Grants Search Results

Leukemia and lymphoma are blood cancers that are a major cause of death in children. Many of these cancers are curable with chemotherapy, but in some people the cancer comes back and is harder to cure. A new treatment called CAR-T cells involves genetic engineering of a cancer patient's own immune system cells to fight cancer, and can cure many people. However, this treatment still does not work well enough in about half the people who get it. Dr. Pauerstein proposes improving the sensitivity of CAR-T cells to cancer using engineered cell adhesion molecules, a type of molecular glue between two cells. CAR-T cells do not attach to cancer cells as strongly as normal T cells do, and this limits their ability to find and kill cancer cells. An engineered adhesion will be used in combination with CARs to improve the ability of CAR-T cells to kill cancer. Dr. Pauerstein and team will also study how changes in cell adhesion affect how CAR-T cells kill cancer. This work should improve cell-based treatments for blood cancers.

Acute myeloid leukemia (AML) is the second most common type of leukemia in children. Despite treatment advancements, over 30% of children with AML cannot be cured. In AML cell populations, the leukemia stem cells (LSCs) make up a small part of the total, but are specially important: they provide a steady supply of new AML cells and are unfortunately very resistant to killing with drugs. Dr. Sui and colleagues believe that if they are able to kill the LSCs, they could cure patients with AML. Dr. Su has found that an enzyme called METTL1 is important in allowing LSCs to safely stay anchored in the bone marrow and identified a drug that inhibits METTL1 and eliminates LSCs. Dr. Su's study explores why METTL1 is important for LSCs and investigates how Dr. Su and team could best use their in-house developed METTL1 inhibitor to treat childhood AML using model systems. If successful, this research could pave the way for a clinical trial, offering hope for improved outcomes for childhood AML patients.

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.

Allogeneic cell therapy is a new approach to cancer treatment that harnesses living cells from healthy donors to fight tumors. To do so, immune cells are isolated from blood and incubated outside the body to expand subsets capable of killing cancer. Dr. Jonus and colleagues have shown that gamma delta (gd) T cells expanded from healthy adults help to eradicate neuroblastoma grown in models. Based on this,Dr. Jonus and team are performing a first-in-child clinical trial of gd T cells for patients with neuroblastoma. Going forward, Dr. Jonus's findings show an opportunity to make gd T cell therapy more effective by expanding a new type of gd T cell, Vd1, with unique properties that should improve both the cell therapy's fitness and its ability to infiltrate into solid tumors. In parallel for a potent second-generation therapy, Dr. Jonus will engineer Vd1 gd T cells to 1) express receptors that help them better recognize neuroblastoma and 2) evade immune recognition so that the therapy is not killed after being infused into a patient.

The first year 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.

Based on progress to date, Dr. Spear was awarded a new grant in 2026 to fund an additional year of this Fellow grant.

Neuroblastoma is a devastating pediatric cancer, with only 50% survival in aggressive "high-risk" disease. Survivors are burdened with life-long side effects from chemotherapy and radiation. Newer therapies, such as cancer vaccines, provide an opportunity to mobilize a patient's own immune system to find and destroy cancer cells. Identifying the unique genetic signature of an individual patient's tumor allows scientists to formulate a personalized vaccine to stimulate the immune system to recognize tumor-specific mutations, called "neoantigens". Dr. Spear has developed a new tool to identify these unique genetic signatures (neoantigens) and test the effectiveness of the neoantigen vaccine in modes. These findings will lay the groundwork to develop a clinical trial using personalized vaccines for high-risk neuroblastoma and other pediatric cancers.

This first 2 years of this grant were funded by and named for the Arden Quinn Bucher Memorial Fund, a St. Baldrick's Hero Fund. Arden’s intelligence, empathy, and dynamic personality charmed everyone and is now her legacy. Before her neuroblastoma diagnosis on October 11, 2007 at age two, she happily played with boundless energy and imagination. Even throughout her difficult months of treatment, Arden bravely managed to keep smiling and learning. This fund supports St. Baldrick’s mission: funding the most promising research, wherever it takes place to provide kids fighting cancers less toxic, more effective treatments allowing them to live longer, healthier lives.

The 3rd year of this grant is funded by Allied World, a global provider of insurance and reinsurance solutions.

Despite remarkable improvements in treatment for children with some types of cancer, pediatric brain tumors remain an area that desperately require more effective and low toxic therapy solutions. Dr. Jun Qi has formed a multi-disciplinary team to identify novel targets for pediatric brain tumors and develop new strategies to suppress the targets for patient treatment. Using a chemical strategy, Dr. Qi and his team aim to disrupt the functions of these targets to effectively inhibit brain tumor cell growth and block tumor progression in the models that resemble the real disease. The study focuses on improving on-target effect and, more importantly, on getting these potential drug candidates into the brain. The proposed study will translate from bench to bedside for patient care and result in a novel therapeutic strategy with significant improvements in survival and reduced morbidity for pediatric brain tumor patients to fulfill the mission of St. Baldrick's Foundation.

This grant is named for the Pray for Dominic Hero Fund. The fund was established in honor of Dominic Liples who lived with joy. He is remembered for compassion and determination while he faced his own difficult battle with a rare and aggressive brain cancer. The Pray for Dominic fund carries on Dominic's legacy of joy and hope by funding research for high-grade gliomas.

The study of genetic disease of cancer predisposition has served as a model for understanding cancer in general. Fanconi anemia is a rare genetic disease of failed blood production and cancer proneness, including leukemia and head and neck cancer. The genes and encoded proteins participate in DNA repair. However, an examination of cancer databases of DNA sequence shows that Fanconi genes are mutated in up to 30% of all head and neck cancers in non-Fanconi patients. Dr. Kupfer and colleagues have studied one particular mutation that resides in the Fanconi FANCD2 gene that interrupts its protein binding to another important gene BLM, which also participates in DNA repair. This proposal will seek to study the normal function of the FANCD2-BLM interaction in the cell and the consequences of its disruption. Dr. Kupfer also seeks to identify ways disruption of the normal pathway will render cancers vulnerable to molecular targeting to improve therapeutics.

Brain tumors are the leading cause of cancer related death in children. The outcomes for high-grade gliomas in children are dismal. Chimeric antigen receptor (CAR) T cells are genetically engineered cells programmed to target cancer cells with high precision. The application of CAR T cells in brain tumors in children is still limited compared to leukemia. One challenge is that CAR T cells need multiple hits to kill brain tumor cells compared with leukemic cells, where a single hit is sufficient. Dr. Abu Arja and team discovered a subset of CAR T cells that are more potent and can more proficiently kill brain cancer cells by increasing their lethality, making a second hit unnecessary. In this project, Dr. Abu Arja is studying the cellular program of this unique subset of potent killer CAR T cells to better understand why they are superior killers. Dr. Abu Arja plans to use these findings to genetically engineer new enhanced CAR T cells to eliminate tumors in children with brain cancers.

The first year of 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”.

Ewing sarcoma (EwS), the 2nd most common childhood bone sarcoma, is an aggressive tumour that primarily affects children, adolescents, and young adults. When EwS tumor cells spread to other parts of the body, known as metastasis, survival is drastically diminished to only 15-20%, which has not changed for decades. Immunotherapy empowers a patient’s own immune system to attack cancer, which has tremendous promise as an alternative to chemotherapies that are often toxic, especially to a growing child. Dr. Sorensen and his team recently identified a protein that is highly expressed on the surface of EwS cells, while showing only minimal to absent expression in normal tissues, nominating IL1RAP as a very promising therapeutic target. With their collaborators at the University of Pittsburgh, they have identified specific antibodies binding to IL1RAP and have engineered these antibodies to be conjugated to a drug that kills EwS cells potently. In this project, they will perform the extensive validation of these compounds to enable the design of early clinical trials for the treatment of EwS. This Better Ewing Sarcoma Therapies (BEST) grant is supported by a unique partnership of funders through the St. Baldrick’s Foundation: D-Feet Cancer, The Faris Foundation, The Shohet Family Fund for Ewing Sarcoma Research, an anonymous donor, and the family and friends of Martha Riedel.

Chimeric Antigen Receptor (CAR)-T cells have been clinically effective in patients with leukemias and lymphomas. Dr. Venkataraman’s goal is to bring similar success in treating a fatal brain tumor in children called DIPG (Diffuse Intrinsic Pontine Glioma). A major obstacle in treating brain tumors with CAR-T cell therapy is a lack of antigens which are tumor specific, or which are absent on normal vital tissues that can lead to off-target toxicities. To overcome this risk, Dr. Venkataraman and colleagues have successfully generated and tested the functionality of a novel “logic-gated” CAR-T cells targeting two distinct antigens, CD99 AND B7H3 that are highly expressed on DIPG but present singly on certain normal cells. This gated “AND” CAR-Ts will have full-activation against DIPG cells having both the antigens while sparing the single antigen expressing normal cells and will now investigate the safety, preclinical efficacy of these CAR-T cells against DIPG and evaluate its translational relevance to DIPG patients.

Children, adolescents and young adults with recurrent or refractory Osteosarcoma have a very poor prognosis, with a dismal 6mo overall survival of less than 5%. Presumably, this poor prognosis is in large part secondary to the development of resistance to chemotherapy and radiation. More recent studies employing therapies that release and activate the patients’ immune cells, called T-cells, and even targeted T-cells have not improved this poor prognosis. Dr. Cairo proposes to investigate novel and innovative methods of combinatorial immunotherapy to circumvent known mechanisms of resistance. Together with colleagues, he proposes to investigate at the bench (in the laboratory) and in models with osteosarcoma alternative methods of combination immunotherapy including natural killer cells (NK) that we have been engineered in the laboratory to also circumvent mechanisms of resistance and to additionally express a single or dual target that are present on the osteosarcoma cells.

They further plan to investigate the efficacy of adding other immunotherapies to enhance the function and persistence of these targeted NK cells with antibodies, and two different NK activating cytokines. They will also investigate the optimal combination of this immunotherapy in children, adolescents and young adults with recurrent or refractory osteosarcoma to determine the safety and efficacy of this approach. Finally, Dr. Cario and team will determine what are the genetic and immune mechanisms of resistance after these novel combinatorial immunotherapy approaches utilizing state-of-the-art laboratory techniques. The goal of this grant is to develop novel combinatorial immunotherapy that will significantly increase the overall survival in children and adolescents with poor risk osteosarcoma.

To make a significant impact for kids fighting osteosarcoma, five funders have banded together with St. Baldrick’s to support this grant – The Helping Osteosarcoma Patients Everywhere (HOPE) Super grant supported by Battle Osteosarcoma, the Faris Foundation, the Zach Sobiech Osteosarcoma Fund of Children’s Cancer Research Fund, the Children’s Cancer Fund NY (supporting the Maria Fareri Children’s Hospital and New York Medical College) and Nationwide Children’s Hospital.

CAR-T cell immunotherapies, treatments that use T cells constructed to recognize tumors and kill them, revolutionized how doctors treat children with B cell leukemia (B-ALL). These killer T cells recognize a specific protein expressed on the surface of the leukemic cells. Unfortunately, leukemia frequently relapses and often finds ways to "switch off" the expression of this protein, making T cells unable to track and kill them. This notion is called "antigen escape," as the tumor finds a way to escape the immune treatment. Dr. Aifantis plans to identify ways to avoid antigen escape by boosting the expression of the surface recognition protein. The study aims to validate such mechanisms in an organism using CAR-T cell models and sequencing patient cells. At the same time, Dr. Aifantis will design screens that will help identify surface antigen-specific regulators, so researchers can one day create combinatorial protocols using CAR-T cells and targeting specific antigen surface expression regulators.

Although germ cell tumors are the most common solid tumors of adolescents and young adults, care and progress has been hampered by fragmentation between pediatric and medical oncology. No major breakthrough in therapy has occurred for over 4 decades, and limited molecular understanding hampers the ability to tailor therapy or discover new drugs that could change outcomes. Through collaboration and joint projects and initiatives, the Malignant Germ Cell Tumors International Consortium (MaGIC) of the world’s leading scientists and clinicians from both pediatric and medical oncology has been able to make some important advances in clinical trial design and biologic understanding of this cancer: a new molecular test to detect relapse, a new molecular test to assign risk group and several jointly designed adolescent and young adult clinical trials using novel drugs and treatment approaches. Funds administered by Dana–Farber Cancer Institute.

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: 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: 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.

Acute Lymphoblastic Leukemia (ALL) are aggressive cancers of B- and T- immune cells. ALL is most common in children but also affects adolescents and young adults. 90% of childhood ALL is curable. However, ~10% of children and ~30% of adolescents and young adults with ALL are not cured. To combat hard-to-treat ALL, Dr. Swaminathan will harness the body’s natural anti-cancer defense mechanism: a type of immune cell called a natural killer (NK) cell. He will also find defective NK cells in children with ALL. Those with fewer defective NK cells tend to survive longer and spend more of their lives free from disease compared to patients with high levels of abnormal NK cells. These findings will inform the development of NK cells as affordable therapies to cure pediatric ALL.

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.

Atypical teratoid/rhabdoid tumors (AT/RT) are the most common malignant brain tumors of infancy. Standard therapies lead to severe toxicities and poor overall survival. Dr. Raabe aims to identify novel therapies to reduce toxicities and improve survival. Dr. Raabe and colleagues found that cancer cells rely on activation of the integrative stress response (ISR) to maintain cell equilibrium and survival. However, if the ISR is activated too intensely or for too long, cells undergo apoptosis and die. Paxalisib and gemcitabine are medications that induce considerable cell stress, further activating the integrative stress response, and extending survival in models of AT/RT. Dr. Raabe is investigating how these medications act together to target cell stress pathways and their impact on survival in models of AT/RT. The findings will translate directly through the International Pacific Pediatric Neuro-Oncology Consortium (PNOC) into a new clinical trial treating children with relapsed or refractory AT/RT.

This grant is named for Hannah’s Heroes, a St. Baldrick’s Hero Fund created in honor of Hannah Meeson and pays tribute to her fight by raising awareness and funding for all childhood cancers because kids like Hannah “are worth fighting for.”

Analyzing the genetic code of each patient's cancer enables researchers to identify specific errors that fuel the cancer in that patient. While the genetic code of patients in the clinics analyzed, it is currently difficult to analyze each patient in the context of all other patients. The Treehouse Consortium enables real-time sharing of pediatric cancer data generated by studies and clinical trials across the world, uniting data from diverse sources into a compendium of unprecedented scale. This group developed and made available to others methods to compare each child's cancer against over 12,000 childhood and adult tumors to improve treatment decisions. Fund administered by University of California, Santa Cruz.

Based on the progress to date, Dr. Lindquist was awarded a new grant in 2025 to fund an additional year of this Fellow grant.

Children with the brain tumor ependymoma have high relapse rates and poor long-term survival. Treatment options for ependymoma are limited and there is no known effective chemotherapy. Dr. Lindquist is working to make a new model of this tumor, to study how the tumor forms and grows, and to test new therapies in this model and patient-derived tumors. The ultimate goal is to identify new therapies that will extend the lives of children with ependymoma.