2023 Childhood Cancer Research Grant Recipients
To date, we've granted over $21 million, funding over 130 research projects that have resulted in 25 treatments and clinical trials available to children today.
Our all-star Medical Advisory Board meets annually to review and evaluate grant requests using the same stringent criteria as the National Cancer Institute. With your support, we fund pediatric cancer research that has the strongest science behind it and the best chance of getting from a research lab to a child’s bedside in the shortest time frame possible.
The following 2023 grants are the result of support from thousands of passionate people from every state and 26 countries who believe in our mission of funding the most promising research for new, improved and less toxic therapies for pediatric cancer.
Seattle Children’s Hospital: $200,000 Grant
Project: Antigen Assimilating Multispecific T Cell Engagers (MTEs) for the Treatment of Diffuse Midline Glioma (DMG); Recipient: Dr. Jim Olson
Diffuse Midline Glioma (DMG) is an aggressive type of brain tumor that occurs in children. Current treatments are ineffective, and the disease is universally fatal. Recognizing the reasons for failure of previous therapeutic approaches, our research project aims to develop a new class of therapeutic that overcomes the barriers to effective treatment. Specifically, we will advance a new class of protein therapeutic that initially targets vulnerable brain tumor cells and converts nearby treatment resistant cells into treatment responsive cells. We hope the outcome of our efforts will establish a new therapy for treatment of this devastating disease and improve the lives of DMG patients and their families.
Dana-Farber Cancer Institute: $200,000 Grant
Project: Targeting Chromatin Complexes in NUP98-rearranged AML; Recipient: Scott Armstrong, MD, PhD*
Pediatric acute myelogenous leukemia (AML) remains a difficult-to-treat disease. Patients with an AML that possess specific genetic abnormalities, such as NUP98-rearrangement, typically do not respond to standard cancer treatments and have a poor prognosis. New therapies are desperately needed that can specifically target the processes driving these types of leukemia. The Armstrong Lab has discovered a new drug (Revumenib) that is showing tremendous clinical responses in children with AML, a particularly challenging subtype of leukemia that has a survival rate around 65%. Our goal is to determine which drugs should be combined with this new approach to further enhance responses.
“We are grateful that Cookies for Kids’ Cancer is supporting our work, focused on the development of new drugs for aggressive subtypes of childhood leukemia." - Dr. Scott Armstrong, Chairman, Department of Pediatric Oncology, Dana-Farber Cancer Institute
Texas Children’s Cancer Center/Baylor College of Medicine: $200,000 Grant
Brain tumors are the leading cause of cancer-related death in children and adolescents. “Ependymoma”, a type of cancer arising from the cells lining the ventricular system of the brain and spinal cord, affects about 200 children in the United States each year. Ependymoma can occur in both children and adults, but it is most common in children younger than 5 years of age. The standard treatment for newly diagnosed ependymoma is surgery followed by radiation therapy to the tumor area (for children older than 1 year). Many children benefit from this treatment, however, the chances of tumor coming back is very high and the traditional chemotherapy offers minimal or no benefit. Repeat surgery is often used to treat tumor recurrences as there is no curative treatment available. The long-term outcomes are poor for children with recurrent ependymoma due to neurological limitations and compromised quality of life, and many die from progressive cancer. For these children, alternative methods of treatment which attack the tumor differently than conventional therapies may be beneficial.
With the support from Cookies for Kids’ Cancer, we will study if an immune-based treatment, called “chimeric antigen receptor T cells” or “CAR T cells”, can be given safely to children and adolescents to treat recurrent ependymoma. In this approach, immune cells called T cells taken from the patient are genetically modified to target a tumor-associated protein HER2 (HER2-CAR T cells), which is present in the majority of ependymoma tumors. To make this investigational treatment available to children closer to their home, and because ependymoma is rare, we have teamed up with Pediatric Brain Tumor Consortium (PBTC) to conduct a clinical trial of HER2-CAR T cells (PBTC-059 trial) through a network of Children’s Hospitals across North America. All HER2-CAR T cells will be manufactured at Texas Children’s Hospital and shipped to the treating institution. In this trial, we will also assess if CAR T cells can be safely combined with surgery to prevent the tumor from growing or coming back. Additionally, we will examine the blood, immune cells, and tumor samples taken from the patients to better understand the effectiveness and the shortcomings of the treatment approach in relation to tumor biology. We believe that, through our collaborations, we will help establish a pathway for conducting multi-center clinical trials of complex cell therapies and thus, make a difference to children with cancer in the long-term.
“With the support from Cookies for Kids’ Cancer … we will help establish a pathway for conducting multi-center clinical trials of complex cell therapies and thus, make a difference to children with cancer in the long-term.” - Meenakshi G. Hegde, MD
Memorial Sloan Kettering Cancer Center: $200,000 Grant
Project: Enhancing the efficacy of GD2 Cancer Vaccine for high-risk neuroblastoma by augmenting seroconversion; Recipient: Nai-Kong V. Cheung, MD, PhD
50-60% of patients with neuroblastoma are initially diagnosed with often inoperable solid tumor and metastatic disease. Intensive chemotherapy, aggressive surgery, and antibody- based immunotherapy have improved remission rates in young patients and to a lesser extent in adolescents and adults. The constraint of FDA-approved antibody-based therapy includes the side effects of pain and neuropathy during infusion. Another type of immunotherapy using a GD2/GD3 vaccine developed at Memorial Sloan Kettering Cancer Center has proven to have no pain nor neuropathic side effects. The addition of an immune enhancing molecule called beta-glucan, a dectin-1 receptor, has increased a critical marker of success for the vaccine therapy called seroconversion. Seroconversion means that the patient has developed circulating antibodies induced by the vaccine and targeting the immune system to neuroblastoma cells. While this is an important finding, evidence suggests that only a fraction of the patients benefitted because of a common genetic change that negates the benefit of beta glucan (dectin-1 single nucleotide polymorphism). This study explores alternative molecules that we know perform similar functions to beta glucan but are dectin-1 independent in mouse models in an attempt to enhance seroconversion. These preclinical studies should lead to FDA investigational new drug status allowing for the potential initiation of clinical trials.
“We appreciate your tireless support of our efforts to find a better and safer treatment for a diagnosis that has consumed me for nearly 40 years. … I won't give up until my promise to parents is fulfilled. … the fight goes on.” - Nai-Kong V. Cheung, MD, PhD
Children's Hospital of Philadelphia: $200,000 Grant
Project: Functional characterization and armoring of peptide-centric CAR T cells to enhance efficacy; Recipient: John M. Maris, MD*
Neuroblastoma is an aggressive childhood cancer and is often lethal despite intensive chemotherapy, radiation therapy, and immunotherapy. New cellular immunotherapies (e.g., chimer antigen receptor [CAR] T cells) have revolutionized the care of children with leukemias but as yet have not made a major impact on childhood solid cancers like neuroblastoma. One of the major barriers to progress is finding immunotherapy “targets” on the surface of cancer cells that are not present on normal cells. We recently developed an “immunopeptidomic” strategy to discover highly specific neuroblastoma cancer targets that are absent in normal body tissues. These are different from conventional CAR T targets (proteins on the surface of cells) as we focus on short fragments of proteins (peptides) derived from highly essential cancer proteins that are required for the cancer cell to survive. A clinical trial of our first “peptide-centric” CAR (PC-CAR) T-cell therapy for neuroblastoma will open in the summer of 2024. With the support from Cookies for Kids’ Cancer, we plan to first discover the key mediators of PC-CAR effectiveness using samples from both mouse and human studies. Then, we plan to add a key immune system modulator, IL-15 to our PC-CAR T cells to both improve the health of the curative T cells and help them find the tumor target. Successful completion of this project will create a “2nd generation” PC-CAR T cell “armored” with IL15 that will safely enhance the efficacy of this cellular therapy in children with neuroblastoma. Lessons learned here will be applicable to various PC-CAR T cell therapies we are developing for childhood cancers.
Texas Children’s Cancer Center/Baylor College of Medicine: $100,000 Grant
Project: To research the Outcomes and Health Risks among Individuals with Genetic Predispositions to Cancer: The ORIGen Study; Recipient: Philip J. Lupo, PhD, MPH
More than 10% of children diagnosed with cancer have a “genetic predisposition,” which could have an impact on treatment decisions and long-term outcomes, as well as genetic testing and counseling strategies for their family members. Despite this, there have been few attempts to comprehensively collect information on outcomes in these children and their family members. To address this critical gap in knowledge, we will develop the Outcomes and Health Risks among Individuals with Genetic Predispositions to Cancer (ORIGen) Cohort – a nationwide study that will lay the groundwork for developing personalized care plans for these children and their families.
St. Jude Children's Research Hospital: $200,000 Grant
Project: Evaluation of PARP1 selective inhibitor AZD5305 in combination with DNA damaging agents in Ewing sarcoma; Recipient: Elizabeth A. Stewart, MD
Ewing sarcoma is an aggressive bone and soft tissue cancer that occurs predominantly in children and young adults. For patients with recurrent disease, the prognosis is poor and new treatments that are less toxic are desperately needed. Dr. Stewart’s proposal aims to evaluate a unique combination of chemotherapy using a new drug called AZD5305. This drug has already been shown to be safe and effective in adult cancer studies; however, it has not been studied in pediatric cancers like Ewing sarcoma.
University of California San Francisco Benioff Children’s Hospital: $200,000 Grant
Project: Exploiting DNA repair mechanisms to overcome CIC-DUX4 sarcoma; Recipient: Ross Okimoto, MD
Our studies will develop a new therapy for children and adolescent/young adult (AYA) patients with a rare, but deadly form of cancer, known as CIC-DUX4 sarcoma. To date, there have been no clinical trials for CIC-DUX4 patients, and therefore, these patients are treated with “salvage” type chemotherapy derived from other types of sarcoma. We believe the most effective way to overcome cancer is to develop therapies that preferentially kill cancers cells but spare our normal cells. In order to design smarter drugs that target cancer specific lesions, we first need to identify the important lesions to target. In some sarcomas, including CIC-DUX4 fusion-positive sarcomas, the specific lesion is already known and fully transparent. These cancer specific lesions termed “fusions”, arise when a piece of DNA breaks and inappropriately attaches to another piece of DNA forming a fusion gene. Fusion genes are often used to diagnose certain cancers, as they can be the main drivers of cancer growth and survival. Therefore, a rationale way to target gene fusions is to identify how these lesions lead to cancer progression. Using CIC-DUX4 (a gene fusion) as a model system we have uncovered the molecular engine that drives tumor proliferation and survival in this deadly type of cancer. Fortunately, there are smart drugs that are already clinically developed that can be repurposed to specifically shut down the engine that drives CIC-DUX4 sarcomas. We want to demonstrate in tumors derived directly from CIC-DUX4 patients that blocking and shutting off this disease promoting engine with these smart drugs can kill CIC-DUX4 cancer cells. Since these drugs are already in advanced clinical trial development, we want to re-purpose them to precisely target CIC-DUX4 sarcomas, which currently has no therapeutic options.
Children’s Oncology Group, Pediatric Early Phase Clinical Trial Network (PEP-CTN): $276,500 grant
Since 2012, we have granted $3,546,800 to the Pediatric Early Phase Clinical Trial Network (PEP-CTN). We match dollar for dollar the funding the National Cancer Institute (NCI) provides for every child enrolled at the 21 participating centers which are located across the country. For seven years of our funding, we actually doubled the amount the NCI funds.
Some key takeaways:
- We are the largest philanthropic supporter of this program which essentially makes phase 1 treatments available for more kids.
- Last year, support from Cookies for Kids’ Cancer went to 23 institutions for 79 children who enrolled on PEP-CTN studies.
- Since 2012, support from Cookies for Kids’ Cancer went to more than 40 institutions for more than 1,000 children who enrolled in early phase studies.
- The Pediatric Early Phase-Clinical Trial Network (PEP-CTN) is comprised of 21 premier Children Oncology Group (COG) pediatric core member sites in the U.S. and 21 non-core member sites in the U.S., Canada and Australia that were selected through a peer review process. Non-core member sites join for
phase 2 components of studies and for pilot studies.
*Members of the Medical Advisory Board whose hospital’s grant request is being reviewed recuse themselves from the discussion to ensure objectivity
Note: The above grants, excluding Children's Oncology Group, are to be allocated over a stipulated time with an additional year of funding being contingent on a progress report.
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