Grants Search Results

Dr. Stanton and colleagues are developing new approaches to understand how DNA is organized for gene expression in a lethal childhood tumor called rhabdomyosarcoma (RMS). In RMS, the standard of care therapies haven't changed substantially in 40 years and patient outcomes haven't improved greatly during this time. New approaches are desperately needed. There is a lack of a fundamental understanding of the mechanisms for how the cancer-causing genes function in RMS. Dr. Stanton and his team are integrating cutting edge approaches in synthetic biology, modeling, and genomics to understand how and why RMS forms, with the granularity of single gene targets for mechanism studies. Through Dr. Stanton's studies, the community will gain an understanding of how the cancer-causing genes are altering the organization of DNA in RMS cells.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. Aiden Binkley who was diagnosed with Stage IV rhabdomyosarcoma at age 8. This bright, funny and courageous little boy believed he got cancer so he could grow up to find a cure for it. His vision is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Neuroblastoma (NB), a cancer that commonly affects young children, often presents with aggressive clinical behavior and poor prognosis, making the identification of effective therapeutic targets essential. NB is known for its resistance to conventional chemotherapy, and one of the mechanisms contributing to this resistance is the activation of a key regulator of gene expression, known as 'NF-kB’. NF-kB activates the expression of genes that contribute to NB survival. NF-kB also plays a role in promoting spread of NB to other parts of the body (e.g. bone marrow, liver, and lymph nodes). Because of its critical role in regulating survival, inflammation, and metastasis, NF-kB presents an attractive target for novel therapeutic strategies in NB. Inhibition of the NF-kB pathway can potentially sensitize NB cells to chemotherapy, reduce tumor growth, and inhibit metastasis. Dr. Letterio and colleagues will explore the activity of a new class of drugs (known as SOTs), that are potent inhibitors of NF-kB.

This grant is named for David's Warriors, a St. Baldrick's Hero Fund. The fund was created in memory of David Heard who battled neuroblastoma until his passing at the age of ten. David inspired his family and countless others to commit to raising money for research to fight pediatric cancer through the St. Baldrick’s Foundation. The Fund honors the amazing spirit with which he lived, embracing life until the very end.

Brain tumors are the deadliest type of cancer that afflicts children. The ability of brain tumor cells to spread (metastasize) outside of the original tumor along the leptomeninges, the covering of the brain and spinal cord, is responsible for making many brain tumors so hard to treat. How cancer cells embedded in the leptomeninges survive, thrive, and resist best treatments is poorly understood. A better understanding of leptomeningeal metastasis is required to make new therapies that can meaningfully increase survival for children diagnosed with aggressive brain cancers. Dr. Reinecke and colleagues will create a way that can identify and screen potential therapies in a cell culture dish, thereby streamlining interventions they take to models of pediatric brain tumors. Dr. Reinecke and colleagues believe that establishing this preclinical platform has the potential to identify therapies that have a chance to positively impact the lives of children diagnosed with metastatic brain tumors.

Ewing sarcoma is an aggressive bone tumor in children, adolescents, and young adults. New therapies with greater efficacy and less toxicity are urgently needed to save the lives of these young patients. Dr. Theisen and colleagues will identify a new vulnerability in the mitochondria (i.e. the powerhouse) of Ewing sarcoma cells as well as several possible drugs that target this pathway. Dr. Theisen and team will determine both the reason that Ewing sarcoma cells have this unique vulnerability and how best to target this pathway therapeutically. In the long term, this will lead to better ways to treat Ewing sarcoma.

This grant is named for Julia's Legacy of Hope, a Hero Fund that honors Julia's positive and courageous spirit and carries out her last wish: "no child should have to go through what I have experienced". Diagnosed at age 16 with Ewing sarcoma, Julia fought cancer and survived only to be stricken in college with acute myeloid leukemia, a secondary cancer as a result of treatment. Through this Hero Fund, her family hopes to raise awareness and funds for childhood cancer research especially for Adolescent and Young Adult (AYA) patients.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Cellular and Immunological Approaches to Prevent Relapse: Pediatric Blood and Marrow Transplant Consortium (PBMTC). For a description of this project, see the consortium grant made to the lead institution: National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Cellular and Immunological Approaches to Prevent Relapse: Pediatric Blood and Marrow Transplant Consortium (PBMTC). For a description of this project, see the consortium grant made to the lead institution: National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Cellular and Immunological Approaches to Prevent Relapse: Pediatric Blood and Marrow Transplant Consortium (PBMTC). For a description of this project, see the consortium grant made to the lead institution: National Marrow Donor Program, Minneapolis, MN.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Cellular and Immunological Approaches to Prevent Relapse: Pediatric Blood and Marrow Transplant Consortium (PBMTC). For a description of this project, see the consortium grant made to the lead institution: National Marrow Donor Program, Minneapolis, MN.

Children receiving bone marrow transplant can have serious complication such as bloodstream infections and graft versus host disease and some children die of these complications. Alteration of the bacteria in the gut by treatments including antibiotics is an important cause of these complications. In a previous study Dr. Davies and colleagues have tested the use of human milk to help keep gut bacteria healthy in very young children and found that this treatment worked. They are now studying a purified sugar from human milk, 2-FL that can be given easily as a medicine. Dr. Davies will also test a novel rapid urine test and a blood test to assess health of the gut bacteria during the study. Current tests require a stool sample and can take a long time. This trial will generate the data needed to perform a large-scale multi-center randomized clinical trial that will best prove how well this treatment works.

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.

This institution is a member of a research consortium which is being funded by St. Baldrick's: New Approaches to Neuroblastoma Therapy (NANT) Consortium. For a description of this project, see the consortium grant made to the lead institution: Children's Hospital Los Angeles, Los Angeles, CA.

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.

Brain and spine tumors are the leading cause of cancer-related death in children and adolescents. While cure can sometimes be achieved with conventional chemotherapy, surgery, and/or radiation, prognosis is dismal for patients whose aggressive brain/spine tumors progress despite these treatments. There is a critical need to develop new effective, well-tolerated therapies for children, adolescents, and young adults with refractory high-grade brain/spine tumors. Lutathera is a targeted radiotherapy which binds to tumor cells that express somatostatin receptors, causing tumor cell death through localized release of radiation, with minimal side effects. Many pediatric and young adult high-grade brain/spine tumors express somatostatin receptors, making them ideal targets for this therapy. Dr. Lazow is conducting a clinical trial to assess the safety and effectiveness of Lutathera in children and young adults with recurrent high-grade brain/spine tumors. Within this trial, she will also 1) evaluate how somatostatin receptor expression varies across different brain/spine tumors and determine clinical, imaging, pathology, and genetic characteristics which correlate with that expression, 2) identify imaging and molecular biomarkers predictive of response to Lutathera and/or disease recurrence, and 3) perform radiation dosimetry to establish optimal dosing of Lutathera in children and young adults, ensuring adequate tumor penetration while minimizing toxicity. If Lutathera proves safe and effective in treating children and young adults with refractory brain tumors, further studies will be planned to expand to a larger patient population and eventually incorporate Lutathera into upfront treatment backbones for these aggressive diseases.

This grant is funded by and named for the Miracles in Memory of Michael Fund, a St. Baldrick's Hero Fund created in memory of Michael Orbany who was diagnosed with medulloblastoma when he was 6 years old. After completing initial treatment, his cancer relapsed within a year and he passed away at the age of nine. Michael had unwavering faith and perseverance, wanting most of all to make others happy. This fund honors his tremendous strength to never ever give up.

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.

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.

Rhabdomyosarcoma (RMS) is the most common malignant soft tissue tumor in childhood. Despite intensification of aggressive therapy involving combination chemotherapy, radiation and surgery, the overall outcome of RMS is among the least improved in childhood cancer. Dr. Huang and colleagues aim to explore a novel concept of applying a clinical available technique of tumor-reduction cryoablation, whereby tumors are damaged by ultra-cold argon gas or liquid nitrogen to release endogenous immune adjuvants, to enhance an efficacious systemic anti-tumor immunity against distant RMS metastasis. He seeks to procure preclinical efficacy and mechanistic data that will enable a rapid translational clinical trial targeting metastatic sarcoma within 3 years.

Bone marrow transplantation is a highly effective treatment for relapsed and difficult to treat forms of pediatric leukemia, but unfortunately has a high risk for dangerous side effects. Viral infections are a major problem in the weeks and months after bone marrow transplant while children's immune systems are still immature. These infections can be debilitating and even deadly while also being very difficult to treat since available antiviral medications frequently do not work. Over the last few years, researchers have had great success in combating these viral infections by taking T-cells (a type of infection fighting cell that is part of the immune system) donated by children's personalized stem cell donors and engineering them to attack and kill certain viruses. Additionally, the rates of side effects using this therapy have been incredibly low. Dr. Rubinstein now intends to offer this therapy as a preventative measure, with the hope that this strategy will decrease the number of patients suffering from dangerous viral infections after bone marrow transplant. This clinical trial has the potential to decrease the number of pediatric cancer survivors who die from infection while also shortening hospitalizations and decreasing the need for other anti-viral medications.

This grant is generously supported by the Rally for Ryan Fund, a St. Baldrick's Hero Fund. Ryan was diagnosed with ALL when he was 7 years old and began treatment immediately. Initially labeled “high risk” due to a poor response, he completed 3½ years of a difficult treatment protocol before relapsing 11 months later. After his third relapse and an unsuccessful immunotherapy trial, Ryan had a bone marrow transplant in December 2020. He is currently fighting graft vs. host disease but is doing well and is optimistic for a good response. The Campanaros created this Hero Fund to celebrate Ryan’s courageous spirit and knowing firsthand the importance of research, to raise funds to find better treatments for kids with cancer.

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 medical school student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty. Therapies that stimulate the immune system to target cancer cells are rapidly providing new and efficacious treatment options for pediatric patients with cancer. However, scientists are finding that solid tumors have evolved methods of inhibiting the immune system, minimizing the effects of these new therapies. One way of suppressing the immune system is through the upregulation of myeloid-derived suppressor cells. This study will help determine the prevalence and function of these cells in pediatric patients with solid tumors so that they can be specifically targeted to improve the efficacy of new immunotherapies.

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.