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Do you ever get a cold sore on your lip, or know someone who does? That sore is caused by a virus that destroys the cells in your lip. As the virus spreads, the sore gets bigger. Viruses are great at killing cells and spreading. But, the sore eventually goes away because the immune system attacks the infected cells, killing them and stopping the viral infection, allowing your lip to heal. Imagine if we could get both the virus and the immune system to kill cancer cells instead of lip cells! Previously Dr. Haworth's team used a safe version of the cold sore virus to infect a common type of hard-to-treat childhood cancer cells. The virus directly killed cancer cells and caused the immune system to attack the cancer cells that the virus missed. Dr. Haworth's team is testing ways to make the virus and immune system work better together. Dr. Haworth is infecting model tumors with the virus, and giving immune cells designed to attack the tumor, hypothesizing that giving both virus and immune cells will cure the tumor. Awarded at The Research Institute at Nationwide and transferred to St. Jude Children's Research Hospital.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

Only 45% of children with high-risk neuroblastoma (NB) are cured. The New Approaches to Neuroblastoma Therapy (NANT) consortium links laboratory and clinical investigators to develop therapies with high potential for improving survival and performs the first testing of these treatments at 14 neuroblastoma centers in North America and 3 in Australia, United Kingdom, and France. NANT studies aim 1) to enhance the ability of the immune system to eliminate NB by targeting both NB cells and surrounding normal cells that help tumor cells grow and resist treatments; 2) to improve treatment of NB using small molecule drugs that target NB gene abnormalities that “drive” tumor aggressiveness including abnormal/mutated ALK protein or abnormally increased MYCN protein; and 3) to use molecular biology to define new treatment targets in NB and normal cells that enhance NB growth and resistance to therapy to improve prediction of outcome with a “biomarker” test for NB cells in blood and bone marrow. The NANT consortium anticipates that these innovative studies will improve survival for children with high-risk neuroblastoma.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

Based on progress to date, Dr. Green was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-grade gliomas (HGG) are aggressive brain cancers that affect both adults and children. Current treatment options are very limited, and the vast majority of patients die of their tumors within five years of diagnosis. One subtype of high-grade glioma that almost exclusively occurs in children, diffuse intrinsic pontine glioma (DIPG), is the last incurable childhood cancer, with zero percent long-term survivors. As the Luke's Army Pediatric Cancer Research Fund St. Baldrick's Scholar, Dr. Green and his team intend to address these tumors by focusing on a new field of cancer treatment called epigenetics, which literally means "above genetics" and refers to all changes to DNA that do not involve changes to the DNA sequence itself, but instead affect which genes are made into protein. Through prior work, Dr. Green's team has found a gene, BPTF, which controls the expression of many other genes and appears to drive HGG and DIPG growth. Dr. Green aims to determine how exactly BPTF drives growth by interacting with other genes, to measure how BPTF inhibition works with drugs called HDAC inhibitors and whether this strategy could work with current standard treatments, and to measure the effect of a new chemical that inhibits BPTF that could serve as a precursor to medicines targeting BPTF.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

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. 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 and 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!

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. 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.