Grants Search Results

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.

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.

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.

Despite breakthroughs in cancer biology, pediatric solid tumors have seen minimal improvement in patient outcomes. Neuroblastoma, the most common solid tumor malignancy of childhood, encapsulates the full spectrum of cancer heterogeneity. Dr. Mosse’s team have shown that a specific ALK inhibitor is far superior than the current targeted therapy being tested in a Children’s Oncology Group Phase 3 trial for patients with an ALK genetic alteration. To ensure that the proper clinical and correlative studies are done to identify all patients whose tumors harbor an ALK genetic alteration using a custom-designed and targeted deep sequence capture panel. In parallel, Dr. Mosse and colleagues will adapt this panel to capture circulating tumor DNA (ctDNA) in the blood, also called “liquid biopsies,” of these patients and follow them over time to learn how they respond to our therapies and if/how their tumors develop resistance. While liquid biopsies have become a validated clinical tool in a subset of adult malignancies, its utility in pediatric cancers remains unproven. Liquid biopsies have the potential to overcome many of the limitations we face with solid tumors, as ctDNA abundance tracks with disease burden, reliably captures tumor genomic heterogeneity, and portends patient outcomes. Dr. Mosse and colleagues hypothesize that there is a critical unmet need to harness minimally invasive ctDNA assays to elucidate actionable targets in high-risk neuroblastoma, monitor response to therapy and disease burden, and establish circulating nucleic acid detection as a clinical biomarker for pediatric solid tumors. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Acute myeloid leukemia (AML) as an aggressive pediatric cancer associated with poor outcomes and few changes in therapies over the past 30 years. The presence of small numbers of persisting leukemia cells after chemotherapy has become an important predictor of leukemia relapse, however, current assays used to detect residual leukemia have limited sensitivity and many patients with “no detectable leukemia” still go on to relapse. This underscores the need to identify and develop novel assays that more accurately determine optimal therapies and that improve upon current leukemia detection approaches for AML. Dr. Meshinchi and colleagues have performed functional genomic profiling (RNA sequencing) in 2000 children, adolescents, and young adults diagnosed with AML over the past 25 years that includes diagnosis, remission, and relapse timepoints. This preliminary data suggests that deep and functional cancer profiling across an unprecedented number of patient samples and timepoints informs relapse risk, enables a precision medicine approach that considers specific alterations within a patient’s specific cancer, and links diagnosis and relapse profiles with the goal of better understanding how/why relapses occur and how best to prevent them. Therefore, Dr. Meshinchi and colleagues plan to leverage this unprecedented dataset to develop an integrated genomic platform that will significantly improve prognostic determination and treatment decisions for children, adolescents, and young adults diagnosed with AML. Successful validation of our assays will therefore fill a critical unmet need in the field of AML, and the resulting product will be an optimized test ready for clinical use.

This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Acute Myeloid Leukemia (AML) is a heterogeneous disease characterized by malignant clonal expansion of undifferentiated progenitor cells. The relapse and refractory AML is one of the biggest challenge faced by clinicians as significant proportion of patients within this category have very poor outcome. Persistence of leukemic stem cells has been associated with higher risk of relapse, additionally these leukemic stem cells also show drug resistance characteristics. Dr. Lamba’s team has recently developed a gene-expression based pediatric leukemic stemness score and drug resistance score that has shown promising results in not only identifying patients with higher risk of relapse and poor outcome but is also suggestive of response post-transplant. Dr. Lamba is also focused on inherited genetic polymorphisms to study pharmacogenomics markers specific to the standard chemotherapy regimen. We recently developed a pharmacogenomic score for are-C the mainstay of AML chemotherapy that associated with treatment outcome and survival. This project seeks to validate the gene expression and genotype-based scores in large cohort of patients treated on a Children’s Oncology Group clinical trial. The results will prepare a sound scientific rationale to incorporate a preemptive testing of patients for genomics based prognostic score that can be incorporated into the risk stratification of patients to guide precision medicine in AML. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Proteins on the surface of cancer cells provide some of the most promising target for new therapies in children with acute myeloid leukemia (AML). It is important to know actual number of molecules of mesothelin and E-selectin on the surface of the cell and how expression differs from patient to patient. Dr. Kolb will look at leukemia cells from 100 children enrolled on a Children's Oncology Group Phase III trial to quantify the amount of mesothelin and E-selectin on the leukemia cells for each. This data will provide proof that the assay works and can be used to determine eligibility for a clinical trial of mesothelin and E-selectin target therapies. Additionally, Dr. Kolb and colleagues will create a web-based portal for physicians to access the expression data for these other proteins on the surface of the leukemia cells. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

Neuroblastoma (NB) is the most common solid tumor in children outside of the central nervous system and children with high-risk NB typically have poor outcomes despite long and toxic upfront therapy. This project will define the evolution of the CAR natural killer T (NKT) cell program post-infusion using peripheral blood and tumor samples. Dr. Heczey will measure the effect of CAR-NKTs on cancer cells, the tumor-associated macrophages (TAMs), and other tumor components and vice versa at the single-cell level. Additionally, Dr. Heczey’s team plans to characterize the interactions between CAR-NKTs and tumor cells with the aim of determining how CAR-NKTs can overcome the challenges and counterattacks mounted by the NB tumor. The results of this project should identify molecular programs of CAR-NKT cells that are crucial to fighting cancer cells; such findings will be highly informative in boosting the anti-tumor activity of NKT cells for cancer immunotherapy. This study will advance the development of an effective, safe therapy for children with relapsed or refractory high-risk NB and should inform the next generation of cell-based immunotherapies This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

High-risk neuroblastoma is a very aggressive childhood solid tumor and approximately only half of patients with high-risk neuroblastoma survive. Dr. Goldsmith will be evaluating biomarkers of an antibody drug targeting GD2 and chemo-immunotherapy in three active clinical trials within the Children’s Oncology Group. Performing the same biologic correlative assays across three trials will not only answer key clinical questions regarding GD2 targeted therapy response in patients at different stages of treatment, but also provide an unprecedented opportunity to evaluate novel biomarkers that may guide treatment for future patients. Dr. Goldsmith hypothesizes that rational selection of therapy based on results of validated biomarker studies will improve the care of children with newly diagnosed high-risk neuroblastoma, thereby reducing the number of children who relapse and reducing the burden of acute and late effects of therapy. This grant is funded through a partnership between the St. Baldrick’s Foundation and the American Cancer Society.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

In Africa, the majority of children who get cancer die from their disease. This happens in many cases because the patients do not get a correct diagnosis. Without a precise and correct diagnosis, these children cannot benefit from the newest curative treatments. To help prevent this from happening, Dr. Lutwama will develop and test a strategy to diagnose pediatric cancer correctly in a manner that is affordable, reliable, and within a shorter time frame in resource-limited settings.

Based on progress to date, Dr. Moghimi was awarded a new grant in 2023, 2024, and 2025 to fund an additional year of this Scholar grant. In recent years, a very successful immunotherapy strategy to modify a patient's immune cells (called T-cells) to attack cancer has been developed for children with leukemia (cancer of blood cells). These modified immune cells are called Chimeric Antigen Receptor T cells (CAR-T cells). These CAR-T cells are very potent and do a better job than any chemotherapy at killing cancer. However, this life-saving tool has been available only to a small group of patients and for a handful of cancers. This is because most cancers don't have the targets those CAR-T cells aim for, or they have a target that can also be found on normal organs. As a result, these CAR-T cells could harm normal organs as collateral damage, a significant adverse effect of treatment that clinicians would want to avoid. In this proposal, Dr. Moghimi is striving to build the next generation of CAR-T cells that solely react to a combination of targets. These cells recognize a tumor only if they have both targets in sight and will not otherwise attack normal organs. CAR-T cells that operate based on a combination of two targets are more accurate than other targeting cells. Using this new generation of CAR-T cells, researchers would be able to significantly expand the availability of this powerful treatment to many more patients. Dr. Moghimi and colleagues will develop these special CAR-T cells for patients with Acute Myeloid Leukemia (AML), another common form of leukemia with a higher mortality rate for children. These results will provide pre-clinical evidence that could quickly translate to new clinical trials for children with relapsed AML through the Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL) consortium, an international collaboration headquartered at CHLA.

Based on progress to date, Dr. Wu was awarded a new grant in 2023 and 2024 to fund an additional year of this Scholar grant. Diffuse midline glioma (DMG) is a fatal brain cancer in children and there are no effective treatments. The brain's natural barrier prevents drugs from reaching the tumor. Focused ultrasound (FUS) uses sound waves to temporarily open the blood brain barrier to increase drug delivery to the protected tumor cells in the brain. Dr. Wu will be using panobinostat, a promising drug tested in cancer cells in the laboratory to examine if FUS can increase its delivery and whether the addition of radiation can further improve the outcomes. This grant was awarded at Columbia University Medicine Center and transferred to Virginia Tech.

The 2023 and 2024 years of this grant is co-funded by the Focused Ultrasound Foundation.

The 2021 and 2022 years of this grant are funded by and named for Hannah’s Heroes, a St. Baldrick's Hero Fund established to honor Hannah Meeson. At age six she was diagnosed with anaplastic medulloblastoma. After a relapse and several additional months of treatment, Hannah currently shows no evidence of disease. Throughout her treatments, Hannah never complained and remained positive and happy. This fund pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Based on progress to date, Dr. Jones was awarded a new grant in 2023 to fund an additional year of this Scholar grant. Acute myeloid leukemia (AML) is a difficult to treat cancer that is associated with death in nearly 4 out of 10 children who are diagnosed with this disease. We know that there are multiple factors that contribute to poor outcomes in these patients, however, researchers don't fully understand all of them. Dr. Jones will gain a greater understanding of the resistance associated with a specific type of AML that is particularly difficult to treat. She hopes to gain clarity about this type of disease to find more specific therapies to target those resistance mechanisms in the cancer cells.

Pineoblastoma (PB) is a rare, highly malignant form of brain tumors in children arising from the pineal gland, a tiny organ deep within the brain. The average 5-year survival rate of PB patients is 58%, but drops to 15% in children less than 5 years of age. Because of the location of the tumor, PB can be very difficult to treat. Current treatments include surgical resection followed by radiation and chemotherapy, however, a significant proportion of surviving patients suffer from severe treatment-related late effects and tumor recurrence. Thus, this presents an urgent need for novel therapeutic modalities to improve PB patient survival while minimizing adverse side effects. Proton therapy is one of the most precise and advanced forms of radiation therapy with pencil-beam scanning that allows for specific treatment of tumors, while sparing surrounding healthy tissues. Recently a highly targeted form of proton therapy, known as “FLASH”, with an ultrahigh dose rate, shows less toxicity and improved healthy tissue sparing, while maintaining effectiveness in eradicating tumor cells. As the recipient of the Lauren’s Pediatric Pineoblastoma Fund Research Grant, Dr. Lu and colleagues are investigating the impact of novel FLASH proton treatment strategies on PB growth and recurrence. This research will further define tumor cell diversity and identify treatment-resistant cells and mechanisms in relapsed tumors, as well as determine the effectiveness of combined proton therapy with immunotherapy on PB. These studies will establish proof-of-principle for potential effective therapeutic interventions in PB eventually leading to reduced long-term treatment related side effects and better survival outcomes for patients with this devastating cancer.

This grant is funded by and named for Lauren’s Pediatric Pineoblastoma Fund. Lauren was diagnosed with pineoblastoma at the age of 3 and relapsed two years later. She has spent half her life in treatment but is defying the 5% survival odds given at relapse as a disease stable, happy 11 year old today. But her family lives with daily uncertainty because chemotherapy is no longer effective and Lauren has visible tumors in her brain and spine that have been dormant for two years. They are acutely aware there are no treatment options. This Hero Fund was established with the goal of making it possible for researchers to include pineoblastoma in brain tumor treatments.

Diffuse intrinsic pontine glioma (DIPG) is a deadly pediatric brain cancer. Tragically, the majority of children diagnosed with the disease die within 12 months because the most effective treatment, radiation, is palliative at best. Therefore, there is a significant need to develop new therapeutic strategies to improve the terrible outcomes for these patients. Looking at genes that are turned on or off in a cancer can be helpful to figure out what is causing cancer growth. DIPGs are known to have mutations in a group of genes called histones that intriguingly regulate whether other genes in the cell are turned on or off. While looking at genes that are turned on or turned off in DIPG, Dr. Tsai found a gene called FOXR2 that is turned on in a subset of these tumors. FOXR is not usually present in the normal brain, but it has high levels in a subset of DIPGs. This is exciting because if researchers can target FOXR2 with new therapies, only tumor cells would be affected, sparing the normal cells in the brain. The goal of this project is to figure out exactly how FOXR2 makes DIPGs grow and to identify strategies that can be used in new treatments to target FOXR2.

A portion of this grant is generously supported by Griffin's Guardians, a St. Baldrick's partner. Griffin's Guardians was created by the Engles in memory of their son, Griffin. Their mission is to provide support and financial assistance to children battling cancer in Central New York, raise awareness about pediatric cancer and provide funding for research.

Children with some kinds of blood cancers (leukemias) are not cured by regular chemotherapy and are at high risk of dying without better treatment options. Dr. Niswander is working to create new treatments that are more personalized for each child’s leukemia cells. The first treatment targets ‘miswired’ communication networks inside the leukemia cells that make them cancerous, and the second treatment uses the body’s own immune system to attack the leukemia cells. Each of these treatments is able to kill a patient’s cancer cells. But, eventually the leukemia cells develop changes that allow them to begin growing again despite the therapy, and the cancer comes back. These two therapies have never been combined together in patients. In this project, Dr. Niswander and colleagues are studying the best ways to combine these new treatments for two kinds of high-risk pediatric leukemias, since often two treatments that work in different ways are better than one. She is hopeful that by using patients’ own leukemia cells they will identify the best personalized treatments for future testing in pediatric patients to improve their chances of cure and living long and healthy lives.

For 2022, this grant is named for the Invictus Fund, a St. Baldrick’s Hero Fund created in memory of Holden Gilkinson. It honors Holden's unconquerable spirit in his battle with bilateral Wilms tumor by funding cures and treatments to mitigate side and late effects of childhood cancer.

In 2021, this grant was generously supported by Super Soph's Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.

Neuroblastoma is one of the most common pediatric tumors, responsible for 12% of all cancer deaths in children under 15 years old. Only about 50% of patients with widespread neuroblastoma will live for ten years after diagnosis. A recent breakthrough in cancer treatments known as CAR T cell therapy reprograms a patient’s own immune cells to recognize tumor cells. While CAR T cell monotherapy works for some cancer types, several research studies using CAR T cells to treat neuroblastoma have been relatively unsuccessful. This is likely due to immune suppression caused by the tumor itself. Interestingly, it is known that if a person with cancer develops an infection, the infection can stimulate an immune response that will promote cancer remission. With this knowledge, Dr. Kudek and colleagues have pioneered an innovative technique to boost CAR T cell therapy response. They have shown that the cancer-destroying function of reprogrammed immune cells is boosted when a weakened infection is introduced into a tumor and found that this treatment combination in bladder cancer led to cure in most of the disease models. Encouraged by these findings, he is pursuing proof-of-principle studies to determine how this treatment approach can be best applied to neuroblastoma treatment.

This grant is named for the LukeStrong a Force Against Neuroblastoma Childhood Cancer Fund. When Luke was 5 years old, he was diagnosed with high-risk neuroblastoma. He is now in his teens and still in active treatment for relapsed neuroblastoma. Since 2014 Luke’s “Never tell me the odds” attitude has inspired his family and friends to shave their heads, fundraise with St. Baldrick’s, and help conquer childhood cancers.