Grants Search Results

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

Each year, approximately 1000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, even the most effective targeted drug BCL2 inhibitor-venetoclax (VEN) cannot eradicate all leukemia cells. The remaining cells cause disease recurrence and result in a very low overall survival rate for AML patients. In preliminary studies, Dr. Li found that targeting an enzyme called ADSS2 promotes pediatric AML cells sensitivity to VEN induced mitochondrial apoptosis, resulting in a synthetic lethality. This study will ask how ADSS2 preserves AML cells fitness and test the effectiveness of a first-in-class ADSS2 inhibitor combined with VEN or other BCL2 family protein MCL1 inhibitor in models of AML. If successful, this could lead to a clinical trial with potential impact for childhood AML patients.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Project. For a description of this project, see the consortium grant made to the lead institution: University of California, Santa Cruz, Santa Cruz, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium. For a description of this project, see the consortium grant made to the lead institution: Baylor College of Medicine, Houston, TX.

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.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Treehouse Childhood Cancer Project. For a description of this project, see the consortium grant made to the lead institution: University of California, Santa Cruz, Santa Cruz, CA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

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.

Years 1 and 2 of this grant are 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.

Year 3 of this grant is made possible through funding from D-Feet Cancer, The Dalton Fox Foundation and named in memory of Dalton, who is the inspiration for the Foundation that is lovingly led by his parents and sister. Dalton loved to tell good jokes and had a contagious sense of humor that lit up any room he walked into. Even on his worst days, he inspired his community and family with his positive outlook. He loved baseball, swimming, fishing and watching Marvel movies and while his time here was much too short, his memory continues to motivate so many. The Foundation is focused on fueling more research for Ewing sarcoma.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Malignant Germ Cell Tumors International Consortium (MaGIC). For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

This institution is a member of a research consortium which is being funded by St. Baldrick's: Precision–based Therapy for Childhood Leukemia. For a description of this project, see the consortium grant made to the lead institution: Dana–Farber Cancer Institute, Boston, MA.

Burkitt lymphoma (BL) is the fastest growing, most aggressive pediatric tumor. In the 1960s, it was universally fatal. Over the past decades, clinical trials identified very high dose chemotherapy therapies as effective. Over 95% of children with BL in the US now survive. However, over 80% of cases of BL arise in children in sub-Saharan Africa (SSA) and other lower income regions where high dose chemotherapy is not currently feasible and in these settings BL is typically fatal. In the study, Dr. Allen builds on the observation that BL tumors from US and SSA are largely indistinguishable, but surprisingly tonsils from children in SSA and US have vastly different gene expression patterns. He therefore hypothesizes that the much higher rate of BL in SSA may not be due to intrinsic cancer cell factors, rather due to the nature of lymphoid tissues out of which the cancer cells grow. If Dr. Allen and colleagues can identify factors that lead to BL, they hope to create opportunities to prevent and treat BL in SSA.

This grant is funded by Danilo Gallinari and the National Basketball Players Association.

This proposal introduces four groundbreaking advancements in the treatment of Ewing sarcoma (EwS), a rare and aggressive cancer. Firstly, Dr. Li and colleagues aim to optimize and assess the effectiveness of cutting-edge anti-IL1RAP ADCs in treating EwS. Secondly, they seek to uncover new insights into the diversity and heterogeneity of targets within EwS tumors. Thirdly, will explore the potential of innovative bispecific ADCs to target a wider range of EwS cells, enhancing treatment efficacy and reducing the risk of relapse and spread. Lastly, Dr. Li will explore the possibility of applying these advancements to other IL1RAP+CD276+ cancers like acute myeloid leukemia (AML). Overall, this research holds promise for improving outcomes and broadening treatment options for patients with these challenging cancers. This grant is funded by and named for the D-Feet Cancer - The Dalton Fox Foundation. D-Feet Cancer - The Dalton Fox Foundation was established to honor and remember Dalton’s contagious smile and sense of humor, even on his toughest days with Ewing Sarcoma. He is an inspiration and the reason for the mission and interest in finding targeted therapies and treatments for Ewing Sarcoma, a pediatric bone cancer.

Adoptive immunotherapy has demonstrated unprecedented success in the treatment of pediatric leukemia. Extending its therapeutic potential to other pediatric malignancies such as glioblastoma (GBM) has proved challenging. In this therapy, T cells are isolated from a patient, expanded outside of the body, and genetically modified prior to reinfusion. The ability of these T cells to recognize and eliminate cancer cells is improved by expressing a protein (CAR) on the T cell surface. An important challenge is to minimize the manipulation of patients' T cells outside the body. Prolonged culture compromises their efficacy. Dr. Ghassemi developed approaches to generate CAR T cells in 1 day. These cells have increased potency. She is combining this recent development with a metabolic strategy to overcome the metabolic nature of tumor environment. This synthetic advancement combined with the production of CAR T cells in 1 day will lead to superior CAR T cells for cellular immunotherapies against pediatric GBM.

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”.

The majority of children with newly diagnosed Burkitt lymphoma (BL) are cured. Unfortunately, the outcome is poor for patients whose disease returns (relapse). The relapse is caused by multiple reasons but mainly is due to drug resistance and suppression by the tumor surroundings. Novel therapeutic approaches are urgently needed. Natural killer (NK) cells can attack cancer cells. Dr. Cairo is developing immunotherapeutic agents to enhance the functions of NK cells to kill BL. Expanded NK cells will be modified by genetic techniques to specifically target CD20 and a special protein will be developed to bind to another surface protein CD19 on BL. A virus will be created to secrete IL21 to enhance NK persistence and function. If successful, the combinatorial therapies will become available to pediatric BL patients in the clinical setting and would offer a potentially more effective and less toxic therapeutic approach, ultimately leading to improved survival.

This grant is funded by and named for Jack's Pack - We Still Have His Back, a St. Baldrick's Hero Fund. Jack Klein was a ten year old who loved life, laughing and monkeys. During his illness, his community of family and friends near and far rallied around him under the moniker "Jack's Pack". Their slogan was "We have Jack's Back". After Jack succumbed to Burkitt's Lymphoma, his "pack" focused their energy and efforts to funding a cure...just as Jack would have wanted.

There has been little recent progress in treating Ewing sarcoma, a pediatric tumor involving bone. Dr. Stegmaier and colleagues have used a technology called CRISPR to identify urgently needed, new therapeutic targets for this disease. They prioritized a class of targets which are expressed in immature but not mature tissues. These proteins are often abnormally re-expressed in cancers such as Ewing sarcoma. Thus, drugs targeting these proteins would be expected to have minimal toxicity. The Stegmaier lab identified the target IGF2BP1 as a top selective gene dependency in Ewing sarcoma; deletion of IGF2BP1 was more deleterious to Ewing sarcoma than all other cancer types screened. Importantly, IGF2BP1 is not expressed in most normal human cells. Dr. Stegmaier will validate IGF2BP1 as a therapeutic target in Ewing and will determine the mechanisms by which Ewing sarcoma cells rely on IGF2BP1 for growth. With IGF2BP1 chemical inhibitors in development, this project has exciting translational potential for patients with Ewing sarcoma.

This grant is funded by and named for The Ben Brandenburg Fund for Ewing Sarcoma Research. Ben passed away at the age of 15. He is remembered for his quick wit, indomitable spirit and bravery. This fund is his lasting legacy and ensures that research is funded so fewer children will have to suffer from Ewing Sarcoma.

Based on progress to date, Dr. Faulk was awarded a new grant in 2024, 2025, and 2026 to fund an additional year of this Scholar grant. Infant leukemia is an aggressive cancer with a very poor prognosis. The leukemia cells in most of these patients have a genetic change in which a gene (KMT2A) is broken and combined with other genes that typically do not interact with one another (this is called "rearranged"). A drug named SNDX-5613 has been developed that directly targets the changes that occur in a cell with a KMT2A rearrangement to specifically kill these leukemia cells, and it has shown promise in treating adult leukemia patients with a KMT2A rearrangement. An upcoming clinical trial will combine SNDX-5613 with traditional chemotherapy for children with leukemia with a KMT2A rearrangement that has come back (relapsed) or proven resistant to typical treatment (refractory). In addition to testing the safety and efficacy of SNDX-5613, studies will be done on patients’ blood and bone marrow samples to better understand how the drug functions in fighting leukemia. This trial represents the next step in evaluating this promising new targeted drug for these deserving patients, and the associated studies are key to helping us understand more about the biology of this leukemia and how to best target it.

This grant is named for the Oh Danny Boy, I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund. Danny O’Brien was just 5 months old when he was diagnosed with a malignant rhabdoid tumor on his liver. This cancer is extremely rare and aggressive. He endured chemotherapy to shrink the tumor for surgery, but the treatment was not effective. At the tender age of 9 months, Danny passed away. Fortunately, he knew nothing but love and affection all of his short life. This fund honors Danny’s courage and his unconditional love even in the midst of his battle with cancer.

High-risk medulloblastoma is a devastating childhood brain cancer that results in death in nearly 50% of patients. To improve future treatments for this disease, Dr. Prensner is studying a category of newly-discovered "dark proteins", which have been excluded from prior work due to their small size and unconventional locations in the human genome. He has found that a group of these dark proteins are critical for medulloblastoma cells to survive. This research will reveal how these dark proteins may point toward new approaches to treat medulloblastoma, which may be critical to define the next generation of anti-cancer therapies in this disease. This grant was awarded at Dana Farber Cancer Institute and transferred to the University of Michigan.

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.

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.