Grants Search Results

Ewing sarcomas are bone cancers that impact many adolescents and young adults. Despite the use of intensive traditional chemotherapy combined with advanced surgical techniques, 30-40% of patients still die after the disease eventually spreads to other organs, such as the lungs and bone marrow. Dr. Hayashi's team believes the key to overcoming this problem lies in the identification of “Circulating Tumor Cells (CTC)”. These are cells that break away from the original tumor and travel through the blood stream, eventually taking root in another organ to form what is called metastatic disease, meaning the cancer has spread from where it started into different areas of the body. These cells undergo multiple changes in order to leave the original tumor and survive in the harsh environment of the blood stream, eventually leaving the blood stream to invade another organ where they multiply and grow. This project aims to dissect each of these complicated steps with the goal of unveiling which element of this devastating process can be targeted to disrupt it.

There is a new and effective cancer treatment for some incurable pediatric blood cancers. The treatment involves programing a patient's own cells to destroy their tumor, a process called cellular immunotherapy. Despite great effort to use cellular immunotherapy to treat 'solid' tumors, which include tumors of the bones, muscles and other parts of the body, we have not been successful yet. One major reason is that the programmed patient cells are designed to recognize a single marker on a cancer cell. In some blood cancers, all of the tumor cells express the same marker increasing the likelihood that cellular immunotherapy can cure the patient. Solid tumors are more heterogeneous than blood cancers, meaning each solid tumor cell may express a different marker. Therefore, cellular immunotherapy is less likely to destroy all solid tumor cells and the chances of achieving a cure is much more difficult. A potential solution is to trigger the body's own immune system to destroy tumor cells that express many different markers, a process called "epitope spreading". Named as the David's Warriors St. Baldrick's Scholar, Dr. Leibowitz focuses his project on testing strategies to augment epitope spreading in pediatric solid tumors so that cellular immunotherapy may become an effective and viable treatment option in the future.

This grant is named for and generously supported by the David’s Warriors Hero Fund created in memory of David Heard who battled neuroblastoma and inspired his family and countless others to commit to raising money for research to fight pediatric cancer. This fund honors the amazing spirit in which he lived, embracing life until the very end.

Based on progress to date, Dr. Osborn was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Abnormal growth of B-cells can result in leukemia, and a cutting-edge treatment option is immunotherapy with T-cells. T-cells can be engineered to express a chimeric antigen receptor (CAR) that is a 'seek and destroy' molecule for the CD19 protein on B-cells. CAR T-cells are the first FDA approved gene therapy and some stunning therapeutic responses have been observed. However, the T-cell activity can be so robust that they cause a massive cytokine storm that can be lethal. Furthermore, normal and cancerous B-cells express the CD19 protein targeted by the CAR, so normal B-cell loss occurs resulting in an impaired immune system. These side effects represent a significant hurdle in the safe and effective treatment of B-cell leukemia. To address this, Dr. Osborn, will express the CAR in a specialized subset of cells called T-regulatory (Treg) cells. Tregs have the same potent killing ability as T-cells but accomplish it without healthy tissue collateral damage. Additionally, he will engineer functional B-cells that are invisible to the CAR. This will allow for normal B-cell numbers and an intact immune system. Dr. Osborn will conduct these studies that are structured to resolve an unmet need, are highly novel, and are poised to make an immediate impact on childhood leukemia. 

The 2022 portion of this grant is named for the Rays of Hope Hero Fund which honors the memory of Rayanna Marrero. She was a happy 3-year-old when she was diagnosed with Acute Lymphoblastic Leukemia (ALL). She successfully battled ALL, 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.

A portion of this grant was funded by and named for the Mighty Mimi Hero Fund. Mimi Enyon was diagnosed with acute lymphoblastic leukemia at the age of 3. Her courage in the fight was unparalleled and she became “Mighty Mimi” to all those she inspired on her way to remission. This fund was established to share Mimi’s cancer journey in an effort to raise awareness and funding for childhood cancer research for kids like her.

A portion of this grant is generously supported by the Stanley Kuzmickas Feeney Fund for Pediatric Cancer Research. It was Christmas 2015 when Stanley was diagnosed with acute lymphoblastic leukemia at 13 months old. He courageously endured treatments for 3½ years. Today, he is in remission and eagerly started school in fall 2019. In his honor, Stanley’s family has organized a head-shaving event each year since July 2016 called “StoshyStrong." With the funds raised, the Feeney family created this Hero Fund to support research in new discoveries, genomics and other biological therapies for the treatment of ALL. Their goal is to one day see personalized treatments for every child.

Despite improvements in outcome for patients with acute lymphoblastic leukemia (ALL), up to 25% of children and 40% of adults fail frontline therapy and their prognosis is dismal, especially for high-risk and relapsed leukemia. Cure rates for ALL patients that are relapsing on therapy is approximately 20% and currently there are no targeted therapies. Dr. Tsirigos' goal is to address this significant clinical need by studying how DNA, our genetic material, is organized inside the nucleus of the cells, i.e. how it is folded in three-dimensional space. Over the past decade, seminal studies have demonstrated that DNA folding is of fundamental importance in that it allows the cell to properly perform its function and retain its identity (e.g. a liver cell versus a blood cell). Very recent studies have demonstrated that disruptions of DNA folding may cause various diseases, such as developmental defects and cancer. However, no study has addressed the question of disruptions of DNA folding on the genome-wide scale in cancer or how such disruptions may be exploited for more effective treatments. Dr. Tsirigos is attempting to answer two key questions. First, are cancer-promoting genes (“oncogenes) capable of disrupting normal DNA folding to transform normal cells into malignant ones? And, second, can drug treatment restore DNA folding and thereby also restore normal cell function?

This grant is made with generous support from the Rally for Ryan Fund. Ryan was diagnosed with high risk ALL when he was 7 years old. He endured 3½ years of treatments with a brave acceptance that this was his fight to win. He recently relapsed and is in the fight again. This fund honors Ryan’s perseverance and his commitment to make a difference for kids with cancer by shaving for St. Baldrick’s and raising funds for research.

Sarcomas are tumors of the bone and soft tissues that comprise up to 20% of cancer diagnoses in children. Despite dismal outcomes for patients with recurrent or metastatic disease, treatment regimens have remained largely unchanged for decades – intense non-specific chemotherapy combined with surgery or radiation. Dr. Tulpule studies Ewing’s sarcoma (ES), a bone tumor caused by a unique genetic change that creates a tumor-specific protein EWS-FLI1. To date, no drug has been identified to directly block the cancer causing EWS-FLI1 protein. His research takes a different approach to combating ES by asking a fundamental question: can we identify a targetable weakness in ES tumors that is caused by the EWS-FLI1 protein? Using a cutting-edge screening technology called CRISPR interference, Dr. Tulpule's team identified a specific vulnerability in ES cells’ capacity to repair damage to their DNA. Normal cells have many backup systems in place to repair DNA damage, but they have shown that EWS-FLI1 causes ES cells to become overly reliant on a single pathway, known as homologous recombination (HR) repair, such that blocking HR is an effective and specific way to kill ES. Dr. Tulpule is building a detailed understanding of why ES cells are so vulnerable to HR pathway blockade and then applying that knowledge towards developing less toxic and more effective treatments for ES patients.

Cancer cells rely on specific nutrients for growth and survival, rendering nutrient restriction as a potential therapeutic strategy. Along this line, acute lymphoblastic leukemia (ALL) cells have been found to be dependent on exogenous supply of asparagine, a nonessential amino acid, for protein synthesis. As a result, depletion of asparagine in the blood stream by L-asparaginase, a chemo-agent, has been successfully used to treat pediatric ALL for 40 years. However, ALL patients can develop resistance to the continuous application of this chemo-agent. Dr. Zhang is determining how ALL cells become resistant to L-asparaginase treatment, and therefore to provide experimental evidence of novel therapeutic targets that can potentially improve the outcome in pediatric ALL patients.

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.

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.

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.

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 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.

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.

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. 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.

Based on progress to date, Dr. Gowda was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Children with high risk B-cell leukemia, especially with loss or dysfunction of IKZF1 gene have very poor outcomes and high relapse rate. Every other child who relapses with high risk leukemia dies from the disease and there has not been much advancement in treatment for this group for the last 30 years. Dr. Gowda and team have found that a cancer promoting protein called casein kinase II (CK2) impairs the important functions of a protein that helps prevent leukemia. Inhibiting the CK2 protein will restore the ability of this protein to function properly and prevent leukemia. Dr. Gowda's team is testing if using a drug that inhibits CK2 protein along with the drugs that already are known to work in leukemia will have stronger anti-leukemia effect and improve the outcome. Using two agents that target same gene or pathway via different mechanisms will ensure effective shutdown of the particular pathway resulting in strong therapeutic effect. This strategy would also help lower the doses of each drug used and reduce their side effects and associated toxicity.

Based on progress to date, Dr. Cimmino was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Vitamin C is essential for maintaining healthy hair, skin, immune system and heart function. In addition to these health benefits, Dr. Cimmino and team propose that vitamin C might be a non-toxic therapeutic for the treatment of patients with pediatric acute myeloid leukemia. Recently, it was discovered that vitamin C enhances the activation of a group of enzymes called TET proteins that are required for normal blood development. A significant fraction of children and young adolescents with acute myeloid leukemia have mutations in TET2, causing impaired TET2 activity and a block in normal blood cell formation. However, only one of the two copies of the TET2 gene is defective in these patients. Dr. Cimmino's team is working to determine if treatment with high-dose vitamin C could enhance the activity of the remaining, non-mutant, TET2 protein, kill leukemia cells and restore normal blood development. Alternative therapies such as treatment with vitamin C might provide a safe and effective strategy to improve outcome for pediatric leukemia patients. Awarded at the New York University School of Medicine, and transferred to University of Miami.

Based on progress to date, Dr. Agarwal was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-risk neuroblastoma is an aggressive cancer of very young children with less than 50% overall survival. Current therapy includes high-dose chemotherapy and radiation, which has long-term toxic side-effects. Despite these intensive therapies, neuroblastoma commonly relapse. This relapse is the primary cause of death from neuroblastoma due to disease spread, drug-resistance, and toxicity. As the Oliver Wells Fund for Neuroblatoma St. Baldrick's Scholar, Dr. Agarwal is focusing his research on developing effective therapeutic approaches to target those tumor cells which escape initial treatment and regenerate drug-resistant disease. Recently, Dr. Agarwal's team discovered a chemotherapy-resistant, highly tumorigenic sub-population of cells in neuroblastoma tumors. These cells escape initial therapy and may cause aggressive, drug-resistant relapsed disease. Furthermore, they found that specific epigenetic enzymes maintain this cell sub-population by activating key genes. These epigenetic modifiers can be successfully targeted with novel epigenetic inhibitors, currently under pre-clinical trials. These exciting findings suggest a new epigenetic therapeutic approach for high-risk neuroblastoma. This grant supports efforts to uncover the mechanisms controlling neuroblastoma tumorigenicity and relapse, and develop an effective targeted approach for high-risk neuroblastoma.

A portion of this grant is funded by and named for the Oliver Wells Fund for Neuroblastoma, a St. Baldrick's Hero Fund. From the moment he was born, Ollie was the center of the Wells family with a contagious smile and a sparkle in his eyes. As the youngest child, it was devastating when they learned the 15 year old toddler had cancer. Oliver was diagnosed with high risk neuroblastoma and spent the next 13 months bravely enduring chemotherapy and radiation, more than a dozen surgeries and a bone marrow transplant. But Ollie persevered and smiled through it all. It was an unfair fight from the beginning and in July 2018, Ollie passed away. The Oliver Wells Fund for Neuroblastoma was established in his memory to raise funds to find cures and give hope to other kids facing the same fight. In this way, the Wells family intends to share Oliver’s joy for life and use his story to help find a cure. 

A portion of this grant was also funded by and named for David's Warriors, a St. Baldrick's Hero Fund. The fund was created in memory of David Heard who battled neuroblastoma until his passing at the age of ten. David inspired his family and countless others to commit to raising money for research to fight pediatric cancer through the St. Baldrick’s Foundation. The Fund honors the amazing spirit with which he lived, embracing life until the very end.

Awarded at the Baylor College of Medicine, and transferred to St. John's University.

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. 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.