cannonball Kids’ cancer Foundation (CKc) today announced the awarding of $680,000 for six research grants to create 133 options for children battling cancer through clinical trials, programs, and laboratory research.
To date, CKc has awarded $1.9 million in pediatric cancer research and program support since the organization’s founding in 2015. Including this latest investment, CKc has created 385 treatment options for pediatric cancer patients in 26 U.S. states, plus Washington, DC, Canada, Scotland and Switzerland.
“These funds are critical to providing support to the underfunded world of pediatric cancer,” stated Michael Wiggins, Chair of CKc’s Board of Directors. “Thanks to our generous donors, and under the guidance of our Scientific Advisory Board, these funds will allow researchers the opportunity to conduct research that will someday secure less toxic, less painful therapies for children with cancer, and drugs and therapies that are developed specifically for children’s forms of cancer.”
CKc’s Scientific Advisory Board (SAB), comprised of leading investigators specifically in the field of pediatric cancer, guides the organization’s team and Executive Board in validating the merits of the science behind the grant proposals it receives. Collectively, the SAB members spend a total of nearly 50 hours of volunteer time each grant cycle reviewing and ranking grant applications to determine which grants to select and ensure CKc is funding the best possible research. This is the first of two invitation-only grant cycles in 2019, which are based on in-person meetings with researchers and hospital visits. Invitations for the second cycle will begin this summer.
Despite being the number one disease killer of children in the United States, pediatric cancers receive only 4% of federal dollars dedicated to cancer research. The task of financing research in pediatric cancer, and thus improving decades-old treatments, has fallen to individual donors through foundations like CKc. Pediatric cancer treatments have gone largely without progressive developments for over 20 years; and, for some forms of childhood cancers, the survival rate is still 0%. CKc has taken a “disruptor” approach to addressing these issues in pediatric cancer by funding primarily innovative, first-of-its-kind research and educating the public on the realities of pediatric cancer, both the rate of survivorship for various cancers and the impact of pediatric cancer treatments on survivors.
2019 Funded Grants
Michael Ortiz, Memorial Sloan Kettering Cancer Center – ($200,000)
A multi-center phase I study of Codrituzumab (GC33, RG7686, RO5137382) in pediatric patients with relapsed or refractory GPC3 expressing solid tumors
Glypican 3 (GPC3) is a protein that is important in fetal development but several pediatric cancers are able to inappropriately re-express this protein to help them grow faster. GPC3 is expressed in hepatoblastomas, yolk sac tumors and choriocarcinomas (which are specific types of germ cell tumors), most Wilms tumors, most rhabdoid tumors, and a minority of rhabdomyosarcomas. Codrituzumab is an antibody that binds to GPC3 and helps the body’s immune system to recognize these GPC3-expressing cancer cells. This drug was studied in a series of clinical trials in adults with liver cancer and was found to be safe. We are interested in determining whether codrituzumab will be safe when given to children with GPC3-expressing tumors that are not able to be treated with standard of care therapies. We will also investigate the ideal dose of codritizumab for children as well as specific blood and tumor markers to help determine which patients will derive benefit from codrituzumab. Once we determine whether and at which dose that codrituzumab is safe, we plan to open up a special arm for children with hepatoblastoma as we expect that targeting GPC3 is going to be particularly effective in these tumors. Given the important role of GPC3 in several pediatric solid tumors combined with the safety in adults, there is a compelling rationale to evaluate codrituzumab in this phase I clinical trial as it could lead to transformative changes in the treatment of several childhood cancers.
This first-in-children immunotherapy research study has the potential to impact up to 28 patients with recurrent or refractory disease from renal and hepatic tumors and will be accessible in New York, Georgia, and Ohio.
Dr. Ortiz was also awarded a Young Investigator Grant ($100,000).
Gregory Friedman, University of Alabama at Birmingham – ($200,000)
Phase 1 Trial of Engineered HSV G207 in Children with Malignant Cerebellar Brain Tumors
Current therapies for childhood brain tumors like surgery, chemotherapy and radiation are very damaging to the developing brain and can result in significant long-term brain injury. Furthermore, approximately 30-40% of children with brain cancer do not survive. Novel therapies are desperately needed to improve outcomes and lessen toxicities. Therapy with a cold-sore virus (HSV) offers an innovative, targeted, less-toxic approach for children with brain tumors. HSV has been successfully engineered to introduce mutations in the virus that prevent infection in normal cells while maintaining the virus’ ability to kill cancer cells and stimulate the child’s immune system to attack to the tumor. We are currently conducting a first-in-children Phase 1 trial of HSV G207 in brain tumors located in the upper part of the brain (cerebrum). We have safely treated 10 children and have seen evidence of significant responses. Our preclinical data indicates that tumor types that arise in the lower part of the brain (cerebellum) are more sensitive to killing by G207 than tumors in the cerebrum. Furthermore, few effective options exist for children with progressive cerebellum tumors. Therefore, we propose to conduct a first-in-human Phase 1 trial of HSV G207 in progressive malignant pediatric cerebellar tumors to determine the safety and tolerability of the therapy. Our secondary goals are to determine the effectiveness of the therapy and the immune response to the therapy. We will also explore specific features of the tumor and in the patient’s blood that may predict a treatment response to oHSV.
The novel drug administration of first-in-children viral immunotherapy in this research study has the potential to impact up to 15 patients with recurrent or refractory disease from brain tumors, which are the deadliest childhood cancers, and the study will be accessible in Alabama.
Sabine Mueller, Children’s Hospital Zurich – ($65,000)
Brain tumors are now contributing to the most cancer related deaths in children. For many brain tumor types, decades of research and clinical trials have not achieved any improvement in the clinical outcome. Genome profiling of these cancers revealed that these tumors have different molecular subgroups. This means that while the tumors occur in the same age range, or location within the brain, the biology of the tumor may be individually different. Consequently, each patient (or groups of patients) will require personalized therapy based on their tumor molecular subtype (in comparison to the more generic tumor type). Because childhood brain tumors are considered rare, international collaborations are needed in order to study a larger patient population in the shortest amount of time. This will allow us to match specific subtypes of brain tumors to the adequate therapy, and change the current “One size fits all” approach. We founded the Pacific Pediatric Neuro-Oncology Consortium (PNOC) in 2013 with the goal of designing clinical trials based on each patients’ biology. To address the need for the international collaboration, we are expanding PNOC internationally (PNOC Global) to develop global collaborations and building infrastructures to conduct multi-center trials so that we can learn how to target the disease faster by integrating a larger patient population. Our first European based PNOC international site will be The University Children’s Hospital in Zurich, Switzerland, which is well positioned to contribute to the larger mission of PNOC as one the largest Children’s hospitals in Europe. Experiences gained by implementing PNOC trials in Zurich, will set the framework to continue onboarding of other European based clinical sites.
The international collaboration of this program will bring novel therapy options from the United States to Europe, and has the potential to impact up to 40 patients with recurrent or refractory disease from brain tumors, which are the deadliest childhood cancers.
Adam Kelly, Glasgow Children’s Hospital Charity – ($65,000)
CKc Foundation and Glasgow Children’s Hospital Charity have worked in partnership for the last two years. Thanks to the commitment and support of CKc Foundation we were able to expand our Schiehallion Clinical Trials Centre at the Royal Hospital for Children, Glasgow, by employing a Research Nurse focusing specifically on increasing the early phase clinical trials portfolio available to children and young people in Scotland who are diagnosed and treated for cancer and blood disorders.
As an extension to this we are now looking to increase the accessibility of our clinical trials by employing an Outreach Research nurse who will visit families in their own homes to support and help them understand clinical trials and treatment. The nurse will cover trial protocols with families in their own environment and in a way they understand. This will give parents control and an added level of support when clinical trials are an option for their child. The nurse will also discuss the treatment that will take place and this will result in more children accessing trials, more data being collated and an increased chance of survival or better quality of life.
The accessibility focus of this program grant initially created treatment options for up to 50 children with leukemia, lymphoma, neuroblastoma, and retinoblastoma by bringing novel clinical research trials from the United States to Great Britain.The continued implementation of this program will extend and enhance the institution’s ability to bring access to less toxic, life-saving treatments for those children.
Elizabeth Beierle, University of Alabama at Birmingham – ($50,000)
Hepatoblastoma (HB) is the most common primary liver tumor in children and its incidence is rising. Although survival rates for pediatric cancers have improved dramatically in the past 30 years, HB remains one of the most difficult childhood tumors to treat. Many children have disease that is resistant to standard treatments that will require novel, innovative, and targeted combinatorial therapies to effectively treat or manage their disease. We plan to demonstrate a driver role for a protein known as PIM3 kinase as a mechanism for HB chemotherapy resistance and recurrence. PIM3 is known to affect cancer growth in adult liver cancer and we have found that PIM3 is expressed in pediatric HB tumor specimens. Further, inhibition of PIM3 resulted in decreased HB tumor cell survival and tumor growth in animals. We believe that there is a small population of cells, called tumor initiating cells, which contribute to chemotherapy resistance and cancer relapse. We have made the novel observation that PIM3 affects this distinct population of HB cells and may render them more susceptible to chemotherapy. The aims of the proposed studies are to show that PIM3 does play a role in the maintenance of this special cell population and that targeting PIM3 results in decreased resistance to standard chemotherapies. We will use cultured HB cells and animals’ models to study these aims. The expected findings will be particularly exciting since there are inhibitors available and currently in clinical trials for PIM3 that could be rapidly advanced for use in pediatrics.
Few options exist for recurrent or resistant hepatoblastomas, and basic laboratory research must first be established and advanced to create clinical trials for children. This research funding enables Elizabeth Beirele’s first-of-its-kind postulation to be further developed in her lab in Alabama, bringing it closer to the important goal of treating children in the clinic.
In the photos (L to R): Dr. Sabine Mueller, Michael Wiggins, Melissa Wiggins, Dr. Julia Glade Bender, Dr. Michael Ortiz, Tony King, Kelly King, Cannon Wiggins (front).
About Cannonball Kids’ cancer Foundation:
Cannonball Kids’ cancer (CKc) Foundation’s mission is to fund innovative and accessible research for children fighting cancer to provide better treatments and quality of life, and to educate for change. CKc was founded in June 2014 by Michael and Melissa Wiggins, parents of Cannon Wiggins, who was diagnosed with Stage IV high-risk neuroblastoma when he was just 20 months old. During Cannon’s treatment, Michael and Melissa learned very little time, effort and funding is devoted to finding cures for children’s cancer compared to adult cancers, and as a result, children are unnecessarily and unjustly lost. CKc aims to stop the tragic reality of children suffering and dying because of the lack of research in the world of children’s cancer treatments.
[EDITOR’S NOTE: The “c” in cancer in the name Cannonball Kids’ cancer Foundation is intentionally lowercase to give the word “cancer” an inferior status.]