Grants Search Results

Based on progress to date, Dr. Conneely was awarded a new grant in 2022 to fund an additional year of this Fellow award. Acute myeloid leukemia (AML) is the second most common blood cancer in children and is difficult to cure. About one quarter of children with AML have a form of the disease called core binding factor (CBF) AML. Despite intense therapy, cancer will come back in one out of three children with CBF-AML. We want to find new ways to treat this common form of AML by learning how the specific combination of mutations in the cancer cells affect their ability to grow and survive. Some patients with CBF-AML have unique mutations that can stop cells from correctly fixing damage, allowing them to grow too quickly. The project will study how these mutations contribute to CBF-AML cells' development, growth, and survival, affecting the cancer cells' ability to grow using cancer cells with these unique mutations. This will help in understanding how this type of AML develops, and may lead to new ways to treat children with this disease.

This grant is generously supported by Double Deckers Destroy AML, a St. Baldrick's Hero Fund. Joel and Seth were not only identical twins but best friends. In an ironic twist of fate, both boys were diagnosed with Acute Myeloid Leukemia just three months apart. With the overlapping diagnoses and treatments, the family was separated for months at a time and looked forward to days when they could be together at home. Joel and Seth both received bone marrow transplants and endured complications from the procedures. Sadly, both boys relapsed. Surrounded by their loving family, Joel died in November 2017 at the age of three, followed by Seth in May, 2019 when he was four years old. The twins were named as 2020 Ambassadors for St. Baldrick's so their story can continue to inspire many. The Double Deckers Destroy AML Hero Fund was established because the Decker family strongly believes more research is needed for AML, especially when the disease has relapsed. They want to support research so other families won’t have to say goodbye too soon.

There are new cancer therapies in which a patient's own immune system is retrained to fight against their cancer. In one of these therapies, known as CAR-T cells, a patient's immune cells are removed from the bloodstream and reprogrammed to target and attack their cancer when the cells are returned to the body. While this therapy has shown great promise, there are still situations, especially with very high-risk cancers, where it does not work. One significant issue that exists with this treatment is that the retrained immune cells do not always stick around after being given back to the patient, which allows the cancer to outlast the therapy and come back. We know that once cancers have resisted a treatment once, it is difficult to use the same treatment again. This projects aims to find ways to alter tumor-targeting immune cells to make them last longer when they are given back to patients, ultimately allowing for a long-term cure for their cancer without the need for further treatment.

This grant is generously supported by the TeamConnor Childhood Cancer Foundation. TeamConnor Childhood Cancer Foundation's mission is to raise funds for national childhood cancer research programs, to build awareness that only a fraction of the NIH’s annual funding supports childhood cancer research, and to support inpatient programs. Founded in 2008 in honor and memory of Connor Cruse, TeamConnor has funded over $4M in pediatric cancer research grants across the United States.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Raman spectroscopy (RS) is used to characterize different types of cancer tissue. Usually RS fingerprints are obtained when a slice of cancer tissue is examined under a microscope. With a new design as a portable hand-held RS probe, the St. Baldrick's Foundation Summer Fellow will use the probe to determine RS fingerprints in cancer cell cultures. If successful, the project results could be used to design uses of the probe in the clinic setting to detect cancer cells in blood or other fluids.

This grant funds a medical student to complete work in pediatric oncology research for the summer. JMML is a rare type of childhood cancer that is really hard to cure. Right now, even our best treatments only stop this cancer for a year or so before it starts to come back. Cancers can be studied in specific models, which allow researchers to try out different drugs and treatments to see what works. The goal of this project is to use these models to find new treatments for JMML. This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.

This grant funds a student to complete work in pediatric oncology research for the summer. There has been little success in curing high risk AML patients, with survival rates remaining at < 25%. This highlights our current reliance on highly intensive cytotoxic therapies and stem cell transplant, and their inadequacies. This project studies the combination of novel target discovery with state-of-the-art stem cell expansion technology. Protein science provides a unique opportunity to generate one of the most impactful therapeutic discoveries in childhood AML in the last 40 years, with minimal toxicity. The summer intern will assist in investigating the impact of drugs on cancer targets while minimizing toxicity toward healthy cells. Results will be used to help identify critical genes involved in cancer growth and disease resistance, and to leverage future work in drug development.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Children with aggressive neuroblastoma have poor cure rates despite intensive treatment, and new therapies are needed. Treatments that inhibit important proteins and pathways in neuroblastoma tumors are likely to be more effective with fewer side effects. Kinases are proteins that control signals in cancer cells, leading to cancer cell growth and spread. This study proposes to test a certain inhibitor to determine its effectiveness against neuroblastoma cells and tumors. The results of these studies will determine whether BLU-667 is effective against neuroblastoma, potentially leading to clinical trials using BLU-667 for treatment of children with neuroblastoma.

This grant funds an undergraduate student and medical student to complete work in pediatric oncology research for the summer. T-cell acute lymphoblastic leukemia is a deadly childhood cancer that affects blood cells. The current treatment uses highly toxic medications. The goal of the proposed project is to test the efficacy of a novel, less toxic, targeted treatment for T-cell acute lymphoblastic leukemia. This award will train the student to perform experiments to test the efficacy of the novel treatment in T-cell leukemia and to determine the mechanisms of drug action against leukemia cells.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. This project will validate a drug for the medulloblastoma, a type of brain tumor, specifically tumors that spread from the original cerebellar location to the covering of the brain and spine (the meninges). This grant is named for the St. Baldrick's Foundation Staff whose generous gifts have helped fund this opportunity and may encourage students to choose childhood cancer research as a specialty.

This grant funds a student to complete work in pediatric oncology research for the summer. Osteosarcoma (OS) is the most common and highly lethal bone cancer affecting children and adolescent populations. New therapies are desperately needed for this highly aggressive disease, as outcome for metastatic OS has not improved over the past few decades despite the utilization of aggressive combination chemotherapy. The summer fellow will focus on testing a novel CA-IX small molecule inhibitor using syngeneic OS tumors in vitro and in vivo. Activities generated through this Summer Fellowship grant will lay the foundation for pre-clinical data for the use of CA-IX inhibitor in future clinical trials.

This grant funds a student to complete work in pediatric oncology research for the summer. This lab specializes in harnessing the power of a particular type of immune cells called macrophages and microglia which are the body's scavengers. This is done by blocking a "don't eat me" signal called CD47. The CD47 protein acts as a "don't eat me" signal to macrophages which normally engulf and devour cancer cells and other diseased and dying cells. It turns out that nearly every kind of cancer uses CD47 to evade these macrophages. Covering up the CD47 a "don't eat me" protein allows the immune cells to find and swallow cancer cells. Here we will test whether the ability of macrophages to eat tumor cells can be increased by blocking another immune dampening molecule called adenosine which is rapidly increased by tumor cells as they grow.

This grant funds a medical student to complete work in pediatric oncology research for the summer. Pediatric glioblastoma (p-GBM) is a lethal brain tumor that can affect children. This cancer is difficult to treat due to several factors, including the tumor's resistance to conventional therapies as well as the sensitivity of the surrounding healthy brain tissue. Children who undergo surgery to remove the brain tumor live an additional three to six years on average, though the quality of life may be low. Phosphoinositide 3-kinase (PI3K) is a protein family that normally regulates cell replication and survival. However, when it functions incorrectly, cells can experience unchecked growth and cause cancer. Inhibiting this protein family is a viable treatment option for cancer but blocking the whole PI3K family has severe side effects. It is imperative to understand each member of the PI3K family to better develop treatments that involve them.

This grant funds a medical student to complete work in pediatric oncology research for the summer. This year it is estimated that 800 children will be diagnosed with osteosarcoma (bone cancer). The lab has identified a gene (WNT5B) that is too high in a subset of osteosarcomas. By making a cell line that removes WNT5B, the lab will compare its growth to the original cells and target this gene in those cancers that have it to design a specific targeted therapy.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. Diffuse intrinsic pontine glioma (DIPG) is a type of children's brain tumor that currently has no cure or effective therapeutic options. This proposal aims to understand whether the target drug of ONC201, ClpP, can be targeted using novel compounds representing new potential therapeutics in DIPG.

This grant funds two students to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty. Project 1: Neuroblastomas are an enigmatic cancer of childhood with subtypes that have extremely good or poor survival. Poor prognosis neuroblastomas contain normal immune cells that help tumors grow. Important questions are 1) what is the repertoire of immune cells in neuroblastomas at time of diagnosis, 2) how the interplay between normal and tumor cells changes when tumors recur. The Summer Fellow will analyze images of tumors at recurrence and compare to the diagnosis images. These findings will provide insights into the types of immune cells that cancer cells rely on and may allow identification of new targets of therapy. Project 2: Decline in brain function may happen after irradiation to the brain in children. It is hard to predict the extent and speed by which it happens. There is suggestion that more rapid injury happens in areas with iron deposition. Using a novel MRI method that allows chemical identification and quantification of iron in the brain, the Summer Fellow will characterize the imaging changes in white matter of the brain in children who have been treated with radiation for their brain tumors. This will allow to then correlate the changes with future outcome of their cognitive function.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. DIPG's are the worst type of brain cancer children can get; there is no cure. This project will try a new approach to change that. Using large publicly available datasets from large experiments, 4 drugs have been identified that theoretically can slow down the growth of DIPG tumors. Researchers will test these four drugs against several DIPG models generated from patients. If results are positive, this could lead to new treatments for this deadly disease.

Ewing Sarcoma (EwS) is an aggressive bone and soft tissue tumor occurring in children and young adults. Approximately 25-30% of patients already have metastases at diagnosis and in spite of aggressive treatment, the survival for patients with metastatic disease remains dismal. EwS is considered an immune cold tumor that is largely resistant to conventional immunotherapy. Alternative treatment approaches are sorely needed, particularly in patients with metastatic disease. Dr. Sorensen and colleagues are using three novel strategies for targeting EwS tumors: 1) Inhibiting an EwS specific fusion protein that drives EwS tumor development. 2) Targeting a surface protein called IL1RAP. 3) Recruiting natural killer (NK) immune cells to EwS tumors and priming them to attack the tumor. This grant is the result of a generous anonymous donation to fund Ewing sarcoma research, specifically. It is in honor of a teenager fighting Ewing sarcoma, and is named the St. Baldrick's - Martha's Better Ewing Sarcoma Treatment (BEST) Grant for All.

Acute lymphoblastic leukemia (ALL) remains the most common cancer of children and young adults. Despite intensified treatments that achieved cure rates around 85%, there is a number of children who will relapse and succumb to therapy-resistant disease. One of the revolutions in the treatment of human cancer the last decade was immunotherapy, the ability of our own immune system to fight cancer. Unfortunately, despite its successes in a number of solid tumours, immunotherapy has not really impacted the treatment of leukemia, with the exception of CAR-T cell treatment of pediatric B-ALL. Indeed, some frequent types of pediatric ALL, and specifically T cell ALL (T-ALL) and its subtypes, have no immunotherapy treatment options. We believe that this is because we still don't understand how the cells of the immune system interact with the leukemia. Actually, researchers don't even know what type of immune cells are there available to fight the disease. Dr. Aifantis is applying a number of single cell techniques to create a map of the immune cells in the bone marrow of children with T-ALL. He is doing this at diagnosis of the disease, after treatment (remission) and when the children relapse. These studies will offer the first map of the immune system in pediatric ALL and will enable researchers to propose ways to activate the immune system to fight the tumour.

Based on progress to date, Dr. Stanton was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant.
Rhabdomyosarcoma (RMS) is a highly aggressive and lethal pediatric cancer affecting children and adolescents and arises in the soft tissue and skeletal muscle of the extremities, head and neck, and reproductive organs. From the clinical perspective, although patient outcomes have improved in general, nevertheless survival rates for some RMS tumors remains at less than 30%. One particularly aggressive subtype is alveolar RMS which is driven by the occurrence of chromosomal translocations resulting in the generation of chimeric or fusion proteins between the PAX3 or PAX7 and the FOXO1 genes. These are known as fusion-positive RMS (FP-RMS) and are associated with reduced relapse-free survival and generally poorer outcomes. But researchers still have limited understanding of how the "fusion" gene itself is driving the tumor, and no subtype-specific therapies exist. Dr. Stanton aims to determine how the fusion gene works with a protein complex known as BAF, to alter the epigenetic state of the cell to keep them dividing and stop the cell from differentiating into mature muscle tissue. His team is exploring the mechanism of how the BAF complex regulates the epigenetic state and memory of the FP-RMS. Furthermore, using small-molecule drugs and genetic depletion strategies (CRISPR) they will determine if FP-RMS tumors are dependent on the BAF complex for survival. Finally, they are working to identify potential novel therapies for patients with aggressive and lethal FP-RMS, to improve their outcome.

The 2021 and 2022 portions of 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!

The 2020 and 2023 portions of this grant is funded by and named for by Berry Strong, a St. Baldrick’s Hero Fund, established in honor of Caroline Berry. Diagnosed with alveolar rhabdomyosarcoma when she was 14, Caroline endured a two-year battle with courage and determination. Throughout treatments of radiation and chemotherapy and undergoing six surgeries, Caroline was a beacon of hope, unselfishly raising awareness and funding for research so no child would have to endure what she did. After a brief remission, scans revealed Caroline had relapsed and she passed away on Thanksgiving in 2018. Caroline is remembered as a bright light, creative, intelligent, funny and feisty who was always eager to share a smile with others. She continues to be an inspiration through the Berry Strong Hero Fund which will continue her legacy and her passion to raise awareness and fund the most promising childhood cancer research.

Lymphoma and leukemia are cancers that often strike children. Some types of these cancers cannot grow or survive without a protein called MALT1. As the Do It For Dominic Fund St. Baldrick's Fellow, Dr. Maurer found that, in some lymphoma cells, when the level of another protein called GRK2 was lowered, it led to more action of the MALT1, and more cancer growth. So, she thinks that GRK2 might be working to stop lymphoma tumors by blocking MALT1. She is working to find out two things: Does the level of GRK2 also affect the growth of leukemia? And how exactly does GRK2 interact with MALT1 to block its tumor-growing action? Understanding this interaction will help to design new treatments that work by blocking the MALT1 and stopping the growth of lymphoma cells, and perhaps leukemia cells too, so that children can be cured.

This grant is named for the Do It for Dominic Fund which honors the memory of Dominic Cairo who battled non-Hodgkins lymphoma and was a hero to his school and community. His family and friends continue to raise funds and support research in the hopes that no child has to go through what Dominic endured.

Based on progress to date, Dr. Velasquez was awarded a new grant in 2022 and 2023 to fund an additional year of this Scholar grant. Childhood acute myeloid leukemia (AML) is a blood cancer that is very difficult to treat in children and adolescents. T cells are one component of the patient's own immune system that helps defend against infections. New cancer treatments use modified T-cells that can ‘see' and kill cells that have CD123, a molecule present on AML, and have shown promising results in studies in the laboratory. Dr. Velasquez is testing if these specific T-cells are safe and can kill leukemia cells in children that have AML that came back after initial treatment. She also wants to see what happens to these T-cells after they have been given to the patient and how it affects the tumor. This information from the treated patients and the study of their T-cells and tumor cells will be useful in finding a cure for AML.