Grants Search Results

Diffuse midline glioma is a devastating disease with no known curative therapy. New treatments using immunotherapy approaches, like CAR T therapy, show promise; however, tumors often escape detection by hiding their target proteins and suppressing the immune response to therapy. Dr. Pomaville seeks to tackle this problem by manipulating RNA modifications in the tumor and body to improve response to CAR T therapy. She has found that drugs that mediate RNA methylation, the most common modification on protein-coding mRNA, increases protein levels of existing CAR T antigens and can induce cell death. This RNA modification mark also influences how the body's immune system responds to tumors. Dr. Pomaville will test this drug in cell-line and immunocompetent models to assess whether manipulation of RNA methylation leads to improved efficacy of CAR T therapy. Dr. Pomaville and team will develop combination strategies that enhance the response to immunotherapy in patients.

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

Dr. Kagami is developing LBSeq4Kids, a novel liquid biopsy test designed specifically for children with cancer. Traditional biopsies are invasive and only provide information from one point in time. In contrast, liquid biopsies use samples of body fluids - such as blood, spinal fluid, or fluid from the eye - to detect tumor DNA in real time. While this method is used in adult cancers, it is not widely available for children due to smaller sample volumes and the genetic variety of tumors. Dr. Kagami and colleagues are developing and clinically validating a three-part testing platform. LBSeq4Kids will help to better personalize treatment and monitor how a child's cancer responds to therapy. This project will also help determine which liquid biopsy tests and body fluids provide the most useful information for different cancers and stages of treatment. The goal is to improve diagnosis, guide more effective targeted therapies, and support better long-term outcomes for children with cancer.

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

In some children with cancer, their bodies become severely inflamed and they develop a condition known as hemophagocytic lymphohistiocytosis, or HLH, which is often deadly. It is not yet understood why some children with cancer develop HLH and others do not. Dr. Meyer is using advanced technologies to carefully study blood cells from children with cancer who develop HLH. She is also working to create the first model of this disease so that she can better study the underlying disease mechanisms. By increasing our understanding of HLH, she seeks to develop strategies that will help doctors quickly identify affected children and determine which medicines are most effective. Her overall goal is to help more children survive this devastating condition. If successful, her findings will apply not only to HLH, but also to other life-threatening immune system disorders resulting from cancer and/or its treatment.

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

Acute myeloid leukemia (AML) is an aggressive and molecularly heterogeneous cancer. Despite recent progress in developing new agents, front-line therapeutic protocols have not changed markedly over the past two decades and are associated with substantial morbidity and mortality. Accordingly, there is a need to develop novel mechanism-based treatments. Pediatric AML patients with NUP98 (nuclear pore complex protein 98) gene rearrangements (NUP98-r) have particularly dismal outcomes, with overall survival rates of 25-35%. Dr. Tian and colleagues are studying important proteins that contribute to a cell's ability to become cancerous in one of the most common types of NUP98-r AML: NUP98::KDM5A. Dr. Tian is also optimizing a compound's ability to directly inhibit KDM5A. Identification of these targets will allow for development of new drugs in the treatment of NUP98-r pediatric AML.

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

The survival rates for children with cancer remains unacceptable and the treatments for childhood cancers continue to carry significant life-altering side effects as most treatments do not specifically recognize cancer cells and affect healthy cells. Dr. Guerra and colleagues are developing a therapy created from the white blood cells of a patient's own immune system, known as immunotherapy. These cells are engineered to target cancer cells by engaging a marker that is present on the surface of cancer cells but not present on healthy cells. When the engineered white blood cells engage this cancer-specific marker, the cells become activated and attack the cancer cells while sparing healthy cells. Additionally, this immunotherapy can be boosted with further engineering approaches to remain active in a patient for long periods of time, allowing for long-term killing of cancer cells which we predict will improve survival while reducing treatment-related side effects.

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

Relapsed/refractory pediatric acute myeloid leukemia (AML) is a challenging disease with poor survival. Combining two classes of drugs--thalidomide analogs (TAs) and menin inhibitors- may improve outcomes for patients with a particular genetic subtype of AML. Dr. Li has shown that TAs can affect the numbers and activity of a type of immune cell called regulatory T cells (Treg), which can prevent the immune system from recognizing and attacking leukemia cells. Treating pediatric AML patients with TAs and menin inhibitor, which is being tested in an upcoming clinical trial, may impair Treg activity and improve immune recognition and targeting of AML, potentially contributing to responses. Dr. Li will study the effects of combination therapy on models. Dr. Li's study will improve the efficacy and safety of new pediatric AML therapies by defining their effects on immune responses.

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

Mixed Phenotype Acute Leukemia (MPAL) is a subtype of leukemia that shares features of the 2 most common types of leukemia: acute myeloblastic leukemia and acute lymphoblastic leukemia. Unfortunately, it is really hard to cure with no consensus treatment. When cells divide, chromosomes can break and the pieces can re-attach to the wrong place resulting in a chromosomal translocation. This new abnormal chromosome can result in the expression of a new gene and a new protein, called a "fusion protein". In MPAL, a common translocation involves the ZNF384 gene that can be fused to over 20 new genes, but the consequences are not well understood. Dr Libbrecht has identified that a novel drug that inhibits BRM/BRG1, essential proteins that maintain the DNA structure, and can kill MPAL cells in vitro. Her studies aim to better understand how BRM/BRG1 inhibition affects the ZNF384 fusion proteins and MPAL cells to validate it as novel therapy for MPAL.

Dr. Lerman is studying the connection between how aggressive childhood brain tumors called diffuse midline gliomas (DMGs) look on MRI scans and the DNA of the tumors themselves. Tumors that look different from each other on MRI scans and have different changes in their DNA grow in different ways. What is not known is how the appearance of the tumor on the MRI scan is related to the changes in the tumor's DNA. By studying this connection, Dr. Lerman hopes to predict how a tumor might grow based only on an MRI scan, which would help patients and families who either cannot or choose not to have a surgical procedure called a biopsy to test the tumor's DNA. Right now, there is no treatment that cures DMG and all patients are treated the same way: with radiation. Dr. Lerman plans to identify groups of tumors that behave similarly, which will help future clinical trials test the right medicine for the right patient.

Teens with leukemia go through tough treatments that make them feel tired and weak, so they spend a lot of time sitting and lying down, which can make side effects worse and put them at risk for chronic diseases like diabetes. Dr. Ivory is testing ReSeT, a program she developed for teenagers getting leukemia treatment to interrupt sitting time with short exercise breaks that will likely improve their lifestyles, heart health, and quality of life. Over 10 weeks, each teenager will use a Fitbit, health coaching, and an app-based support group to slowly increase their activity. After testing ReSeT in 30 teenagers to see if they can do it and what they think, she will fine-tune ReSeT and test it again in 10 more teenagers and compare how they do with 10 teenagers who didn't get the program to see if the program works. The goal is to use small behavior changes to help teenagers with cancer be more active during and after treatment to improve their lifelong health.

Acute myeloid leukemia (AML) is harder to cure than most other types of childhood leukemia and lymphoma. Treatments are toxic and require patients and their families to spend up to a year in the hospital. Childhood AML survivors often have serious side effects later in life from their treatment. We need new treatments for AML that are less toxic and more effective. AML is often caused by mutations in a protein called N-Ras that tell the leukemia to grow and divide much more quickly than healthy tissue. If we could shut down this abnormal N-Ras signaling, it would stop the leukemia from growing. Unfortunately, no approved drugs exist that target mutant N-Ras proteins. Dr. Decker and his colleagues are testing a new drug called ABD778 that selectively blocks the growth of AML cells with mutant N-Ras. The results of this research could move drugs like ABD778 closer to the clinic and pave the way for new treatments for childhood AML.

This grant is named for the Cody Thompson Memorial Hero Fund. Cody spent much of his childhood in the hospital. As an adult he embraced the health and happiness of children and was an avid St. Baldrick's volunteer. This grant is a tribute to the compassion he carried throughout his life.

Dr. Dionysiou and team are studying a small molecule naturally found in the body called miR-21. This molecule could make a treatment called allogeneic hematopoietic cell transplantation (allo-HCT), which cures children with aggressive leukemia, safer and more effective. This treatment uses immune cells from a donor, but it can cause a serious problem called graft-versus-host disease (GVHD), where the donor cells attack the patient's healthy tissues. The challenge is to stop this attack without weakening the donor cells' ability to fight the cancer. By understanding how miR-21 controls the immune response, Dr. Dionysiou hopes to find ways to prevent GVHD while allowing the donor cells to attack the cancer, making this life-saving treatment safer and more effective.

This grant is generously supported by 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.

In the past decade, new therapies that train the immune system to recognize and kill cancer cells have revolutionized cancer care. Unfortunately, cancers arising from connective tissue like bone have not responded to these immunotherapies. Despite almost four decades of trialing/testing progressively more intensive chemotherapy, outcomes for osteosarcoma (the most common bone tumor) remain dismal once it has spread beyond the initial site. Dr. Smith wants to understand why these immunotherapies have failed by studying a model closely resembling human osteosarcoma. Based on his findings, Dr. Smith will test novel immunotherapies to prioritize the next generation of osteosarcoma human clinical trials.

Based on progress to date, Dr. Aziz-Bose was awarded a new grant in 2026 to fund an additional year of this Fellow grant.

Even after being cured, childhood cancer survivors face challenges to living a healthy life, and one major challenge is heart disease. Heart health is closely linked to healthy eating, but many survivors cannot eat as healthily as they want because they don't have access to, or can't afford, healthy foods ("food insecurity"). Dr. Aziz-Bose will enroll survivors in this study to ask what they are eating, and understand whether they experience food insecurity and other conditions that put heart health at risk. Survivors will also be interviewed for their ideas about how to support healthy eating, including the best ways to directly give families healthy foods, an approach called "food is medicine." Using this information, Dr. Aziz-Bose will fine-tune a "food is medicine" intervention that she developed, and test it on a larger scale to see its impact on food insecurity and heart health. The goal being to understand and tackle barriers to healthy eating so all survivors can have the best health possible.

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

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.

Leukemia and lymphoma are blood cancers that are a major cause of death in children. Many of these cancers are curable with chemotherapy, but in some people the cancer comes back and is harder to cure. A new treatment called CAR-T cells involves genetic engineering of a cancer patient's own immune system cells to fight cancer, and can cure many people. However, this treatment still does not work well enough in about half the people who get it. Dr. Pauerstein proposes improving the sensitivity of CAR-T cells to cancer using engineered cell adhesion molecules, a type of molecular glue between two cells. CAR-T cells do not attach to cancer cells as strongly as normal T cells do, and this limits their ability to find and kill cancer cells. An engineered adhesion will be used in combination with CARs to improve the ability of CAR-T cells to kill cancer. Dr. Pauerstein and team will also study how changes in cell adhesion affect how CAR-T cells kill cancer. This work should improve cell-based treatments for blood cancers.

Based on progress to date, Dr. Spear was awarded a new grant in 2026 to fund an additional year of this Fellow grant.

Neuroblastoma is a devastating pediatric cancer, with only 50% survival in aggressive "high-risk" disease. Survivors are burdened with life-long side effects from chemotherapy and radiation. Newer therapies, such as cancer vaccines, provide an opportunity to mobilize a patient's own immune system to find and destroy cancer cells. Identifying the unique genetic signature of an individual patient's tumor allows scientists to formulate a personalized vaccine to stimulate the immune system to recognize tumor-specific mutations, called "neoantigens". Dr. Spear has developed a new tool to identify these unique genetic signatures (neoantigens) and test the effectiveness of the neoantigen vaccine in modes. These findings will lay the groundwork to develop a clinical trial using personalized vaccines for high-risk neuroblastoma and other pediatric cancers.

This first 2 years of this grant were funded by and named for the Arden Quinn Bucher Memorial Fund, a St. Baldrick's Hero Fund. Arden’s intelligence, empathy, and dynamic personality charmed everyone and is now her legacy. Before her neuroblastoma diagnosis on October 11, 2007 at age two, she happily played with boundless energy and imagination. Even throughout her difficult months of treatment, Arden bravely managed to keep smiling and learning. This fund supports St. Baldrick’s mission: funding the most promising research, wherever it takes place to provide kids fighting cancers less toxic, more effective treatments allowing them to live longer, healthier lives.

The 3rd year of this grant is funded by Allied World, a global provider of insurance and reinsurance solutions.

Brain tumors are the leading cause of cancer related death in children. The outcomes for high-grade gliomas in children are dismal. Chimeric antigen receptor (CAR) T cells are genetically engineered cells programmed to target cancer cells with high precision. The application of CAR T cells in brain tumors in children is still limited compared to leukemia. One challenge is that CAR T cells need multiple hits to kill brain tumor cells compared with leukemic cells, where a single hit is sufficient. Dr. Abu Arja and team discovered a subset of CAR T cells that are more potent and can more proficiently kill brain cancer cells by increasing their lethality, making a second hit unnecessary. In this project, Dr. Abu Arja is studying the cellular program of this unique subset of potent killer CAR T cells to better understand why they are superior killers. Dr. Abu Arja plans to use these findings to genetically engineer new enhanced CAR T cells to eliminate tumors in children with brain cancers.

The first year of this grant is funded by and named for the Be Brooks Brave Fund. Despite his diagnosis at age 5 with inoperable brain and spinal tumors, Brooks taught so many people what life is truly about--love. He was BRAVE beyond his years with an inspiring “faith over fear” attitude. This Hero Fund hopes to raise money for high-grade glioma research so no other family will hear the words, “there is no cure”.

Osteosarcoma is the most common bone tumor in children yet the survival rate remains low, below 75%. Children who are born with or develop certain mutations or who have been exposed to radiation or chemotherapy are more likely to get this cancer. However, not enough is known about how osteosarcomas develop. To learn more, researchers must better understand how normal bone cells become osteosarcoma cells. Dr. Cantor and colleagues have previously seen that patients with this cancer have elevated serum levels of abnormal DNA sequences (repetitive element DNAs) that may affect how these cells behave. Dr. Cantor and colleagues are creating models that mimic the cancer formation process to define the factors that drive the production of these abnormal DNA sequences and the effects of such sequences on the osteosarcoma cell behavior. Through these studies, Dr. Cantor hopes to learn more about this previously unrecognized abnormality. Better understanding of this process may allow researchers to develop new therapeutic approaches for children with osteosarcoma.

Acute Myeloid Leukemia (AML) is a blood cancer that sadly takes the lives of many children each year, and major efforts are being made to save these lives. One idea has been to teach the patient's body to fight off the AML like it would fight off an infection. This strategy alters the patient's immune system by making CAR-T cells, which are cells that fight cancer. CAR-T cells have been successful in curing patients with another similar type of blood cancer, but when it was tried in patients with AML, the approach was less successful. Dr. Anand's project is to understand why it didn't work as well so that further improvements that lead to cures for kids with AML can be made.

This grant is generously supported by the JJ's Angels Hero Fund which honors the memory of Juliana LaMonica and her courageous battle with AML.
Diagnosed at the age of two, Juliana underwent a bone marrow transplant but passed away shortly after turning three. Her sweet spirit and charismatic personality continue to inspire people to support the funding of pediatric cancer research through Team JJ’s Angels.

Based on the progress to date, Dr. Campbell was awarded a new grant in 2025 to fund an additional year of this Fellow grant.

The goal of this project is to engineer immune cells to target cancer, particularly a type of pediatric cancer called acute myeloid leukemia (AML). AML cells develop strategies to escape surveillance by the immune system. Despite current therapies, cancer cells are able to survive and progress. Natural killer (NK) cells play an important role in the immune response to cancer by recognizing and killing tumor cells. NK cell activity is regulated by activating and inhibitory receptors. Tumor cells express proteins that provide inhibitory signals to NK cells, blocking NK cell anti-tumor functions and allowing for tumor escape. Dr. Campbell and colleagues propose to tip the balance in favor of immune cell activation by knocking out a key NK cell inhibitory receptor, TIGIT. Dr. Campbell hypothesizes that eliminating NK cell TIGIT expression will remove inhibitory "brakes" on NK cell activation and enhance anti-tumor activity. The purpose of this study is to develop an effective cellular therapy for pediatric AML.