Grants Search Results

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!

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

Dr. Sykes is developing new therapies for childhood leukemia and lymphoma. Specifically, he is looking at a type of leukemia that develops from abnormal T-cells and is named acute lymphoblastic leukemia (T-ALL). T ALL is a particularly deadly disease if it does not respond to therapy (refractory) or if it responds initially and then comes back (relapsed). When a normal T cell becomes a leukemia cell, it develops certain advantages and certain disadvantages. Therefore, one way to kill a leukemia cell is to identify these disadvantages and to exploit those using specific drugs. This research focuses on how leukemia cells make DNA and RNA building blocks called nucleotides. An enzyme called DHODH is essential to the process of making nucleotides within the leukemia cell. Drugs that inhibit this enzyme rapidly kill the leukemia cells and spare the life of normal cells. Researchers call this approach 'nucleotide starvation' because it starves the leukemia cells of these DNA and RNA building blocks. Normal cells have back-up systems to deal with periods of nucleotide starvation. Dr. Sykes believes that leukemia cells have lost these back-up systems and that is why they are so sensitive to starvation. So far his research has shown that this nucleotide starvation approach works extremely well in leukemia cells outside of the body and in leukemia cells in laboratory mouse leukemia models. The fact that many DHODH inhibitor drugs are already available and have already been tested in humans suggests that clinical trials are feasible and could begin in a timely manner. Dr. Sykes hopes that DHODH inhibitor therapy will be effective treatment for children with T ALL, especially those children who have run out of other good treatment options.

Dr. Nguyen uses the patient's own immune T cells and armors them in the laboratory with a chimeric antigen receptor (CAR) to recognize neuroblastoma cells and kill them. Although she has demonstrated a robust anti-tumor effect of CAR T cells in models of neuroblastoma, she noticed that they can be overwhelmed by too many tumor cells and work less effectively. However, the CAR T cell function was restored when the cells were engineered to express tethered IL15 and -21 on their surface. Though these cytokines activated the CAR T cells and improved their function, models also showed signs of toxicity, which she hypothesizes to be caused by cytokine-driven CAR T cells. Therefore, this project will describe the manifestation and understand the cause of CAR T cell-associated toxicities in our neuroblastoma model. Furthermore, Dr. Nguyen and colleagues propose to reduce the side effects by engineering tethered cytokines that are predominantly expressed in the vicinity of tumor cells (conditional expression). The completion of this project will forge a new direction for the use of cytokines in CAR T cell therapy and render a new fourth-generation CAR T cell therapy safer for translation into the clinic. This honor award is without funding.

Cancer research allows scientists to modify specific immune cells to recognize and kill cancer. One type of immune cell is called the cytotoxic killer T cell. This T cell has a receptor (TCR) that is used to recognize a structure on the cancer cell's surface called a peptide-major histocompatibility molecules complex I (pMHC I). pMHC I complexes are diverse and are rarely shared amongst patients. This diversity prevents the use of a classic TCR across multiple patients to avoid tissue injury that known as graft versus host disease (GVHD). To bypass these limitations, Dr. Elsabbagh propose to develop T cells expressing a TCR that can target a protein called CD1d. Unlike MHC I, CD1d is not diverse and is well expressed on various childhood cancers including acute myeloid leukemia (AML), which has been known for high rates of treatment-related toxicity and disease recurrence. These modified cells will be pre-made and used universally in any AML or other children’s cancers that expresses CD1d. Dr. Elsabbagh and team will also attach a recent discovered enhancing protein called MyD88 to the created receptor to enhance their anticancer activity. They expect these modified T cells will be able to recognize and kill children AML cells.

The AACR-St. Baldrick's Foundation Award for Outstanding Achievement in Pediatric Cancer Research has been established to bring attention to major research discoveries to the pediatric cancer research community and to honor an individual in any sector who has significantly contributed to any area of pediatric cancer research, resulting in the fundamental improvement of the understanding and/or treatment of pediatric cancer. The recipient will nominate an emerging leader conducting research in the academic sector to receive a research grant. The 2022 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. David Malkin at The Hospital for Sick Children (SickKids). Dr. Alanna Church at Boston Children's Hospital received the 2022 research grant. Dr. Church's research interests are in bringing molecular testing to the clinical care of children with cancer to improve diagnoses and treatments.

Based on progress to date, Dr. Almaliti was awarded a new grant in 2025 and 2026 to fund an additional year of this International Scholar grant. There is no nice way to tell someone they've got a brain tumor, and with a child it's unimaginable. In fact, brain tumors are the leading cause of solid tumor cancer death in children. Proteasome inhibitors are a recently discovered drug class that is effective in many types of cancer and have reduced side effects to normal cells. Dr. Almaliti aims to develop novel potent and selective proteasome inhibitors that will specifically kill brain cancer in children. This innovative approach should result in the discovery of new clinical leads for treating brain cancers in children.

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.

This project focuses on engineering a component called a macrophage. Macrophages talk to T-cells, using a protein called a cytokine, helping to boost T-cell effects and eradicate a child's cancer. Macrophages within children who respond to engineered T-cells long term (do not relapse) are able to release cytokines or talk with T-cells more effectively. On the reverse side of that same coin, those small percentages of patients who present with serious side effects have macrophages that are too effective at talking to T-cells or are releasing too many cytokines. Dr. Gustafson is developing a new technology that predicts how effective macrophages will be at talking to T-cells. This technology can be used to prevent toxicity and reduce relapse, allowing for more kids to live healthy cancer-free lives. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Childhood Hodgkin lymphoma (a cancer of white blood cells) is highly curable. Modern chemotherapy regimens effect a cure in over 95% of children diagnosed, however, about 15-20% will suffer a recurrence of their lymphoma and need additional highly intensive chemotherapy and bone marrow transplantation. These intensive regimens have many serious chemotherapy-related side effects (infections, mouth sores, etc.). Dr. Wadhwa’s study will investigate a novel predictor of cancer relapse and serious chemotherapy-related side effects by studying the role of body composition at Hodgkin lymphoma diagnosis in cancer-free survival and chemotherapy toxicities. Body composition has already been shown to be a significant predictor of cancer relapse and chemotherapy toxicities in adults with cancer. Dr. Wadhwa’s team will examine the body composition of children at cancer diagnosis, its association with cancer-free survival and serious chemotherapy related toxicities, how body composition changes during cancer treatment, and whether changes in body composition during cancer treatment impacts survival. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure. New Mexico is a minority-majority state with over 50 percent of the state's population comprised of a large Hispanic and Native American population. With support from St. Baldrick's the University of New Mexico will increase the enrollment of their unique patient population in national and international clinical trials in order to increase the diversity of patient representation in these trials.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure. In El Paso many of the people are Hispanic, underserved in healthcare, and are mostly below the poverty line. Due to these facts, they do not get equal opportunities to participate in clinical research and are disadvantaged. Support from St. Baldrick's will facilitate clinical research in underserved populations with minorities.

The impact that cancer has on a child/teen reaches far beyond the physical ailment. The psychological impact can be just as devastating. Recent studies have shown that 32% of adolescent and young adult patients suffer from symptoms of post-traumatic stress disorder (PTSD). Akron Children's Hospital treats a minimum of 90 newly diagnosed children and adolescents with cancer annually. Akron Children's will assess patient and family experiences during and post cancer treatment. The goal of the study will be to create a uniform infrastructure to formalize pathways to support the emotional needs of patients and their families.

This grant has been funded by and named for The Abbey E. Foltz Fund, a St. Baldrick’s Hero Fund. Abbey was diagnosed with osteosarcoma of the right tibia when she was 14 years old and a freshman in high school. She loved school, spending time with her family and friends and dancing. All that changed as she battled cancer with ongoing treatments and surgeries. Yet through it all, Abbey remained positive, focused on helping others and aspired to be a nurse. Sadly, she passed away while in her first year of college. Her family carries on Abbey’s legacy of making a difference for patients and their families with this Hero Fund by funding childhood cancer research in Northeastern Ohio.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Myelodysplastic Syndrome (MDS) are clonal stem cell disorders characterized by abnormal cell growth and shape, lack of mature blood cells, and increased risk of acute myleoid leukemia (AML) development. Approximately 10,000 new cases are diagnosed every year in the United States. Survival ranges from months to years, and bone marrow transplantation remains the only cure. To identify new drug targets associated with more specificity and less morbidity and mortality, it is essential to understand the molecular course of MDS. Unbiased sequencing studies have identified over 45 recurrent somatic mutations in MDS patient samples. Of these pathways, splicing factor and epigenetic regulator mutations are the most common. Point mutations in splicing factor 3b subunit 1 (SF3B1) are found in less than 25% of MDS patients. Dr. Wallace and colleagues have shown that mutations in SF3B1 lead to an altered function, name upstream cryptic 3 splice site selection. Epigenetic regulators, including the de novo DNA methyltransferase, DNMT3A, are the second most common class of mutations identified in MDS. Studies on the effect of loss of DNMT3A expression have primarily been limited to the effects of altered methylation in regions outside of the gene body, such as enhancer accessibility. Although sequencing studies have shown that both splicing factor and epigenetic regulator mutations commonly co-occur as early mutations in MDS pathogenesis, it is unknown how altered DNA methylation and aberrant mRNA splicing can cooperate to promote MDS progression. Using cell line and animal model systems, Dr. Wallace will determine whether the cooperation of epigenetic regulator and splicing factor mutations lead to a more aggressive form of MDS. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Through this partnership with The Children's Cancer Foundation, proceeds from St. Baldrick's events in Hong Kong fund life-saving research in Hong Kong. The St. Baldrick's Foundation is proud to partner with the Children's Cancer Foundation and has been doing so since 2008.

This grant funded two projects. Project 1: Acute lymphoblastic leukemia (ALL) is a common childhood cancer. The survival rate for children with good risk factors is over 90% with low-intensity chemotherapy. However, children with high-risk disease have a higher chance of treatment failure and relapse. Two genetic mutations, MEF2D and KMT2A, have been found to have poor outcomes with conventional chemotherapy. The Chinese Children Cancer Group (CCCG) ALL 2020 protocol in Hong Kong Children's Hospital is adding the drug bortezomib to the treatment to improve outcomes for high-risk patients with these mutations. Project 2: patients with chemotherapy. However, 2-4% of patients under one year old, known as infant ALL, have a low survival rate of about 40%, especially when there is a genetic change involved. This patient group experiences more relapses in the bone marrow and central nervous system. Previous clinical trials have shown limited success. This project aims to improve the treatment of infant ALL with three new drugs: blinatumomab, venetoclax, and bortezomib. The study involves more than 20 hospitals in Hong Kong and mainland China, and patients will be monitored closely for safety and effectiveness.

This grant supports a Clinical Research Associate for the early phase trial program to ensure that more kids can be treated on clinical trials, often their best hope for a cure.

Diffuse midline glioma (DMG) is an aggressive pediatric cancer and outcomes are dismal, with a life expectancy of less than a year. It is particularly difficult to treat as they are commonly located in the brainstem near sensitive structures, meaning that surgical removal is not feasible. Recent advances in technology have led to development of “liquid biopsy,” which works by detecting small pieces of DNA that break off from the tumor and are found in the cerebrospinal fluid (CSF) and blood (termed cell-free DNA, cfDNA). This is important because these “liquids” are typically much easier to access than the tumor itself, which is particularly important in these brainstem tumors. Dr. Souweidane’s project will monitor cfDNA in the cerebrospinal fluid (CSF) and blood of DMG patients over time. In this study, Dr. Souweidane will implant ventricular access devices at time of standard biopsy in newly diagnosed DMG patients to provide on-going minimally-invasive access to CSF, and then will integrate this assay into early-stage clinical trials for DMG patients to see if it can be an effective biomarker of early response. Dr. Souweidane’s team believes the completion of these experiments will establish the utility of cfDNA liquid biopsy in DMG and will also, by guiding decision-making and management for brain tumor patients, dramatically change how we treat these devastating diseases. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

This grant supports a Clinical Research Associate to ensure that more kids can be treated on clinical trials, often their best hope for a cure. While facing an epidemic, natural disasters, and a pandemic, Hospital HIMA San Pablo Caguas has served 13-30% of new cancer patients age 0-19 years old in Puerto Rico. Puerto Rico's 99% Hispanic or Latino population has limited access to participation in clinical research. A Clinical Research Associate for childhood cancer research will facilitate an increase in the number of studies, patient participation, and data completion rate.