Grants Search Results

This grant funds a student to complete work in pediatric oncology research for the summer. Dr. Satake and colleagues are studying a rare and aggressive childhood kidney cancer called malignant rhabdoid tumor of the kidney (RTK). Children with RTK have extremely poor outcomes (survival rate 25%) despite lots of different treatments since tumors continue to grow even with treatment and tend to relapse. They believe that the investigational drug called OTS964 may be a new potential treatment. They also believe that OTS964 may be even more effective when used with navitoclax, a drug which has a different mechanism of killing cancer cells. In this project They plan to test the new treatment using these drugs in a human RTK mouse model, and to study the mechanism of actions, with the goal of finding a new treatment for RTK patients. This work is being completed under the mentorship of Dr. Noriko Satake.

This grant funds a student to complete work in pediatric oncology research for the summer. The Kohanbash lab is investigating how B cells, a critical part of the immune system, function in aggressive brain tumors in children. B cells produce antibodies to fight infections or cancer and carry unique surface markers called B cell receptors, which help them recognize and respond to specific threats. However, in cancer, B cells may behave unpredictably, sometimes helping the body fight against tumors, but in other cases, supporting tumor growth. By examining the specific features of B cell receptors in pediatric high-grade gliomas, they aim to better understand how these immune cells influence tumor development and progression. The goal of this project is to learn more about how the immune system responds to brain tumors in children and how altering these responses could lead to more effective treatments. Ultimately, this research hopes to improve therapies and outcomes for children diagnosed with deadly brain tumors, offering them better chances for recovery and long-term well-being. This work is being completed under the mentorship of Dr. Gary Kohanbash.

This grant funds a student to complete work in pediatric oncology research for the summer. Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue sarcoma in the United States, with approximately 400 new cases annually. Outcomes for high-risk RMS remain dismal, with three-year event-free survival rates as low as 20-30%. There is an urgent need for innovative therapeutic strategies that are more precise, have less toxicity and have significantly improved efficacy and survival benefits. A subset of RMS tumors have mutations in the RAS gene family, therefore, exploiting these mutations as therapeutic targets is an attractive and targeted therapeutic strategy. Dr. Ladanyi's research aims to test a recently developed RAS inhibitor (RMC-6236) in preclinical patient-derived disease models harboring mutations in RAS. This agent is in clinical trials for adult cancers with some RAS mutations. The St. Baldrick's Foundation Summer Fellow will help to generate the preclinical data necessary for a Phase 1 clinical trial testing RMC-6236 in children with RAS-driven RMS. This work is being completed under the mentorship of Dr. Marc Ladanyi.

This grant funds a student to complete work in pediatric oncology research for the summer. Children diagnosed with diffuse midline glioma (DMG), an aggressive brain tumor, face limited treatment options. A new drug called ONC201 has decreased tumor growth in some patients but not others. This research aims to understand why this difference in response occurs. Researchers have discovered that ONC201 affects how cells modify their genetic instructions (RNA), specifically through a process called m6A modification, which affects thousands of genes. When treating tumor cells with ONC201, this group observed a significant decrease in these modifications. The St. Baldrick's Foundation Summer Fellow plans to help study whether this change happens in all tumor cells or only in those that do not respond well to the drug. By analyzing these samples, they hope to identify markers that could predict which patients will benefit most from this treatment. This knowledge could lead to more effective ways to use ONC201 and help develop better treatments for children with DMG. This work is being completed under the mentorship of Dr. Jessica Foster.

This grant funds a student to complete work in pediatric oncology research for the summer. Infant acute lymphoblastic leukemia (ALL) is a fast-growing blood cancer often caused by changes in the KMT2A gene, which helps leukemia cells survive and spread. This gene creates harmful fusion proteins that work with a partner protein called menin to keep the cancer growing. New drugs like Revumenib block menin and have shown promise in adults, and a clinical trial is testing whether they can help infants whose leukemia has returned or resisted treatment. To better understand how the drug works, Dr. Ernst and her team are developing a novel B-cell acute leukemia model to study the disease more closely. This model allows them to compare what happens when menin is completely removed versus when it is only blocked by the drug. By studying these cancer cells using advanced gene analysis, they hope to find differences that explain why some cases resist treatment. This research could help doctors use menin-blocking drugs more effectively for infants with aggressive leukemia. This work is being completed under the mentorship of Dr. Patricia Ernst.

This grant funds a student to complete work in pediatric oncology research for the summer. Children with aggressive neuroblastoma tumors have poor cure rates despite intensive treatment, and new therapies are needed. Kinases are proteins that control signals in cancer cells leading to cancer cell growth and spread, and we have developed a new drug, getretinib, that inhibits the RET kinase that is important for neuroblastoma tumor growth. This project will test getretinib to determine its effectiveness against neuroblastoma cells and tumors, and evaluate cells before and after treatment with getretinib to determine how getretinib kills neuroblastoma cells and to identify specific genes and proteins that are important for neuroblastoma cell responses and resistance. The results of these studies will determine whether and why getretinib is effective against neuroblastoma, leading to clinical trials using new drugs directed against RET for treatment of children with neuroblastoma. This work is being completed under the mentorship of Dr. Pete Zage.

This grant funds a student to complete work in pediatric oncology research for the summer. Childhood brain tumors like Diffuse Intrinsic Pontine Glioma (DIPG) are difficult to treat, especially when they have mutations in a gene called TP53. These mutations make cancer cells resistant to radiation, which is the main treatment for DIPG. With support from the St. Baldrick's Foundation, the summer student researcher is working with Dr. Blackburn's team to study whether existing drugs can help radiation work better in TP53-mutant tumors. Using zebrafish, they have identified several promising drugs and will now investigate how they make cancer cells more vulnerable to treatment. This research could help lead to better therapies for children with DIPG. This work is being completed under the mentorship of Dr. Jessica Blackburn.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The St. Baldrick's Foundation Summer Fellow in the Goldsmith Laboratory at Emory University is working to develop new cell-based immunotherapies for neuroblastoma. They work alongside a team of researchers seeking to apply the unique properties of gamma delta T cells as an effective, off-the-shelf adoptive cell therapy for children with neuroblastoma. During the summer fellowship with St. Baldrick's Foundation, the student will investigate the expansion and anti-neuroblastoma activity of a specific type of gamma delta T cell called the VD1 subset. This work is being completed under the mentorship of Dr. Kelly Goldsmith.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Osteosarcoma is a rare and aggressive bone cancer that affects mostly children and older adults. For decades, effective therapies to treat this disease have been lacking, including novel immunotherapy treatments used for other cancers. To understand this failure, it is necessary to study the interaction between the immune system and the tumor itself. As such, the St. Baldrick's Foundation Summer Fellow has developed a tool to measure the activity of the immune system as the tumor progresses. In this system, cancer cells targeted by the immune system will turn green when visualized under a fluorescent microscopic. This tool will help us understand the behavior of effector T lymphocytes, a key cell population in the human body responsible to kill cancer cells and other foreign cells. As such, this summer project will help us understand unique characteristics of this tumor and accelerate the cause for more effective therapies. This work is being completed under the mentorship of Dr. Tyler Jacks.

This grant funds a student to complete work in pediatric oncology research for the summer. Neuroblastoma, a pediatric solid tumor, has a poor prognosis in cases with MYCN proto-oncogene amplification requiring novel therapeutic strategies. Although immune checkpoint inhibitors have shown dramatic effects in adult cancers, their efficacy in neuroblastoma is limited due to its immunosuppressive tumor microenvironment (TME). To address this, the Weiss lab developed a novel neuroblastoma model (Mycn-nGEMM) to study immuno-oncology. They identified that macrophage migration inhibitory factor (Mif), a cytokine with pro-tumorigenic properties, is highly expressed in both human and mouse neuroblastoma tumors. Furthermore, Mif inhibition was found to reduce immunosuppressive tumor-associated macrophages (TAMs),, suggesting that targeting Mif could enhance anti-tumor immunity in neuroblastoma. The St. Baldrick's Foundation Summer Fellow will investigate Mif's role in TAMs and the TME using genetic and pharmacological approaches to reveal the immunosuppressive mechanisms of neuroblastoma. This work is being completed under the mentorship of Dr. William Weiss.

This grant funds a student to complete work in pediatric oncology research for the summer. Ewing Sarcoma (ES) is a type of cancer that is usually found in the bones of children, teens, and young adults yet tends to spread to other areas of the body, making it difficult to target and treat. Understanding why this type of cancer develops and why it travels to different areas of the body is crucial in being able to develop new targeted treatments that work more effectively with fewer side effects than standard treatments like chemotherapy, surgery, and radiation. In ES, a specific protein called EWS::FLI1 is not found in normal cells. This protein does not work correctly like normal proteins in normal cells and causes the ES cells to divide and grow uncontrollably, creating tumors. If the broken protein in ES cells could be turned off with a new medication, it would stop the ES cells from growing into tumors and spreading in the body, stopping the disease. Ideally, the medication would only work in ES cells but so the patient would not experience side effects from the medicine. This work is being completed under the mentorship of Dr. Jeffrey Toretsky.

This grant funds a student to complete work in pediatric oncology research for the summer. Neuroblastoma is a pediatric cancer that develops in immature nerve cells and accounts for 15% of cancer-associated deaths among children. The Feng laboratory has recently reported that MYCN-driven cancers secrete a signaling molecule to hijack the body's immune system to promote tumor development and aggressiveness. This proposal seeks to characterize whether this signaling molecule explores macrophages, a type immune cells, to foster the development of MYCN-driven neuroblastoma. Successful completion of this research will help researchers understand the crosstalk between the tumor and immune cells and may identify new therapeutic strategy to treat high-risk neuroblastoma and other MYCN-driven childhood cancers such as medulloblastoma and glioma. This work is being completed under the mentorship of Dr. Hui Feng.

This grant funds a student to complete work in pediatric oncology research for the summer. Pediatric sarcomas are devastating childhood cancers. New therapies that have fewer side effects but can more effectively kill cancer cells are urgently needed. There are multiple factors that make pediatric sarcomas hard to treat. One of those reasons is that not all pediatric sarcoma cells are like each other. Some grow very fast, whereas some others grow more slowly but can migrate and form metastases. Dr. Langdon's laboratory is working on developing combination therapies that can limit the impact of all the different types of pediatric sarcoma cells. During the next summer, the St. Baldrick's Foundation Summer Fellow's project will be to discover reasons why these combinations work so well against pediatric sarcoma cells. They will also learn several new techniques and gain experience in mentoring and more effective communication strategies. This work is being completed under the mentorship of Dr. Casey Langdon.

Children everywhere in the world get cancer but their chances of surviving differ based on where they live. Disparities in childhood cancer diagnoses and survival have been described by sex and age, but there are gaps in this literature from countries with limited resources. The first goal of Dr. Force's project is to analyze how childhood cancer diagnoses and survival differ by sex, age, and world region, using data from the most comprehensive international collection of hospital cancer registries, and to assess potential underlying drivers of these disparities, which would be beneficial in identifying interventions to improve equity in childhood cancer outcomes. The second goal of Dr. Force's project is to compare childhood cancer data from hospitals and population-based cancer registries, to determine whether hospital data could be used to supplement information on childhood cancer burden where data is currently lacking in global models, better illuminating the disparities that exist globally.

Hepatoblastoma is the most common liver tumor diagnosed in early childhood, and new therapies are urgently needed to improve survival and reduce treatment related morbidity. Immunotherapy is a type of cancer treatment that harnesses the body's own immune system to target and attack cancer cells. While some immunotherapies have been very successful against certain tumor types in adult patients, they have been largely unsuccessful in treating pediatric tumors. This demonstrates how little we know about how the pediatric immune system responds to tumors. Using samples and models of hepatoblastoma, Dr. Cabric's research aims to identify the key immune cells involved in recognizing and responding to hepatoblastoma. Identifying the key immune cells involved in tumor immunity, and mechanisms that allow tumors to escape detection and deletion by the immune system, will allow us to find novel targets for future immunotherapies that work in children.

This grant is funded by Allied World, a global provider of insurance and reinsurance solutions.

Cancer patients take life-saving drugs that, unfortunately, can result in peripheral nerve damage. For example, many patients receiving cisplatin experience permanent hearing loss. There is one therapy that has been approved to mitigate cisplatin-induced hearing loss, however, the reduction in hearing loss is modest (< 30%) and this mitigating treatment is associated with poorer overall survival rates due to inhibition of cisplatin's cancer-fighting properties. Thus, it is approved for low-risk pediatric patients only. To develop a better alternative, Dr. Rutherford and colleagues are testing novel compounds they have developed at Washington University, which have shown to protect the ear from noise trauma. With hearing tests and with anatomical measurements of the cochlea, Dr. Rutherford will attempt to prevent hearing loss following cisplatin treatment in models. After this innovative project proves successful, subsequent model studies will determine if Dr. Rutherford's therapy inhibits cisplatin's cancer-fighting role.

The study of genetic disease of cancer predisposition has served as a model for understanding cancer in general. Fanconi anemia is a rare genetic disease of failed blood production and cancer proneness, including leukemia and head and neck cancer. The genes and encoded proteins participate in DNA repair. However, an examination of cancer databases of DNA sequence shows that Fanconi genes are mutated in up to 30% of all head and neck cancers in non-Fanconi patients. Dr. Kupfer and colleagues have studied one particular mutation that resides in the Fanconi FANCD2 gene that interrupts its protein binding to another important gene BLM, which also participates in DNA repair. This proposal will seek to study the normal function of the FANCD2-BLM interaction in the cell and the consequences of its disruption. Dr. Kupfer also seeks to identify ways disruption of the normal pathway will render cancers vulnerable to molecular targeting to improve therapeutics.

Despite remarkable improvements in treatment for children with some types of cancer, pediatric brain tumors remain an area that desperately require more effective and low toxic therapy solutions. Dr. Jun Qi has formed a multi-disciplinary team to identify novel targets for pediatric brain tumors and develop new strategies to suppress the targets for patient treatment. Using a chemical strategy, Dr. Qi and his team aim to disrupt the functions of these targets to effectively inhibit brain tumor cell growth and block tumor progression in the models that resemble the real disease. The study focuses on improving on-target effect and, more importantly, on getting these potential drug candidates into the brain. The proposed study will translate from bench to bedside for patient care and result in a novel therapeutic strategy with significant improvements in survival and reduced morbidity for pediatric brain tumor patients to fulfill the mission of St. Baldrick's Foundation.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

Diffuse midline glioma (DMG), previously known as diffuse intrinsic pontine glioma (DIPG), is a deadly childhood tumor with no effective treatments. Dr. Li's project seeks to understand the genetic and epigenetic dysregulation of DMGs. Through cutting-edge single-cell analyses and advanced AI models, researchers aim to map the tumor's epigenetic landscape, identify key regulatory elements, and predict the function of risk mutations. This knowledge could pave the way for new targeted therapies and improve DMG outcomes.

This grant is funded by and named for #Joe Strong 71, a St. Baldrick’s Hero Fund created in memory of Joe Purdue. Joe was a talented football player and cherished friend and son. He was diagnosed with DIPG shortly after graduating from high school, cutting short his plans to attend college. He is remembered for determination as he battled the most lethal form of brain cancer. #Joe Strong 71 carries on Joe's legacy by funding research for DIPG.

Whilst it is well known that damage to our DNA can cause cancer, it is still not fully understood what causes such DNA damage in many childhood cancers. Dr. Wang and colleagues recently made a breakthrough by discovering that our own body produces a natural toxin called formaldehyde that causes DNA damage and an aggressive blood cancer in children. This was a shocking discovery as it had previously been thought that formaldehyde mainly came from industrial chemicals found in factories. Dr. Wang's overall aim in this research proposal is to unravel exactly where formaldehyde toxins are made in our body. This knowledge can help to identify children at risk of developing blood cancers, and to develop strategies to modulate the production of formaldehyde as novel therapies against blood cancers.

The first year of this grant is is generously supported by RowOn 4 A Cure, a St. Baldrick's Hero Fund. Rowan was a happy, spunky, funny, smart, and smiley little girl. With that same tenacity, she faced her cancer diagnosis of a rare form of acute myeloid leukemia when she was three. Despite intense chemotherapy and radiation and a successful cord blood transfusion, Rowan relapsed after a brief remission. The family relocated in search of another treatment option but before one could be found, Rowan sadly passed away. RowOn 4 A Cure was established to honor Rowan and continue her fight against AML by raising awareness and funds for research to find better options for treatment of relapsed AML and ultimately, a cure for the disease. Her family remembers Rowan’s perseverance during tough treatment days and intend to make an impact as they “Row On” to find a cure.