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Telomeres are special sequences of DNA located at the ends of every chromosome, and are essential to maintaining proper cellular function. If telomeres are damaged or degraded, they may cause healthy cells to transform into cancer cells. Dr. O'Sullivan and his team have discovered a protein called RAD51AP1 that appears at high levels in neuroblastoma tumor cells, and they have determined that having less of this protein stops telomere damage in cells. Dr. O'Sullivan is investigating the consequences and impact of RAD51AP1 inhibition on the proliferation and survival of neuroblastoma tumor cells.

This consortium is conducting the first multi-institutional, genomics-based precision medicine trial for children with relapsed, refractory, and very high-risk leukemias. They are deploying new technologies to sequence all currently known cancer-promoting genes in an individual child's leukemia. Next, guided by a multi-disciplinary group of leukemia experts, they will make treatment recommendations based upon the genomic findings in the child's leukemia and the availability of a relevant targeted drugs for children. Results from this project will define the types and frequency of specific mutations in children with relapsed/refractory leukemia, and assess the impact of the treatment recommendations on their clinical care. The consortium will also perform laboratory-based studies to determine the response to therapy in lab models derived from the patient's leukemia cells, to identify new drug targets for these children. Data generated in this proposal will inform future genomically-based targeted therapy trials for children with leukemia with the long-term goal of precise, safe, and more efficacious therapies for children with these diseases. Funds administered by Dana-Farber Cancer Institute.

This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age 6 she was diagnosed with anaplastic medulloblastoma. After a relapse and additional treatment, Hannah currently shows no evidence of disease. Throughout her treatments, Hannah never complained and remained positive and happy. This fund pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Based on progress to date, Dr. Sayour was awarded a new grant in 2019 to fund an additional year of this Scholar grant. For children affected by medulloblastoma, the development of more effective and specific therapies that will not add further toxicity to existing treatments is critical in improving clinical outcomes. Dr. Sayour, the Hannah's Heroes St. Baldrick's Scholar, is investigating methods to harness the immune system to destroy these tumors though the use of nanoparticle vaccines. Nanoparticles can deliver messages to the immune system teaching it to kill cancer cells. Dr. Sayour is studying possible immune targets for nanoparticles in medulloblastoma, identifying underlying tumor resistance mechanisms, and establishing an optimum vaccine approach which he will evaluate in medulloblastoma models.

This grant is named for Hannah's Heroes, a St. Baldrick's Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Initiative. For a description of this project, see the consortium grant made to the lead institution: University of California Santa Cruz, Santa Cruz, CA.

Based on progress to date, Dr. Shah was awarded a new grant in 2019 to fund an additional year of this Scholar grant. Finding a donor for a bone marrow transplant can be difficult for patients that are mixed-race or from an ethnic minority, as bone-marrow comes from donors that are genetically compatible. Dr. Shah is developing a method utilizing hemogenic endothelial cells (HEC) that will have the same effect, without requiring bone marrow from a matched donor. Dr. Shah is investigating FDA-approved drugs that stimulate the HECs to produce a novel and safe alternative to bone marrow-generated Hematopoietic stem cells, which will potentially treat childhood blood cancers. Awarded at the Brigham and Women's Hospital and transferred to Nationwide Children's Hospital.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

This grant funds a student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

Childhood cancer survivors can have problems with their lungs due to their cancer treatment. Dr. Kirchhoff is completing the first study to examine how air pollution affects the health of childhood cancer survivors.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Initiative. For a description of this project, see the consortium grant made to the lead institution: University of California Santa Cruz, Santa Cruz, CA.

Langerhans cell histiocytosis (LCH) is a rare cancer of the immune cells that can cause a wide range of symptoms, ranging from a rash to lethal multi-organ disease. Dr. Zinn hypothesizes that a patient's symptoms are determined by a combination of the patient's specific mutation and the specific immune cell that becomes mutated. Dr. Zinn is investigating the causes of LCH in order to develop the most effective and safe therapies for each patient.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

Adolescents and young adults (AYA) with cancer have an increased risk of developing secondary cancers, cardiovascular, metabolic and bone diseases as well as cognitive impairments, which can reduce their survival and quality of life. Furthermore, most AYA cancer survivors do not meet the recommended guidelines for physical activity. Dr. Nock is conducting a pilot study using 'cybercycling' (stationary cycling with interactive video gaming) to improve quality of life in AYA cancer survivors. She will also see if this exercise program improves their motivation to exercise, body composition (weight, body fat), fatigue, depression, and sleep habits.

Medulloblastoma (MB) is the most common malignant pediatric brain tumor, yet currently has no optimal treatment options. Medulloblastoma has been classified into 4 major subgroups, and Dr. Chen is targeting methylation mutations to develop improved therapeutics for two highly-aggressive subgroups of medulloblastoma. To facilitate this, Dr. Chen is establishing precision models of this disease to screen and test for therapeutics. To systematically identify protein targets required for survival of MB cells, Dr. Chen and colleagues are performing a genome screen to look for possible targets, in order to enhance understanding of this disease and lead to novel therapeutic routes.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Pediatric LEukemiA Precision-based Therapy (LEAP) Consortium. For a description of this project, see the consortium grant made to the lead institution: Dana-Farber Cancer Institute, Boston, MA.

Pediatric cancer patients that have high-risk solid tumors can be very difficult to treat, particularly when their cancer has come back after previous treatment or has spread to multiple areas of the body. Dr. Stewart is looking for specific mutations in the tumors of these patients, and testing new cancer drugs to customize treatment to give patients with relapsed high-risk solid tumors with better treatment options.

This grant is made with generous support from the Invictus Fund, a St. Baldricks's Hero Fund which was created in memory of Holden Gilkinson. It honors Holden's unconquerable spirit in his battle with bilateral Wilms tumor by funding cures and treatments to mitigate side and late effects of childhood cancer.

This grant funds a doctoral student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

Based on progress to date, Dr. Llosa was awarded a new grant in 2019 to fund an additional year of this Scholar grant. Osteosarcoma is a tumor that forms in the bones and is the most common bone tumor of childhood. Dr. Llosa is investigating how the immune system interacts with cancer cells from osteosarcoma tumors. Dr. Llosa's focus is on immunotherapy, a type of cancer treatment designed to boost the body's natural defenses to fight the cancer, and one of the most promising current approaches for treating tumors. Immunotherapy uses materials either made by the body or in a laboratory to improve or restore immune system function with the final goal of stopping the growth of tumors. Dr. Llosa is studying the immune microenvironment of metastatic osteosarcomas to assess their potential for checkpoint blockade (where immune responses are allowed through an checkpoint in malignant cells in order to fight the cancer) as a therapeutic option.

This grant is made with generous support from the Ethan Jostad Foundation, established by Kim and Chris Jostad in 2011 in memory of their son, Ethan, who was taken by Alveolar Rhabdomyosarcoma at the age of nine. In addition to funding cutting-edge pediatric cancer research, the foundation's mission is to provide emotional and financial support to children and families impacted by the disease.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Initiative. For a description of this project, see the consortium grant made to the lead institution: University of California Santa Cruz, Santa Cruz, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Initiative. For a description of this project, see the consortium grant made to the lead institution: University of California Santa Cruz, Santa Cruz, CA.

Neuroblastoma is a common type of childhood cancer that arises from nerve-like cells and causes tumors just outside the brain. Dr. George is studying tumor cells from patients to discover new molecular targets for treatment of neuroblastoma. Dr. George and his team are growing tumor cells from patients and examining their electrical properties in order to identify specific proteins that carry electrical current that may be new targets for treating neuroblastoma using existing non-cancer drugs.