Grants Search Results

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.

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.

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.

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.

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.

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. Van Remortel 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.

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.

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.

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”.

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 grant is 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.

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.

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.

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.

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

Children with the brain tumor ependymoma have high relapse rates and poor long-term survival. Treatment options for ependymoma are limited and there is no known effective chemotherapy. Dr. Lindquist is working to make a new model of this tumor, to study how the tumor forms and grows, and to test new therapies in this model and patient-derived tumors. The ultimate goal is to identify new therapies that will extend the lives of children with ependymoma.

Cancer cells compete with the body for food. Some cancer cells use fat to grow, spread, and hide in the brain. When cancer cells hide in the brain, it is hard for chemotherapy reach them due to the blood brain barrier, which allows cancers to come back when they come out of hiding. Dr. Wang and colleagues are investigating how childhood leukemia uses fat to survive in the brain and how drugs that starve leukemia of fat can kill leukemia cells hiding in the brain.

The second year of 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 the progress to date, Dr. Kuo was awarded a new grant in 2025 to fund an additional year of this Fellow grant.

Ewing sarcoma (EwS) is a malignant cancer of bone and soft tissues that occurs mainly in children, adolescents and young adults. If the tumors spread, fewer than 1/3 will survive. For some pediatric cancers, recent progress has led to new treatments that use one's own immune system to target cancer cells. However, immunotherapy has not been successful for EwS because we don't know enough about how EwS tumor cells evade the immune system. The tumor microenvironment (TME) is an intricate ecosystem consists of cancer cells and the host's immune system. Dr. Kuo's project will focus on dissecting the TME of EwS, to understand how tumors develop. Using EwS tumors removed from pediatric patients during their cancer diagnosis and treatment, Dr. Kuo will use newly-developed techniques to map the TME and use a genetic model of EwS developed at CHLA to examine tumor/immune cell interactions in living tissue. The long-term goal of this work is to identify new treatment options for children with EwS.

This grant is funded by and named for The Shohet Family Fund for Ewing Sarcoma Research. In his freshman year of college, Noah was diagnosed with Ewing sarcoma. He endured many months of chemotherapy and had limb salvage surgery. Able to return to school, Noah had no evidence of disease for 2½ years until April 2018 when routine scans revealed he had relapsed. He passed away in May 2021 at the age of 25. Noah and his family were always passionate about the need for curative treatments for diseases of the AYA population. The Shohet family intends to raise funds for this Hero Fund in Noah's memory to find cures for Ewing sarcoma and to carry on his legacy of possibilities and hope.

Based on the progress to date, Dr. Young was awarded a new grant in 2024 to fund an additional year of this Fellow grant.

Osteosarcoma is a bone tumor that usually occurs in children and young adults and can be deadly especially when the tumor spreads to other body parts. The treatment strategy for this disease has not seen significant improvement in over 30 years, and there is no specific treatment for tumors that have spread throughout the body. In this project, the major goal is to identify factors that control the spread of osteosarcoma in order to develop new therapies to extend the lives of patients. Currently, Dr. Young is investigating whether osteosarcoma cells block the activation of one part of the patient's immune system, protecting the cancer cells from an immune attack and allowing them to spread throughout the body. This work has the potential to uncover new treatments to harness the immune system to fight this devastating disease.

This grant is named for the Team Jackson Hero Fund. The fund was established in honor Jackson Schmitt who died six days after his diagnosis with osteosarcoma from a stroke. Jackson’s story was told worldwide and his legacy lives on through funding life-saving osteosarcoma research.

One of the most exciting recent developments in cancer treatment is the growing ability to use the body's own immune system to directly fight tumors. However, these treatments still do not work on most patients, and we think it is critical to understand how each cancer type avoids the immune system. Dr. Schwartz is investigating how neuroblastoma, one of the most common pediatric solid tumors, escapes destruction by the immune system. To accomplish these goals, he will use cutting-edge technologies to dissect the immune biology in a model of neuroblastoma, with a particular focus on studying an important type of cancer-killing cell called a 'CD8 T cell'. Dr. Schwartz thinks him and his colleagues have identified an important new way that neuroblastoma evades these T cells. Their preliminary results suggest that neuroblastoma directly causes T cell death, limiting the ability of T cells to survive and kill enough tumor cells. He is trying to learn how neuroblastoma causes the death of T cells and find ways to block this immune evasion strategy. Most importantly, he predicts that combination treatment designed to block neuroblastoma's ability to kill T cells along with existing immune therapies will drastically improve the ability of the immune system to eradicate 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.

Based on the progress to date, Dr. Rahnama was awarded a new grant in 2024 to fund an additional year of this Fellow grant.

Acute Myeloid Leukemia (AML) is a blood cancer that affects children. While there have been important advances in treatment and care of pediatric patients with AML, 20-40% relapse and have poor outcomes. Novel therapies are urgently needed to combat this disease. One treatment modality under investigation involves manipulation of the body's immune system by reprogramming immune cells with inherent anti-leukemia properties to specifically target AML cancer cells. Dr. Rahnama is focused on the study of natural killer (NK) cells as immune cells of interest. NK cells can be engineered to express Chimeric Antigen Receptors (CARs) that recognize specific proteins on leukemia cells in order to attack and kill them. The site where the CAR-modified NK cell and the target leukemia cell come together is known as the immunological synapse (IS). The IS is highly organized and plays a key role in activating the NK cell. Dr. Rahnama aims to better understand the interaction between CAR-modified NK cells and target leukemia cells by studying the biology of the IS as related to how tightly the two cells interact. Her goal is to improve CAR-NK cell design for ultimate use as pediatric AML treatment. This grant was awarded at Johns Hopkins University School of Medicine and transferred to the University of California, San Francisco.

This grant is funded by and named for the Aiden's Army 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!