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This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

The University of Illinois at Chicago, Rush University Medical Center, and John H. Stroger Hospital of Cook County Program exists to meet the needs of an extremely diverse population who currently struggle with cancer or who have survived this terrible disease but are at great risk for many long-term health problems. UIC, Rush, and Stroger Medical Centers anchor the near west side of Chicago and serve incredibly vulnerable patients and families, the majority of whom have very limited personal resources, medical knowledge, and English language skills. This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Golisano Children's Hospital is committed to providing excellent care to all children, adolescents and young adults who are being treated or have been treated with cancer in their region. This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Adolescent Young Adult (AYA) Cancer Care at Bon Secours St. Francis Health System was established in 2012 to improve care of AYAs in Upstate South Carolina via novel care delivery model integrating pediatric and medical oncology treatment and psychosocial teams. This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

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.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

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.

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.

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.

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.

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.

Leukemia and lymphoma are blood cancers that are a major cause of death in children. Many of these cancers are curable with chemotherapy, but in some people the cancer comes back and is harder to cure. A new treatment called CAR-T cells involves genetic engineering of a cancer patient's own immune system cells to fight cancer, and can cure many people. However, this treatment still does not work well enough in about half the people who get it. Dr. Pauerstein proposes improving the sensitivity of CAR-T cells to cancer using engineered cell adhesion molecules, a type of molecular glue between two cells. CAR-T cells do not attach to cancer cells as strongly as normal T cells do, and this limits their ability to find and kill cancer cells. An engineered adhesion will be used in combination with CARs to improve the ability of CAR-T cells to kill cancer. Dr. Pauerstein and team will also study how changes in cell adhesion affect how CAR-T cells kill cancer. This work should improve cell-based treatments for blood cancers.

Acute myeloid leukemia (AML) is a blood cancer that affects children. AML treatment involves intensive chemotherapy that requires over 140 days in the hospital. This places immense financial burden on families including medical bills, transportation costs, childcare, and missed days from work. This burden and resulting distress are called financial toxicity. Dr. Zheng's research is focused on measuring financial toxicity and trying to figure out what can be done about it. One important idea to consider is that many parents quit their jobs or reduce their hours to care for their child. Dr. Zheng plans to use surveys and interviews to gain a clearer picture of how work disruptions develop over AML treatment and lead to financial toxicity. Dr. Zheng wants to identify what work arrangements and policies could offer the most support. Ultimately, it could lead to advocating for more flexible work schedules, remote work options, or other accommodations that could make a real difference for these families.

Hepatoblastoma is the most common liver tumor diagnosed in early childhood, and new therapies are urgently needed to improve survival and reduce treatment related morbidity. Immunotherapy is a type of cancer treatment that harnesses the body's own immune system to target and attack cancer cells. While some immunotherapies have been very successful against certain tumor types in adult patients, they have been largely unsuccessful in treating pediatric tumors. This demonstrates how little we know about how the pediatric immune system responds to tumors. Using samples and models of hepatoblastoma, Dr. Cabric's research aims to identify the key immune cells involved in recognizing and responding to hepatoblastoma. Identifying the key immune cells involved in tumor immunity, and mechanisms that allow tumors to escape detection and deletion by the immune system, will allow us to find novel targets for future immunotherapies that work in children. This grant is funded by Allied World, a global provider of insurance and reinsurance solutions.

Brain tumors are the leading cause of cancer related death in children. The outcomes for high-grade gliomas in children are dismal. Chimeric antigen receptor (CAR) T cells are genetically engineered cells programmed to target cancer cells with high precision. The application of CAR T cells in brain tumors in children is still limited compared to leukemia. One challenge is that CAR T cells need multiple hits to kill brain tumor cells compared with leukemic cells, where a single hit is sufficient. Dr. Abu Arja and team discovered a subset of CAR T cells that are more potent and can more proficiently kill brain cancer cells by increasing their lethality, making a second hit unnecessary. In this project, Dr. Abu Arja is studying the cellular program of this unique subset of potent killer CAR T cells to better understand why they are superior killers. Dr. Abu Arja plans to use these findings to genetically engineer new enhanced CAR T cells to eliminate tumors in children with brain cancers. The first year of this grant is funded by and named for the Be Brooks Brave Fund. Despite his diagnosis at age 5 with inoperable brain and spinal tumors, Brooks taught so many people what life is truly about--love. He was BRAVE beyond his years with an inspiring “faith over fear” attitude. This Hero Fund hopes to raise money for high-grade glioma research so no other family will hear the words, “there is no cure”.

Even after being cured, childhood cancer survivors face challenges to living a healthy life, and one major challenge is heart disease. Heart health is closely linked to healthy eating, but many survivors cannot eat as healthily as they want because they don't have access to, or can't afford, healthy foods ("food insecurity"). Dr. Aziz-Bose will enroll survivors in this study to ask what they are eating, and understand whether they experience food insecurity and other conditions that put heart health at risk. Survivors will also be interviewed for their ideas about how to support healthy eating, including the best ways to directly give families healthy foods, an approach called "food is medicine." Using this information, Dr. Aziz-Bose will fine-tune a "food is medicine" intervention that she developed, and test it on a larger scale to see its impact on food insecurity and heart health. The goal being to understand and tackle barriers to healthy eating so all survivors can have the best health possible. This grant is funded by Allied World, a global provider of insurance and reinsurance solutions.

Neuroblastoma is a devastating pediatric cancer, with only 50% survival in aggressive "high-risk" disease. Survivors are burdened with life-long side effects from chemotherapy and radiation. Newer therapies, such as cancer vaccines, provide an opportunity to mobilize a patient's own immune system to find and destroy cancer cells. Identifying the unique genetic signature of an individual patient's tumor allows scientists to formulate a personalized vaccine to stimulate the immune system to recognize tumor-specific mutations, called "neoantigens". Dr. Spear has developed a new tool to identify these unique genetic signatures (neoantigens) and test the effectiveness of the neoantigen vaccine in modes. These findings will lay the groundwork to develop a clinical trial using personalized vaccines for high-risk neuroblastoma and other pediatric cancers. This grant is funded by and named for the Arden Quinn Bucher Memorial Fund, a St. Baldrick's Hero Fund. Arden’s intelligence, empathy, and dynamic personality charmed everyone and is now her legacy. Before her neuroblastoma diagnosis on October 11, 2007 at age two, she happily played with boundless energy and imagination. Even throughout her difficult months of treatment, Arden bravely managed to keep smiling and learning. This fund supports St. Baldrick’s mission: funding the most promising research, wherever it takes place to provide kids fighting cancers less toxic, more effective treatments allowing them to live longer, healthier lives.