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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.

This grant supports the Childhood Cancer Survivorship Program at Valley Children's Hospital, providing critical patient data for survivorship researchers.

Childhood cancer kills more children each year in the US than any other disease. Upstate Golisano Children's Hospital's mission is to conquer childhood cancer through superior care, research, and comprehensive support. Upstate Golisano Children's Hospital provides the medical expertise for children, adolescents, and young adults in the central New York area. They do this by participating in national treatment trials sponsored by the Children's Oncology Group (COG). 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 Center for Cancer and Blood Disorders at Phoenix Children's Hospital is the only program in the Southwest offering phase I clinical trials for children with cancer, enabling patients to receive the newest, most promising treatments available. These studies are critical for patients whose cancers cannot be cured by currently available treatments. The Early Drug Development Program Clinical Research Assistant supported by this grant will help to conduct these cutting edge trials.

This grant supports a Clinical Research Nurse to ensure that more kids can be treated on clinical trials, often their best hope for a cure. In the past, children diagnosed with cancer in the Rio Grande Valley had to travel to cities such as Houston or San Antonio to get state-or-the-art treatment through clinical trials. Physicians at the Vannie Cook Clinic in the Rio Grande Valley now have access to the most advanced trials and latest medications through Children’s Oncology Group and other Texas-based clinical trials. This gives patients the opportunity to have the optimal outcome from their cancer therapy. More importantly, parents know that their child is getting the best care available, anywhere. That gives families hope that their child will live a long, healthy life.

Malignant rhabdoid tumor (MRT) and atypical teratoid/rhabdoid tumor (AT/RT) are rare but devastating childhood cancers. Most children diagnosed with MRT and AT/RT are under the age of two, and most will die from their disease despite intensive treatment interventions. New insights into what causes these cancers, and new therapies, are desperately needed. Genetically, MRT and AT/RT are simple cancers, caused by loss of just one gene called SMARCB1. If we are to understand and treat MRT and AT/RT, therefore, we need to understand how loss of SMARCB1 causes cancer.

As the recipient of the Oh Danny Boy I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund St. Baldrick's Research Grant, Dr. Tansey is working on an innovative molecular mechanism through which loss of SMARCB1 causes MRT and AT/RT. He proposes that these mutations drive cancer by stimulating the activity of a known pro-tumorigenic gene called MYC. Dr. Tansey further proposes that MRT and AT/RT can be effectively treated by drugs that block the actions of MYC, currently being developed by us and others. He is testing this model and exploring its therapeutic implications. Completion of this work has the potential to transform the understanding of how MRT and AT/RT form and how they can be treated. Danny O’Brien was five months old when he was diagnosed with a rare malignant rhabdoid tumor on his liver. Despite treatment to shrink the tumor, he passed away at the tender age of 9 months. Fortunately, he knew nothing but love and affection for all of his short life. This fund honors Danny’s courage and remembers his unconditional love in the midst of his battle with cancer.

The shape and function of bone, fat, muscle, and other connective tissues evolve through a carefully orchestrated process that leads mesenchymal stem cells (MSCs) to progressively differentiate into more lineage-restricted tissue-specific phenotypes. As this occurs, MSCs must interpret their surrounding extracellular milieu. When everything works correctly, normal mesenchymal tissues emerge. When disrupted, as tragically occurs with Ewing sarcoma (ES), the aberrant fusion protein (FP) acts as powerful transcription-factor that corrupts the epigenetic program and locks ES in an undifferentiated state unable to interpret or respond to the biophysical cues present in the tumor microenvironment. Attempts to understand the FP’s effect upon tumor-ECM interactions within monolayer culture systems that lack a native tumor microenvironment has contributed, not unexpectedly, to spurious results that overestimate the clinical effectiveness of chemotherapy. To close this gap, Dr. Ludwig's multi-disciplinary team is using an innovative 3D tissue engineered model, pioneered by his laboratory, to assess next-generation EWS-FLI1-targeted therapies within a physiological microenvironment that cannot readily be studied in vivo. This project will shed new light on ES biology and promises to improve the ability to co-target the FP and other proteins that maintain the aggressive, poorly differentiated state of ES.

This grant is generously supported by the Shohet Family Fund for Ewing Sarcoma Research. Noah was diagnosed with Ewing sarcoma in his freshman year in college. After limb salvage surgery and chemotherapy, he was able to return to school. Two years later, Noah relapsed. This Hero Fund honors his courageous fight and hopes to raise funds for Ewing sarcoma research.

We can now manipulate the immune system with remarkable precision and efficacy to fight certain cancers. However, tumors adapt to reduce immunotherapy efficacy. This is true for solid tumors including osteosarcoma. Therapy-refractory metastatic osteosarcoma represents a pressing unmet need, as the outcome has not improved for over 3 decades. This multi-institutional collaborative proposal explores tumor-extrinsic pathways by which pulmonary metastatic osteosarcoma evade immunity. Dr. Huang’s team is focusing on key molecules responsible for such evasion, against which existing or emerging therapeutic agents are available currently or in the very near future. Therefore, uncovering these pathways could realistically offer multiple new clinical trials for pediatric and AYA metastatic osteosarcoma in the next 3 years. This Osteosarcoma Collaborative St. Baldrick's Grant to Cure Osteosarcoma is funded through the generosity of the Osteosarcoma Collaborative.

Despite the use of multimodal treatments and the implementation of several clinical trials worldwide, pediatric cancers survival rate has come to a standstill for the last decade. Moreover, intensive therapies are not devoid of long-term side effects, notably increasing lifetime risk for secondary malignancies. The duty of the pediatric oncologist is to propose the most adequate treatment to cure pediatric patients with the best quality of life for a long time. Therefore, understanding the biological underpinnings of pediatric malignancies is crucial to develop new therapeutic paths adapted to the specificities of a young organism. A major pitfall is the lack of adequate experimental models. To overcome this problem, Dr. Broutier is developing patient-derived 3D-organoid models (mini-tumor growing in a dish) of pediatric cancers. Beside their broad interest for research community, she will use them to identify mechanisms involved in cell death resistance in pediatric cancers, as a key step towards development of new targeted therapies adapted to children and adolescents.

Nearly half of all children diagnosed with Acute Myeloid Leukemia (AML) will suffer a relapse after initially successful treatment. Whereas therapy efficiently clears the bloodstream of leukemia cells, frequent evidence of residual drug resistant disease points to a leukemia protective role of the bone marrow microenvironment. The mechanism by which the bone marrow acquires these protective abilities is not clear. Dr. Kurre recently observed that so called stroma cells, that provide bone marrow structure and support are functionally altered. Pilot studies conducted by Dr. Kurre also identified a new mechanism by which AML changes these stroma cells. In this project Dr. Kurre is studying pediatric AML samples to understand how the altered stroma protects leukemia cells from the effect of drugs commonly used to treat children with AML. The long term goal is to develop treatment approaches that reduce the burden of relapse, by maintaining initial remissions in children with AML, and without further escalating drug toxicity. Awarded at Oregon Health and Science University, and transferred to The Children's Hospital of Philadelphia.

Adoptive immunotherapy has demonstrated unprecedented clinical success in the treatment of leukemia. In this therapy, T cells are isolated from a patient, expanded outside of the body, and genetically modified prior to reinfusion. The ability of these T cells to recognize and eliminate cancer cells is improved by expressing a protein (CAR) on the T cell surface. This protein increases "specificity," the ability to recognize cancer cells, and "function," the ability to destroy those cancer cells. An important challenge in cellular immunotherapy is to minimize the manipulation of patients' T cells outside the body. Prolonged culture protocols trigger functional exhaustion and compromises their efficacy upon return to the body. A critical issue involves the pre-requisite "activation" step necessary for CAR expression on the cell surface of T cells. By optimizing culture conditions, Dr. Ghassemi developed approaches to express CAR in dormant T cells without the need for activation. Importantly, her preliminary findings show that elimination of this activation step retains effector function and potency of CAR T cells in models of the pediatric cancer ALL. This project is providing insight into the regulatory components influencing CAR expression in dormant T cells which will lead to superior CAR T cells for cellular immunotherapies against ALL.

This grant is supported by TEAM ABBY Gives, a St. Baldrick's Hero Fund. Abby was diagnosed with Pre-B ALL when she was almost five years old. She had a successful bone marrow transplant, but battle battled graft vs. host disease (GVHD) for years. Abby and her treatment team worked hard over many years to keep the GVHD in check. Sadly, Abby passed away on October 19, 2021. This fund unites the incredible support of family and friends in Abby's memory and inspires others to join the fight for cures and better treatments.

In the U.S., children with a blood cancer called Hodgkin lymphoma (HL) are usually treated successfully. Some of these children will suffer health problems several years later because of the treatment they received. Because of this, doctors use powerful imaging tools to identify patients who are likely to do well or not. Those who are likely to do well require less treatment and those who are less likely to do well can receive more treatment. But in low-income countries like Malawi, these tools are unavailable, and the children there often receive treatment that may be unnecessary. Scientists have found unique abnormalities in adults with HL that can tell us who is less likely to do well. Here, Dr. Ozuah is testing whether these abnormalities are present in children and could be used to decide how best to treat children with HL in low-middle income countries

Osteosarcoma is the most common primary bone tumor in childhood. The survival rate remains dismal, mainly due to ineffective therapeutic approaches for the relapsed/metastatic patients. One major obstacle of treating osteosarcoma is lack of suitable preclinical models. Dr. He's studies have established the first cultured osteosarcoma tissue model (an organoid). Dr. He aims to establish the first biobank of osteosarcoma organoids from patients as an open resource for the field, and utilize this organoid biobank to evaluate a novel class of therapeutics targeting key signaling pathways in osteosarcoma cells. This study will provide a powerful platform for predicting clinical treatment responses and developing new therapeutics for treating osteosarcoma.

Pediatric brain tumor patients are at risk for deficits in cognitive functioning, including attention, memory, and processing speed. Evaluation of these functions by neuropsychologists is important for connecting patients with appropriate interventions to reduce academic or vocational difficulties. Despite the importance of neuropsychological services, traditional assessments are time consuming and costly for both providers and families. As such, neuropsychological surveillance is often limited. Due to advances in technology, computerized neuropsychological batteries were developed that can be completed via internet in the home. As the recipient of the Grace for Good Fund St. Baldrick's Supportive Care Research Grant, Dr. Raghubar is determining if children diagnosed and treated for brain tumor can successfully complete web-based tests. Patients currently receiving annual neuropsychological evaluations will be asked to complete a web-based battery. She will evaluate 1) the rate of successful completion; 2) the relationship between performance on the computerized and traditional neuropsychological tests; and 3) the degree to which patients are similarly categorized as demonstrating impaired versus non-impaired neurocognitive abilities when assessed using either form of assessment. This line of research has the potential to improve the clinical care of patients and survivors. She may also find a solution to the problems that have plagued late-effects research for decades, such as poor participation and high dropout rates.

This grant is named for the Grace for Good Fund in honor of Grace Carey and celebrates her survivorship from medulloblastoma. While Grace handled her treatments with minor setbacks, she now faces challenges wrought by the very medications and procedures that saved her life. This fund was inspired by her desire to help other kids with cancer and supports research of brain tumors and the multitude of challenges facing survivors post treatment.

Despite cure after cancer, the majority of childhood cancer survivors are diagnosed with chronic health problems such as diabetes, hypertension, or heart disease within 30 years of cancer diagnosis and treatment. Hypertension and diabetes are important health problems associated with heart disease and result in early death compared to survivors without heart disease. Compared to siblings, childhood cancer survivors treated with radiation have a higher risk of developing diabetes at a young age. Even though important, the reasons for developing diabetes in cancer survivors are poorly understood. Insulin is an important hormone and affected by cancer treatment. Dr. Mostoufi-Moab is for the first time examining cancer treatment effects (radiation vs. chemotherapy) on making insulin and breaking down blood sugar in childhood cancer survivors, age 15-30 years. She will use tests to measure glucose breakdown, insulin secretion, and mathematical modeling to evaluate the body's handling of fat, all important steps for understanding the why cancer therapy causes diabetes in CCS. Understanding the steps leading to diabetes will help doctors chose the correct treatments for diabetes and for scientists to test options to prevent diabetes in cancer survivors. Achieving these goals will improve quality of life and avoid early death due to cancer treatment-related conditions in childhood cancer survivors.

Many chemotherapy drugs used in childhood cancer have the potential to damage peripheral nerves and thus may impact a child’s ability to function. Early detection of this nerve damage is important to tailor treatment plans and initiate rehabilitation interventions. Currently, no measure of chemotherapy-induced peripheral neuropathy (CIPN) exists that is appropriate for use in children ages 1-4 years, an age group that is more commonly impacted by a number of childhood cancers. Thus, Dr. Gilchrist is finalizing the development of a scale of CIPN specifically tailored for young children that focuses on eliciting responses to test nerve function (such as tendon reflexes and strength of muscle groups) as well as developmentally appropriate measures of motor function (such as grasping an object or standing on one foot). She is also comparing results from 25 children ages 1-4 years being treated for cancer with neurotoxic treatments and 25 age and gender-matched controls to determine if the measure is both reliable and valid. If successful, she will be able to provide an objective measure for this common treatment side-effect that should positively impact both research and clinical care for these young children.

With increased survival for children with cancer, efforts that prevent long-term health problems are important for improving the quality of life and life expectancy of these children. Diet and fitness are two critical factors for healthy survivorship, but interventions for survivors of childhood cancer have had limited impact, focus almost exclusively on physical activity, and often exclude caregivers, the primary nutrition gatekeepers in the home. Although research supports a key role for the gastrointestinal (GI) microbiome in regulating weight and health outcomes, no studies have examined the “obesogenic” microbiome in the context of interventions for these survivors. Harvesting Hope for Kids (HH4K) is a unique, biobehavioral lifestyle intervention delivered over 8 weeks during the summer in a university-based, cancer survivor garden. It was adapted from a successful intervention for survivors of adult-onset cancer, with pilot data supporting its feasibility in children. In line with St. Baldrick’s mission to improve outcomes for children with cancer, this randomized controlled trial is evaluating the efficacy of HH4K to improve dietary and physical activity patterns in 40 survivors of pediatric cancer (i.e., ages 8-12; < 2 years off treatment). Results will support a larger, multi-institutional trial and improve survivorship care to prevent costly, long-term morbidity.

This award allows Dr. Parsons the freedom to pursue discovery without the restrictions of a normal grant. The questions he is exploring include: What are the biologically and clinically-relevant genomic alterations in high-risk and rare pediatric cancers? What are the most useful and cost-effective clinical sequencing tests for childhood cancer patients? How can clinical genomics/precision oncology be most effectively implemented for diverse patients and families in varied clinical settings? What clinical benefit can precision oncology approaches and the use of molecularly-targeted therapies offer to childhood cancer patients? He is optimistic that a "team science" approach, bringing together investigators from diverse disciplines, departments, and institutions, will continue to yield critical data and discoveries that guide pediatric cancer research and clinical care in the next decade.