Grants Search Results

Brain tumors are the leading cause of cancer-related deaths in children, and ependymomas are the third most common kind. Recent studies have shown that educating the patients own immune system to fight cancers immunotherapy can be safe and effective. Dr. Kohanbash's team has identified three peptides that might activate immune cells to specifically fight one of the more lethal types of ependymoma. Dr. Kohanbash is testing these peptides in the lab. He is also looking at how immunotherapy could help fight all six types of ependymoma that affect kids, and thus is studying relevant characteristics in the largest-ever series of pediatric ependymoma tumors as well as in ependymoma patients already participating in a clinical trial of a vaccine based on another peptide. A portion of this grant is generously co-supported by the Henry Cermak Fund for Pediatric Cancer Research and the Team Campbell Foundation. The Henry Cermak Fund for Pediatric Cancer Research, a St. Baldrick's Hero Fund was created in memory of a brave boy who had an amazing spirit throughout his battle with a brain tumor. This fund is dedicated to Henry’s wish that “no one gets left out.” The Team Campbell Foundation, a St. Baldrick's partner, was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine for five years. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities. A portion of Dr. Kohanbash's grant was also generously supported by the Henry Cermak Fund for Pediatric Cancer Research.

Based on progress to date, Dr. Ladle was awarded a new grant in 2020 to fund an additional year of this Scholar grant. As the Aiden's Army Fund St. Baldrick's Scholar, Dr. Ladle is using the body's own immune system to destroy cancer - specifically a class of cancer in children originating from connective tissues called sarcomas. Using fire as an analogy, Dr. Ladle seeks to build an intense flame of a powerful immune response which will specifically kill the cancer cells. To create this fire, one must follow specific steps. The kindling, which must be easily burned, is protein targets on the cancer cells (termed tumor antigens) recognized by the immune system. Next, the spark to ignite the kindling is initial inflammation in the tumor against these tumor antigens. Finally, to feed the fire, fuel or lighter fluid can be added in the form of recently approved immune modulator drugs which, when infused into patients, bind to immune cells residing in the tumor and activates them to kill the tumor cells. Each ordered step is essential in building an effective fire. This project addresses each of these key aspects for generating a successful immune response to treat sarcomas – creating new tumor antigens, adding inflammation to jump start the immune response against these antigens, and combining with new immune modulators allowing the immune cells to be active in destroying sarcomas. This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. When he was 8 years old, Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma. He had a huge tumor in his pelvis and the cancer had metastasized to his lungs. But this bright, funny and courageous boy believed he got cancer so he could grow up to find a cure for it. Aiden’s story has inspired so many people and his vision to cure cancer is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Rhabdomyosarcoma is a pediatric cancer of the developing skeletal muscle. The mechanisms that drive this tumor are poorly understood. From Dr. McEvoy's preliminary analysis, one possible mechanism is epigenetic deregulation of a group of long noncoding RNAs (lncRNA). This is exciting because lncRNAs play a role in tumorigenesis in other cancer types, including a subset of pediatric tumors. This presents a unique opportunity to develop novel therapeutic approaches for children with rhabdomyosarcoma. Dr. McEvoy's team hypothesizes that lncRNA deregulation is essential for rhabdomyosarcoma development. This study is working to understand the underlying mechanisms that drive this disease and identify potential new therapies. These results will have tremendous impact on patients, especially those with metastatic disease since only 20-40% will survive using current treatments.

The EWSR1-FLI1 family of cancer genes causes Ewing sarcoma. However, no drugs currently exist that specifically block the action of EWSR1-FLI1 to cause cancer cells to grow. The McFadden Lab has engineered a "self-destruct button" into the EWSR1-FLI1 gene in Ewing sarcoma cells cultured in the laboratory, and these cells stop growing when the EWSR1-FLI1 gene is turned off. Dr. McFadden is using this laboratory tool to identify proteins that work with EWSR1-FLI1, and identify other genes it controls to cause Ewing sarcoma cells to grow. These studies will help identify new ways to stop the growth of Ewing sarcoma cells.

Women who are diagnosed with cancer before 40 are often concerned about whether they will be able to have children in the future. Women can freeze their eggs or embryos (called fertility preservation) to help protect their fertility, but these services may not be widely available. Dr. Nichols has looked at how often women in North Carolina have children after cancer treatment and whether the health of their babies is different from women without cancer. She is further examining the use of fertility preservation after diagnosis and its association with birth rates and outcomes. This research will provide information to improve the long-term health of AYAs with cancer.

The immune system not only fights infection, but can also fight cancer cells. Recently, doctors have been able to use patients' own immune cells to help treat their cancer. These immune cells can also attack the patient's normal tissues, which is harmful. Dr. Richman is working to learn how normal tissues might be protected while still allowing the immune cells to effectively kill the cancer cells.

Based on progress to date, Dr. Tarlock was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Acute myeloid leukemia (AML) is a cancer of white blood cells and almost half of children diagnosed with AML will not be cured, even with very intensive chemotherapy and in some cases bone marrow transplant. Many of the mutations in the leukemia that contribute to development of the cancer have been identified, but cannot be used for therapeutic benefit, especially in children. Dr. Tarlock and colleagues have performed genomic testing on the cells of many children diagnosed with AML and found that approximately 40% of children abnormally express the protein mesothelin on their leukemia cells. Dr. Tarlock and colleagues will develop a phase I clinical trial to test a new therapy strategy that uses principles of the immune system to deliver chemotherapy only to mesothelin-positive leukemia cells. They will develop a clinical assay for mesothelin detection in AML to identify children who will benefit from mesothelin-targeted therapy, and investigate methods to optimize disease response to mesothelin-targeting immune therapies. This grant is generously supported by Rhys’ Pieces of the Cure, a Hero Fund created to honor Rhys Goldman and his journey with cancer. He was diagnosed with pre-B acute lymphoblastic leukemia just 2 weeks before his 6th birthday and endured treatment for three years. Rhys missed a lot of school and life during those years but since marking the end of treatment in July 2018, he has been enjoying swimming, singing in a boys’ choir, chess tournaments, playing with his dogs and going to school. Rhys’ Pieces for the Cure was created to ensure more research is funded for the treatment of pediatric cancer that is specifically focused on less toxic cures for kids.

Based on progress to date, Dr. Parameswaran was awarded new grants in 2019 and 2020 to fund additional years of this Scholar grant. Acute Myeloid Leukemia (AML) is the second most common acute leukemia in children, and current treatment strategies are inadequate to cure AML. Dr. Parameswaran is developing a new strategy using Natural Killer cells, which are a type of white blood cells with potential to kill cancer cells. Cancer cells often produce a protein that makes Natural Killer cells less active, which helps the cancer cells escape from NK cell-mediated killing. Dr. Parameswaran and her team are developing methods to stop this NK cell inactivation and thereby improve NK cell function to treat pediatric AML. This grant is generously supported by Rays of Hope, a St. Baldrick's Hero Fund created in memory of Rayanna Marrero. She was a happy 3 year old when she was diagnosed with Acute Lymphoblastic Leukemia. She battled ALL and won but a treatment induced secondary cancer claimed her life at age eight. Rayanna had an amazing attitude and loved life. She, like so many kids facing childhood cancer, did not allow it to define who she was. This Hero Fund aspires to give hope to kids fighting cancer through research.

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.

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.

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.

Children continue to die from leukemia because malignant cells overcome chemotherapy by turning off normal genetic safety checkpoints. Dr. Nalepa has found that loss of the same checkpoints makes cancer growth dependent on other genes, which represent possible anti-cancer targets. Dr. Nalepa is investigating the weak points of the cancer cells, and testing these precision-medicine strategies in new models of childhood leukemia. Dr. Nalepa will use the results of this testing to generate personalized therapy driven by cancer-specific mutations that will be more effective and less toxic for children who suffer from leukemia.

The human immune system is made up of a complicated network of cells including cells that help fight diseases such as cancer, and cells that prevent the immune system from fighting disease. Key cells that stop immune fighter cells from destroying cancer are called T regulatory cells (Tregs). Dr. Schultz is studying a new way to stop these Tregs and allow the good fighter cells to resume their ability to destroy cancer cells. This therapy will allow immune cells to put up a stronger fight against cancer and lead patients with cancer closer to cure.

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.

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.

Based on progress to date, Dr. Roberts was awarded a new grant in 2019 to fund an additional year of this Scholar grant. Dr. Roberts is focused on developing new treatments for patients with metastatic bone tumors. These tumors, once they have spread to the lungs, are incredibly difficult to treat. Dr. Roberts and his team will study the pathways that proteins move between osteosarcoma cancer cells and lung tissues. These experiments are helping Dr. Roberts and his team to better understand the biology that lets those tumors grow in the lung and will evaluate treatments which might prevent metastases from growing, and make them treatable when they do.

Based on progress to date, Dr. Brinkman was awarded a new grant in 2018 to fund an additional year of this Scholar grant. Survivors of childhood brain tumors are at-risk for problems with developing social relationships. These problems can lead to feelings of isolation, loneliness and social anxiety. Dr. Brinkman believes this is due to changes in a survivor's brain due to their cancer and its treatment. Dr. Brinkman's research aims to examine how the structure and function of the brain is related to survivors’ understanding of social information and their social behavior. These results aim to help with the identification of treatments for social problems. This new award is generously supported by the Team Campbell Foundation, established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine for five years. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

Based on progress to date, Dr. Chertok was awarded a new grant in 2018 to fund an additional year of this Scholar grant. The immune system efficiently fights infections in the normal brain, but malfunctions in brain tumors. Dr. Chertok hopes to empower the immune system to fight tumors by delivering agents that alter immune system activities. To do this, Dr. Chertok's research aims to develop tiny devices the size of blood cells that carry agents in the blood stream and release them with pre-designed timing after localized activation from outside the body. These devices will be used to modify how the immune system responds to brain tumors. This approach could lead to safe and effective therapies for children with brain cancer. A portion of this grant is generously supported by Griffin's Guardians, a St. Baldrick's partner. Griffin's Guardians was created by the Engles in memory of their son, Griffin. Their mission is to provide support and financial assistance to children battling cancer in Central New York, raise awareness about pediatric cancer and provide funding for research.

Based on progress to date, Dr. Kalish was awarded a new grant in 2018 to fund an additional year of this Scholar grant. Beckwith-Wiedemann syndrome (BWS) is a genetic disorder that affects thousands of children. Up to 25% of children with BWS develop cancer, most commonly liver and kidney cancer. The same genetic changes that cause BWS also cause other types of cancer, however we do not currently understand how. Dr. Kalish's research is studying how genetic changes that cause BWS lead to cancer using a large group of BWS patients and models. The goal of this research is to use the knowledge gained from these models to improve care for patients with BWS and related cancers.

Based on progress to date, Dr. Knoechel was awarded new grants in 2018 and 2019 to fund additional years and named the Emily Beazley's Kures for Kids St. Baldrick's Scholar. T-cell acute lymphoblastic leukemia (T-ALL) is a cancer of childhood and young adults that originates from the white blood cells in the bone marrow. It is a disease for which the initial treatment is often unsuccessful and the disease comes back quickly. Dr. Knoechel is investigating the epigenetic state changes that are responsible for drug resistance in T-ALL to develop new treatment strategies for patients with relapsed leukemia. 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 through the Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund.