Grants Search Results

Dr. MacDonald and colleagues have developed a new test that detects cancer cells in the blood and other fluids of patients with brain tumors. They have discovered that children with a particular type of brain tumor often have cancer cells in their blood. They can also use their test to follow how treatment changes the number of cancer cells in the blood to find out if the treatment being given is working to kill all the remaining cancer cells in the brain. This is the first time such a test has been able to do this using a simple blood draw. Dr. MacDonald and colleagues will use their test for children with other types of brain tumors. Dr. MacDonald and team will study the cancer cells in the blood to see why and how these brain cancer cells are still alive after treatment. This will help to identify the "steps" in which the cancer cells take to avoid being killed by the treatments being given and ultimately will then lead to new treatments targeting these steps to cure childhood brain tumors.

This grant is named for Hannah’s Heroes, a Hero Fund established to honor Hannah Meeson. At age 6 she was diagnosed with anaplastic medulloblastoma. After a relapse and additional 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.”

Children diagnosed with a brain tumor called diffuse midline glioma (DMG) have no options for a cure. Dr. Ramakrishna and team at Stanford Medicine have developed a new treatment, called CAR T cells, for these children by training their immune system to find and kill cancer cells. Excitingly, children treated on a clinical trial with these CAR T cells have improved symptoms and reduced tumor sizes. Unfortunately, for some patients, tumors grew after treatment, suggesting a need to understand how to improve the treatment. An immune cell, called a myeloid cell, was identified surrounding treatment of the first patients. Dr. Ramakrishna and colleagues will seek to understand these myeloid cells in the context of CAR T cell activity in patients. Modeling these myeloid cells in the lab, Dr. Ramakrishna and team will test approaches to improve CAR T cell activity against DMG cells. This project will improve understanding of CAR T cells in patients and develop new treatments for children with these devastating brain tumors.

Sneha Ramakrishna, MD, is a pediatric hematology-oncologist at Stanford Medicine Children’s Health and an assistant professor - University Medical Line in Pediatrics - Hematology & Oncology at Stanford Medicine.

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.

Children with a brain tumor diagnosis face the daunting prospect of surgery, radiation and chemotherapy. While these treatments can be successful, some brain tumors continue to grow and spread in the brain, which can make them impossible to treat. In addition, current treatments can adversely impact a child's cognitive, emotional and physical well-being. New treatments against the most aggressive brain tumors to reduce the burden of tumors in children and increase their quality of life are needed. Dr. Stewart and colleagues will develop new technologies and test new drugs that will allow to rapidly transplant patient tumor cells into models to make hundreds of model avatars that can then be treated with a panel of drugs to identify the most effective treatments for a child's specific tumor, focusing on very aggressive tumors first. Dr. Stewart and team expects this information will be used to help guide doctors decisions on the best treatment options to eliminate the child's tumor and minimize side effects.

This grant is funded by and named for the Kai Slockers Pediatric Cancer Research Fund. Kai was diagnosed at 2½ with Atypical Teratoid Rhabdoid Tumor (ATRT), a rare and very aggressive brain cancer. Within two weeks of diagnosis, he passed away, a mere 3 months shy of his third birthday. When Kai took his last breath, the cloudy sky opened up with a bright ray of sun that streamed through the windows of his hospital room – the darkness of the disease was replaced with the light of hope and the peace of no more suffering. Whenever the sun is out, his family thinks of him, assured that his legacy of hope shines on. This Hero Fund was created in his memory and will support research to help other kids with cancer have a better chance to fight and survive. It has a special focus on brain tumor research, specifically treatments that could minimize the harsh effects of brain tumor treatment.

Ewing sarcoma commonly appears in a bone among patients between 10-20 years old. About 25% of patients present with a clinically detectable metastatic disease. Despite aggressive chemotherapy and radiation, almost no improvement has been seen in patients with metastatic disease (80% mortality). The failure to stop Ewing's sarcoma metastasis is partially due to the lack of understanding about the molecular pathways that regulate its spreading. Dr. Yang and colleagues will study several upstream regulatory genes of TWIST1 in Ewing sarcoma metastasis and test whether drugs targeting these genes will block metastasis with higher specificity and fewer side effects than conventional therapy. In the long term, this research will lead to novel therapeutic regimens for Ewing sarcoma metastasis.

This grant is named for Kelly Bielaski's Wish for Ewing Sarcoma Research, a St. Baldrick's Hero Fund. Kelly passed in December 2025 at the age of 29 after an almost 2-year battle with Ewing sarcoma. She wished her journey would help change the future for others facing Ewing sarcoma. Through her fund and this research, Kelly's love for life lives on.

Brain cancer is the leading cause of cancer-related deaths in children, highlighting the urgent need for more effective treatments. While chimeric antigen receptor (CAR) T-cell therapy has transformed outcomes for children's blood cancers, it has shown limited success in brain tumors. Dr. Huang and colleagues will initiate a phase I trial in children with HGG and DIPG to assess safety and immune effects utilizing the understanding that CD70, a protein driving tumor growth, is a promising CAR T-cell target for high-grade gliomas (HGG) and diffuse intrinsic pontine gliomas (DIPG).

Spitz tumors are rare skin growths that occur in children and share some features with melanoma, a type of skin cancer. However, Spitz tumors often behave differently than melanoma, with many appearing harmless even if they spread to nearby lymph nodes. This makes diagnosing and treating them particularly challenging. Some Spitz tumors can resemble melanoma in their aggressive behavior, while others remain indolent, causing uncertainty about how they will progress. This research focuses on understanding two key aspects of these tumors: First, Dr. Boris and colleagues aim to determine whether Spitz tumors spread widely and early in their development. Second, they will investigate whether the immune system plays a critical role in controlling these tumors. Spitz tumors often contain unique genetic changes leading to fusion proteins, which may act as signals that the immune system can recognize and target. To study this, Dr. Boris will collect blood samples from children with Spitz tumors and analyze them for traces of tumor DNA, which could indicate how widely the tumors have spread and if they persist in the body after surgery. Additionally, they will study how the immune system interacts with these tumors by identifying specific markers on the tumor cells and testing whether the patient’s immune cells can recognize and respond to them. This multidisciplinary team includes experts in melanoma, pediatric dermatopathology, and immune system research, all of whom are dedicated to improving the care of children with Spitz tumors. By uncovering how these tumors behave and how the immune system interacts with them, the aim is to develop better tools for diagnosing and monitoring these tumors, and reduce the need for invasive surgeries.

This grant is funded through a partnership between the St. Baldrick’s Foundation and the Melanoma Research Alliance.

A child diagnosed with rhabdomyosarcoma (RMS) today is subjected to essentially the same therapy used fifty years ago. While Drs Mosse and Maris’s research programs have traditionally focused on neuroblastoma (NBL), both labs have recently extended their research programs to other pediatric solid tumors with high unmet need. Here they propose two parallel and complementary projects designed to create new immunotherapies for RMS, with Project 1 focused on the discovery of new immunoncology targets and cellular therapies, and Project 2 focused on the IND-enabling studies of a novel antibody-drug conjugate (ADC) directed against ALK for fusion positive RMS. This grant is supported by Alice's Arc US Inc. Alice's Arc operates in the UK and USA and is dedicated to funding research into finding a cure and less harsh treatments for rhabdomyosarcoma. The charity has created a global community of families, scientists and doctors coming together to help achieve this mission. Families are at the heart of the work and they can choose to create an Arc in their child/young person's name to represent their experience with rhabdomyosarcoma. This enables funds to be pooled for research, powerful advocacy and a long-term, sustainable platform to grow the charity and fund a pipeline of rhabdomyosarcoma research.

The recent development of therapies that stimulate the immune system to eliminate cancer has transformed treatment options for many patients. However, these therapies have generally been less successful in treating childhood cancers, in part because cancers in younger patients typically have acquired fewer of the genetic alterations that can be recognized by T cells, the immune cell type most commonly used for cancer treatment. This project aims to harness the cancer clearing functions of macrophages, a distinct immune cell type that can recognize and kill even those cancer cells that carry few genetic alterations. Dr. Kamber and colleagues will focus on identifying strategies that unleash macrophage anti-cancer functions in the context of Burkitt lymphoma, an aggressive form of lymphoma that is among the most common types of cancer in children.

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.

In some types of cancer that affect children and teenagers, there are special proteins called 'fusion oncoproteins' that play a big role. These proteins are made when a gene called MLLT10 gets mixed up with other genes. These cancers are very dangerous and don't respond well to treatments available now. Dr. Lui and colleagues research is focused on the most prevalent MLLT10 fusion oncoproteins, common in a type of cancer called T-cell acute lymphoblastic leukemia (T-ALL) in kids and young adults. Findings show that this fusion protein makes groups of biomolecules called 'condensates,' which can mess up how cells read and use their genetic instructions. Dr. Lui believes that by studying these MLLT10 fusion oncoproteins in detail, they may learn how they change cells and find ways to stop them. Dr. Lui also believes if they can figure out how MLLT10 fusion oncoproteins work, it may also help to understand other similar fusion proteins. That knowledge could help develop better treatments for these kinds of cancer.

This grant is funded by and named for Emily Beazley's Kures for Kids Fund, a St. Baldrick's Hero Fund. At the age of 8, Emily was diagnosed with Stage III T-cell lymphoblastic non-Hodgkin’s lymphoma and battled through three relapses. Her family prayed for a miracle but discovered Emily herself was the miracle, inspiring a community to come together to show love and change lives. She had a dream of starting a foundation to fund research and named it “Kures for Kids”. Today, Emily's family and friends carry on her dream and her mission in her memory.

It is known that children with cancer have higher rates of hospitalization, ICU admission, and death than children without cancer and COVID-19. Children with cancer and COVID-19 also frequently have changes in their chemotherapy. Yet, critical data is lacking regarding COVID-19 in children with cancer and guidelines about how to manage these vulnerable children. Dr. Johnston and collegaues will leverage the national registry of children with cancer and COVID with data on >2,400 children from >100 institutions to examine (1) how the clinical course of children with cancer and COVID-19 compares to earlier in the pandemic, (2) how the clinical course of COVID-19 in children with cancer is impacted by vaccination and antiviral therapy, and (3) physician and healthcare systems factors that influence COVID-19 management. Dr. Johnston will use that information, literature review, and expert discussion to inform an expert panel tasked with developing guidelines for management of COVID-19 in children with cancer.

The ultimate goal of this project is to define the safest, most effective therapies for children and young adults with Langerhans cell histiocytosis (LCH), which aligns with St. Baldrick's mission to find cures for childhood cancers and give survivors long and healthy lives. LCH is a blood cancer most common in children that creates destructive inflammatory lesions that can be fatal. LCH is caused by mutations activating the MAPK growth pathway in developing blood cells. Current front-line therapy fails to cure over 50% of patients with disseminated disease, and safe and effective options for subsequent therapy is not known. High-dose chemotherapy can be effective, but is toxic. MAPK inhibitor therapy alone does not appear to be durable based on early trials. Dr. Allen and colleagues hypothesize that MAPK inhibition will make cells more sensitive to chemotherapy. Dr. Allen will therefore test safety and efficacy of a new approach of combining chemotherapy with targeted MAPK inhibitor therapy.

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.

Cancer patients take life-saving drugs that, unfortunately, can result in peripheral nerve damage. For example, many patients receiving cisplatin experience permanent hearing loss. There is one therapy that has been approved to mitigate cisplatin-induced hearing loss, however, the reduction in hearing loss is modest (< 30%) and this mitigating treatment is associated with poorer overall survival rates due to inhibition of cisplatin's cancer-fighting properties. Thus, it is approved for low-risk pediatric patients only. To develop a better alternative, Dr. Rutherford and colleagues are testing novel compounds they have developed at Washington University, which have shown to protect the ear from noise trauma. With hearing tests and with anatomical measurements of the cochlea, Dr. Rutherford will attempt to prevent hearing loss following cisplatin treatment in models. After this innovative project proves successful, subsequent model studies will determine if Dr. Rutherford's therapy inhibits cisplatin's cancer-fighting role.

Medulloblastoma (MB) is an aggressive childhood brain cancer that is fatal in 40% of patients. MB shows abnormal activation of growth pathways in tumor cells which help them to grow. Dr. Ganesan's studies show that there are T cells (immune cells) within MB and they have potential to kill tumor cells. However, immunotherapy that boosts the activity of these T cells have not been successful and it is not known why. The goal of this project is to understand why immunotherapy has not worked in MB and whether immunosuppressive myeloid cells contribute to this restraint. Dr. Ganesan and colleagues will also study if combined treatment that inhibits the growth pathways (targeted therapy) and stimulates the T cells/immune system (immunotherapy) may together lead to greater tumor killing in MB mouse models. To test if combined therapy would work in humans, Dr. Ganesan will coculture a 3D version of patient’s brain tumor with their own T cells expanded from their tumor. If effective, these studies may lead to new treatments for MB.

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.

The AACR-St. Baldrick's Foundation Award for Outstanding Achievement in Pediatric Cancer Research has been established to bring attention to major research discoveries to the pediatric cancer research community and to honor an individual in any sector who has significantly contributed to any area of pediatric cancer research, resulting in the fundamental improvement of the understanding and/or treatment of pediatric cancer. The recipient will nominate an emerging leader conducting research in the academic sector to receive a research grant. The 2024 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. Ching-Hon Pui at St. Jude Children's Hospital. Dr. Shawn Lee at National University of Singapore received the 2024 research grant. Dr. Lee's research interests are in pharmacogenomics, immunogenomics and leukemia.

Survivors of pediatric brain tumors have a high risk of medical problems that can negatively affect the quality of their lives. Particularly concerning are effects on brain development, including learning and emotional well-being, and metabolism, which can lead to obesity and muscle loss. There is an urgent need for tools that can better predict which children are most at risk so that they can be offered treatments to prevent these problems. Dr. Kann's and colleagues have developed medical imaging tools that use artificial intelligence on routine brain scans to track and predict 1) muscle weakness and malnutrition, and 2) brain development in children. Dr. Kahn and team will test these tools in large datasets from hospitals and clinical trials of pediatric brain tumor patients and survivors to predict the risk of these negative effects in each patient. The tools developed may be used in clinical trials to improve quality-of-life for childhood brain tumor survivors.

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.

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.

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.