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The most difficult cases of Ewing sarcoma to treat are when it comes back after completing initial treatment. Dr. Brian Ladle and colleagues at Johns Hopkins University and the National Cancer Institute believe the immune system is capable of mounting a powerful immune response against Ewing sarcoma as an effective treatment. Dr. Ladle and team will identify the best immune targets in Ewing sarcoma and discover ways to activate the immune system against the most promising targets. These targets will be tested in their lab using models.

To make a significant impact for kids fighting Ewing sarcoma, five funding partners have banded together with St. Baldrick’s to support this grant – Advancing Cures for Ewing Sarcoma (ACES) award supported by the Sam Day Foundation, The Faris Foundation, Rutledge Cancer Foundation, The Shohet Family Fund for Ewing Sarcoma Research (a St. Baldrick’s Foundation Hero Fund), and Alan B. Slifka Foundation.

Dr. Stanton and colleagues are developing new approaches to understand how DNA is organized for gene expression in a lethal childhood tumor called rhabdomyosarcoma (RMS). In RMS, the standard of care therapies haven't changed substantially in 40 years and patient outcomes haven't improved greatly during this time. New approaches are desperately needed. There is a lack of a fundamental understanding of the mechanisms for how the cancer-causing genes function in RMS. Dr. Stanton and his team are integrating cutting edge approaches in synthetic biology, modeling, and genomics to understand how and why RMS forms, with the granularity of single gene targets for mechanism studies. Through Dr. Stanton's studies, the community will gain an understanding of how the cancer-causing genes are altering the organization of DNA in RMS cells.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. Aiden Binkley who was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Dr. MacDonald and colleagues have developed a new test that detects cancer cells in the blood and other fluids of patients with brain tumors. They have discovered that children with a particular type of brain tumor often have cancer cells in their blood. They can also use their test to follow how treatment changes the number of cancer cells in the blood to find out if the treatment being given is working to kill all the remaining cancer cells in the brain. This is the first time such a test has been able to do this using a simple blood draw. Dr. MacDonald and colleagues will use their test for children with other types of brain tumors. Dr. MacDonald and team will study the cancer cells in the blood to see why and how these brain cancer cells are still alive after treatment. This will help to identify the "steps" in which the cancer cells take to avoid being killed by the treatments being given and ultimately will then lead to new treatments targeting these steps to cure childhood brain tumors.

Children with a brain tumor diagnosis face the daunting prospect of surgery, radiation and chemotherapy. While these treatments can be successful, some brain tumors continue to grow and spread in the brain, which can make them impossible to treat. In addition, current treatments can adversely impact a child's cognitive, emotional and physical well-being. New treatments against the most aggressive brain tumors to reduce the burden of tumors in children and increase their quality of life are needed. Dr. Stewart and colleagues will develop new technologies and test new drugs that will allow to rapidly transplant patient tumor cells into models to make hundreds of model avatars that can then be treated with a panel of drugs to identify the most effective treatments for a child's specific tumor, focusing on very aggressive tumors first. Dr. Stewart and team expects this information will be used to help guide doctors decisions on the best treatment options to eliminate the child's tumor and minimize side effects.

Children diagnosed with a brain tumor called diffuse midline glioma (DMG) have no options for a cure. Dr. Ramakrishna and team at Stanford Medicine have developed a new treatment, called CAR T cells, for these children by training their immune system to find and kill cancer cells. Excitingly, children treated on a clinical trial with these CAR T cells have improved symptoms and reduced tumor sizes. Unfortunately, for some patients, tumors grew after treatment, suggesting a need to understand how to improve the treatment. An immune cell, called a myeloid cell, was identified surrounding treatment of the first patients. Dr. Ramakrishna and colleagues will seek to understand these myeloid cells in the context of CAR T cell activity in patients. Modeling these myeloid cells in the lab, Dr. Ramakrishna and team will test approaches to improve CAR T cell activity against DMG cells. This project will improve understanding of CAR T cells in patients and develop new treatments for children with these devastating brain tumors.

Sneha Ramakrishna, MD, is a pediatric hematology-oncologist at Stanford Medicine Children’s Health and an assistant professor - University Medical Line in Pediatrics - Hematology & Oncology at Stanford Medicine.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

Each year, approximately 1,000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, no drugs can eradicate all AML cells in pediatric patients, and cells remaining after treatment often cause disease recurrence and poor survival. Dr. Li and colleagues have found that two mitochondrial enzymes that function in energy metabolism, known as DHODH or SDH, shield leukemia cells from eradication by immune cells. Dr. Li will use models relevant to pediatric AML to ask how these factors block anti-cancer immune responses and test effectiveness of a first-in-class leukemia cell-specific DHODH inhibitor combined with existing immune therapy in eradicating AML. If successful, this study will lead to development of new anti-leukemia drugs that could approach a cure for childhood AML.

A subset of deadly pediatric cancers, known as sarcomas, are caused by a mysterious, mutated protein called CIC::DUX4. Treating this disease is very difficult, in part because little is known about its biology. The teams of Dr's. Wood and Hendrickson have assembled the world's best collection of CIC::DUX4 laboratory models, using them to discover that CIC::DUX4 sarcomas require a protein called COP1 for their survival, whereas normal tissues in bodies do not. Dr. Wood and colleagues will perform studies to understand how well future drugs targeting COP1 will work as therapies for this deadly disease while defining why this disease needs COP1 in the first place. Together, these studies will identify a highly promising new therapeutic strategy for CIC::DUX4 sarcoma while also revealing insights that could lead to additional new types of drugs for this disease.

Neuroblastoma (NB), a cancer that commonly affects young children, often presents with aggressive clinical behavior and poor prognosis, making the identification of effective therapeutic targets essential. NB is known for its resistance to conventional chemotherapy, and one of the mechanisms contributing to this resistance is the activation of a key regulator of gene expression, known as 'NF-kB’. NF-kB activates the expression of genes that contribute to NB survival. NF-kB also plays a role in promoting spread of NB to other parts of the body (e.g. bone marrow, liver, and lymph nodes). Because of its critical role in regulating survival, inflammation, and metastasis, NF-kB presents an attractive target for novel therapeutic strategies in NB. Inhibition of the NF-kB pathway can potentially sensitize NB cells to chemotherapy, reduce tumor growth, and inhibit metastasis. Dr. Letterio and colleagues will explore the activity of a new class of drugs (known as SOTs), that are potent inhibitors of NF-kB.

This grant is named for David's Warriors, a St. Baldrick's Hero Fund. The fund was created in memory of David Heard who battled neuroblastoma until his passing at the age of ten. David inspired his family and countless others to commit to raising money for research to fight pediatric cancer through the St. Baldrick’s Foundation. The Fund honors the amazing spirit with which he lived, embracing life until the very end.

Ewing sarcoma commonly appears in a bone among patients between 10-20 years old. About 25% of patients present with a clinically detectable metastatic disease. Despite aggressive chemotherapy and radiation, almost no improvement has been seen in patients with metastatic disease (80% mortality). The failure to stop Ewing's sarcoma metastasis is partially due to the lack of understanding about the molecular pathways that regulate its spreading. Dr. Yang and colleagues will study several upstream regulatory genes of TWIST1 in Ewing sarcoma metastasis and test whether drugs targeting these genes will block metastasis with higher specificity and fewer side effects than conventional therapy. In the long term, this research will lead to novel therapeutic regimens for Ewing sarcoma metastasis.

Brain cancer is the leading cause of cancer-related deaths in children, highlighting the urgent need for more effective treatments. While chimeric antigen receptor (CAR) T-cell therapy has transformed outcomes for children's blood cancers, it has shown limited success in brain tumors. Dr. Huang and colleagues will initiate a phase I trial in children with HGG and DIPG to assess safety and immune effects utilizing the understanding that CD70, a protein driving tumor growth, is a promising CAR T-cell target for high-grade gliomas (HGG) and diffuse intrinsic pontine gliomas (DIPG).

A child diagnosed with rhabdomyosarcoma (RMS) today is subjected to essentially the same therapy used fifty years ago. While Drs Mosse and Maris’s research programs have traditionally focused on neuroblastoma (NBL), both labs have recently extended their research programs to other pediatric solid tumors with high unmet need. Here they propose two parallel and complementary projects designed to create new immunotherapies for RMS, with Project 1 focused on the discovery of new immunoncology targets and cellular therapies, and Project 2 focused on the IND-enabling studies of a novel antibody-drug conjugate (ADC) directed against ALK for fusion positive RMS. This grant is supported by Alice's Arc US Inc. Alice's Arc operates in the UK and USA and is dedicated to funding research into finding a cure and less harsh treatments for rhabdomyosarcoma. The charity has created a global community of families, scientists and doctors coming together to help achieve this mission. Families are at the heart of the work and they can choose to create an Arc in their child/young person's name to represent their experience with rhabdomyosarcoma. This enables funds to be pooled for research, powerful advocacy and a long-term, sustainable platform to grow the charity and fund a pipeline of rhabdomyosarcoma research.

The recent development of therapies that stimulate the immune system to eliminate cancer has transformed treatment options for many patients. However, these therapies have generally been less successful in treating childhood cancers, in part because cancers in younger patients typically have acquired fewer of the genetic alterations that can be recognized by T cells, the immune cell type most commonly used for cancer treatment. This project aims to harness the cancer clearing functions of macrophages, a distinct immune cell type that can recognize and kill even those cancer cells that carry few genetic alterations. Dr. Kamber and colleagues will focus on identifying strategies that unleash macrophage anti-cancer functions in the context of Burkitt lymphoma, an aggressive form of lymphoma that is among the most common types of cancer in children.

This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

The AACR-St. Baldrick's Foundation Award for Outstanding Achievement in Pediatric Cancer Research has been established to bring attention to major research discoveries to the pediatric cancer research community and to honor an individual in any sector who has significantly contributed to any area of pediatric cancer research, resulting in the fundamental improvement of the understanding and/or treatment of pediatric cancer. The recipient will nominate an emerging leader conducting research in the academic sector to receive a research grant. The 2024 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. Ching-Hon Pui at St. Jude Children's Hospital. Dr. Shawn Lee at National University of Singapore received the 2024 research grant. Dr. Lee's research interests are in pharmacogenomics, immunogenomics and leukemia.

In some types of cancer that affect children and teenagers, there are special proteins called 'fusion oncoproteins' that play a big role. These proteins are made when a gene called MLLT10 gets mixed up with other genes. These cancers are very dangerous and don't respond well to treatments available now. Dr. Lui and colleagues research is focused on the most prevalent MLLT10 fusion oncoproteins, common in a type of cancer called T-cell acute lymphoblastic leukemia (T-ALL) in kids and young adults. Findings show that this fusion protein makes groups of biomolecules called 'condensates,' which can mess up how cells read and use their genetic instructions. Dr. Lui believes that by studying these MLLT10 fusion oncoproteins in detail, they may learn how they change cells and find ways to stop them. Dr. Lui also believes if they can figure out how MLLT10 fusion oncoproteins work, it may also help to understand other similar fusion proteins. That knowledge could help develop better treatments for these kinds of cancer.

This grant is funded by and named for Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

It is known that children with cancer have higher rates of hospitalization, ICU admission, and death than children without cancer and COVID-19. Children with cancer and COVID-19 also frequently have changes in their chemotherapy. Yet, critical data is lacking regarding COVID-19 in children with cancer and guidelines about how to manage these vulnerable children. Dr. Johnston and collegaues will leverage the national registry of children with cancer and COVID with data on >2,400 children from >100 institutions to examine (1) how the clinical course of children with cancer and COVID-19 compares to earlier in the pandemic, (2) how the clinical course of COVID-19 in children with cancer is impacted by vaccination and antiviral therapy, and (3) physician and healthcare systems factors that influence COVID-19 management. Dr. Johnston will use that information, literature review, and expert discussion to inform an expert panel tasked with developing guidelines for management of COVID-19 in children with cancer.

Cancer patients take life-saving drugs that, unfortunately, can result in peripheral nerve damage. For example, many patients receiving cisplatin experience permanent hearing loss. There is one therapy that has been approved to mitigate cisplatin-induced hearing loss, however, the reduction in hearing loss is modest (< 30%) and this mitigating treatment is associated with poorer overall survival rates due to inhibition of cisplatin's cancer-fighting properties. Thus, it is approved for low-risk pediatric patients only. To develop a better alternative, Dr. Rutherford and colleagues are testing novel compounds they have developed at Washington University, which have shown to protect the ear from noise trauma. With hearing tests and with anatomical measurements of the cochlea, Dr. Rutherford will attempt to prevent hearing loss following cisplatin treatment in models. After this innovative project proves successful, subsequent model studies will determine if Dr. Rutherford's therapy inhibits cisplatin's cancer-fighting role.

The study of genetic disease of cancer predisposition has served as a model for understanding cancer in general. Fanconi anemia is a rare genetic disease of failed blood production and cancer proneness, including leukemia and head and neck cancer. The genes and encoded proteins participate in DNA repair. However, an examination of cancer databases of DNA sequence shows that Fanconi genes are mutated in up to 30% of all head and neck cancers in non-Fanconi patients. Dr. Kupfer and colleagues have studied one particular mutation that resides in the Fanconi FANCD2 gene that interrupts its protein binding to another important gene BLM, which also participates in DNA repair. This proposal will seek to study the normal function of the FANCD2-BLM interaction in the cell and the consequences of its disruption. Dr. Kupfer also seeks to identify ways disruption of the normal pathway will render cancers vulnerable to molecular targeting to improve therapeutics.

Survivors of pediatric brain tumors have a high risk of medical problems that can negatively affect the quality of their lives. Particularly concerning are effects on brain development, including learning and emotional well-being, and metabolism, which can lead to obesity and muscle loss. There is an urgent need for tools that can better predict which children are most at risk so that they can be offered treatments to prevent these problems. Dr. Kann's and colleagues have developed medical imaging tools that use artificial intelligence on routine brain scans to track and predict 1) muscle weakness and malnutrition, and 2) brain development in children. Dr. Kahn and team will test these tools in large datasets from hospitals and clinical trials of pediatric brain tumor patients and survivors to predict the risk of these negative effects in each patient. The tools developed may be used in clinical trials to improve quality-of-life for childhood brain tumor survivors.

Despite remarkable improvements in treatment for children with some types of cancer, pediatric brain tumors remain an area that desperately require more effective and low toxic therapy solutions. Dr. Jun Qi has formed a multi-disciplinary team to identify novel targets for pediatric brain tumors and develop new strategies to suppress the targets for patient treatment. Using a chemical strategy, Dr. Qi and his team aim to disrupt the functions of these targets to effectively inhibit brain tumor cell growth and block tumor progression in the models that resemble the real disease. The study focuses on improving on-target effect and, more importantly, on getting these potential drug candidates into the brain. The proposed study will translate from bench to bedside for patient care and result in a novel therapeutic strategy with significant improvements in survival and reduced morbidity for pediatric brain tumor patients to fulfill the mission of St. Baldrick's Foundation.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

Medulloblastoma (MB) is an aggressive childhood brain cancer that is fatal in 40% of patients. MB shows abnormal activation of growth pathways in tumor cells which help them to grow. Dr. Ganesan's studies show that there are T cells (immune cells) within MB and they have potential to kill tumor cells. However, immunotherapy that boosts the activity of these T cells have not been successful and it is not known why. The goal of this project is to understand why immunotherapy has not worked in MB and whether immunosuppressive myeloid cells contribute to this restraint. Dr. Ganesan and colleagues will also study if combined treatment that inhibits the growth pathways (targeted therapy) and stimulates the T cells/immune system (immunotherapy) may together lead to greater tumor killing in MB mouse models. To test if combined therapy would work in humans, Dr. Ganesan will coculture a 3D version of patient’s brain tumor with their own T cells expanded from their tumor. If effective, these studies may lead to new treatments for MB.