Grants Search Results

A subset of deadly pediatric cancers, known as sarcomas, are caused by a mysterious, mutated protein called CIC::DUX4. Treating this disease is very difficult, in part because little is known about its biology. The teams of Dr's. Wood and Hendrickson have assembled the world's best collection of CIC::DUX4 laboratory models, using them to discover that CIC::DUX4 sarcomas require a protein called COP1 for their survival, whereas normal tissues in bodies do not. Dr. Wood and colleagues will perform studies to understand how well future drugs targeting COP1 will work as therapies for this deadly disease while defining why this disease needs COP1 in the first place. Together, these studies will identify a highly promising new therapeutic strategy for CIC::DUX4 sarcoma while also revealing insights that could lead to additional new types of drugs for this disease.

Acute myeloid leukemia (AML) is harder to cure than most other types of childhood leukemia and lymphoma. Treatments are toxic and require patients and their families to spend up to a year in the hospital. Childhood AML survivors often have serious side effects later in life from their treatment. We need new treatments for AML that are less toxic and more effective. AML is often caused by mutations in a protein called N-Ras that tell the leukemia to grow and divide much more quickly than healthy tissue. If we could shut down this abnormal N-Ras signaling, it would stop the leukemia from growing. Unfortunately, no approved drugs exist that target mutant N-Ras proteins. Dr. Decker and his colleagues are testing a new drug called ABD778 that selectively blocks the growth of AML cells with mutant N-Ras. The results of this research could move drugs like ABD778 closer to the clinic and pave the way for new treatments for childhood AML.

This grant is named for the Cody Thompson Memorial Hero Fund. Cody spent much of his childhood in the hospital. As an adult he embraced the health and happiness of children and was an avid St. Baldrick's volunteer. This grant is a tribute to the compassion he carried throughout his life.

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 2025 SBF-AACR Award for Outstanding Achievement in Pediatric Cancer Research went to Dr. Charles Mullighan at St. Jude Children's Research Hospital, Inc.. Dr. Nicole Michmerhuizen at the University of Rochester Medical Center received the 2025 research grant. Dr. Michmerhuizen's research interest is in Acute myelogenous leukemia (AML).

Teens with leukemia go through tough treatments that make them feel tired and weak, so they spend a lot of time sitting and lying down, which can make side effects worse and put them at risk for chronic diseases like diabetes. Dr. Ivory is testing ReSeT, a program she developed for teenagers getting leukemia treatment to interrupt sitting time with short exercise breaks that will likely improve their lifestyles, heart health, and quality of life. Over 10 weeks, each teenager will use a Fitbit, health coaching, and an app-based support group to slowly increase their activity. After testing ReSeT in 30 teenagers to see if they can do it and what they think, she will fine-tune ReSeT and test it again in 10 more teenagers and compare how they do with 10 teenagers who didn't get the program to see if the program works. The goal is to use small behavior changes to help teenagers with cancer be more active during and after treatment to improve their lifelong health.

Each year, approximately 1,000 Americans aged 20 years or younger are diagnosed with acute myeloid leukemia (AML). Currently, no drugs can eradicate all AML cells in pediatric patients, and cells remaining after treatment often cause disease recurrence and poor survival. Dr. Li and colleagues have found that two mitochondrial enzymes that function in energy metabolism, known as DHODH or SDH, shield leukemia cells from eradication by immune cells. Dr. Li will use models relevant to pediatric AML to ask how these factors block anti-cancer immune responses and test effectiveness of a first-in-class leukemia cell-specific DHODH inhibitor combined with existing immune therapy in eradicating AML. If successful, this study will lead to development of new anti-leukemia drugs that could approach a cure for childhood AML.

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).

Vincristine is a chemotherapy drug commonly used to treat cancer in children and young adults, but it can cause vincristine-induced peripheral neuropathy (VIPN), a side effect that leads to numbness, pain, weakness, and difficulty with balance. These symptoms can severely impact daily life and may require chemotherapy dose reductions or discontinuation, potentially affecting cancer treatment outcomes. Currently, doctors use a grading system to assess VIPN severity, but this method is not sensitive enough to detect early symptoms and is difficult to implement consistently. Therefore, there is a need for a more reliable and accessible way to identify VIPN early. Dr. Belsky will utilize a potential solution of a blood test to measure neurofilament light chain (NfL), a substance released during nerve damage. Dr. Belsky and colleagues will explore whether NfL levels can detect VIPN in children and young adults to improve the ability to monitor nerve damage, enabling doctors to adjust treatments earlier, optimizing cancer care.

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!

High-grade glioma (HGG) is an aggressive brain tumor treated with surgery and radiation/chemotherapy. With such aggressive treatment, most children are not cured. Infant-Type Hemispheric Glioma (IHG) has a better cure rate than other types of HGGs. Commonly diagnosed in patients younger than 1, IHGs are large tumors and occupy half of a baby's brain. Surgery is often complicated with life-threatening bleeding in the brain with severe adverse effects further compounded by chemotherapy. Therefore, though IHGs have favorable cure rates, they require more effective and less toxic therapy. IHGs have genetic defects called receptor tyrosine kinase fusion, which are targetable by medicines called tyrosine kinase inhibitors (TKI), commonly used in adults. When used in IHG, tumor size is reduced without surgery or chemotherapy. Dr. Bagchi's clinical trial will treat based on the tumor's genetic defects using TKI and integrate quality of life measures so children survive & thrive.

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.

One in three children with newly diagnosed cancer has unmet resource needs such as food, housing, transportation, or utilities. These social needs increase during cancer care and are linked to worse outcomes for children and their parents. Government benefits such as the Supplemental Nutritional Assistance Program (SNAP or food stamps) improve child and maternal health outcomes. Dr. Umaretiya will conduct a pilot study using ASSIST, (a benefits navigator intervention to help families enroll and stay on government benefits such as SNAP) among 40 families of children with newly diagnosed cancer and test whether it is feasible and acceptable to families, allowing to identify barriers and facilitators to use ASSIST intervention.

The most common source of pain when treating children is needles. Avoiding painful procedures is a cause of stopping treatment for children with cancer in Low and Middle-Income Countries (LMICs). Dr. McNeil and colleagues will utilize an evidence-based care bundle to reduce needle pain based off of past results used in hospitals with LMICs with high patient and parent satisfaction which reduced pain by a significant amount. Keeping in mind the large differences in resources and cultures between the different hospitals, Dr. McNeil and colleagues will be able to understand the key features of using the care bundle in different hospitals. Dr. McNeil's team will identify different adaptations each hospital uses and study the ability of a hospital to continue to implement care.

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.

In the past decade, new therapies that train the immune system to recognize and kill cancer cells have revolutionized cancer care. Unfortunately, cancers arising from connective tissue like bone have not responded to these immunotherapies. Despite almost four decades of trialing/testing progressively more intensive chemotherapy, outcomes for osteosarcoma (the most common bone tumor) remain dismal once it has spread beyond the initial site. Dr. Smith wants to understand why these immunotherapies have failed by studying a model closely resembling human osteosarcoma. Based on his findings, Dr. Smith will test novel immunotherapies to prioritize the next generation of osteosarcoma human clinical trials.

This grant funds a student to complete work in pediatric oncology research for the summer. B-cell acute lymphoblastic leukemia (B-ALL) is the most common pediatric cancer, affecting over 3,000 children in the U.S. annually. A groundbreaking treatment called CAR T-cell therapy involves collecting a patient's immune cells, engineering them to recognize and kill cancer cells, and reinfusing them into the patient. While effective for many, CAR T-cell therapy often fails over time because the engineered cells can "burn out" or cancer cells change to avoid detection. To solve these problems, Dr. Pulsipher's lab is developing a smarter CAR T-cell system that uses multiple signals to activate, reducing burnout, and targets more than one cancer marker, making it harder for the cancer to hide. If successful, this approach could significantly reduce relapses in B-ALL patients, offering them a better chance at long-term remission. This work is being completed under the mentorship of Dr. Michael Pulsipher.

This grant funds a student to complete work in pediatric oncology research for the summer. Rhabdomyosarcoma (RMS) is the most common pediatric soft-tissue sarcoma in the United States, with approximately 400 new cases annually. Outcomes for high-risk RMS remain dismal, with three-year event-free survival rates as low as 20-30%. There is an urgent need for innovative therapeutic strategies that are more precise, have less toxicity and have significantly improved efficacy and survival benefits. A subset of RMS tumors have mutations in the RAS gene family, therefore, exploiting these mutations as therapeutic targets is an attractive and targeted therapeutic strategy. Dr. Ladanyi's research aims to test a recently developed RAS inhibitor (RMC-6236) in preclinical patient-derived disease models harboring mutations in RAS. This agent is in clinical trials for adult cancers with some RAS mutations. The St. Baldrick's Foundation Summer Fellow will help to generate the preclinical data necessary for a Phase 1 clinical trial testing RMC-6236 in children with RAS-driven RMS. This work is being completed under the mentorship of Dr. Marc Ladanyi.

This grant funds a student to complete work in pediatric oncology research for the summer. The Kohanbash lab is investigating how B cells, a critical part of the immune system, function in aggressive brain tumors in children. B cells produce antibodies to fight infections or cancer and carry unique surface markers called B cell receptors, which help them recognize and respond to specific threats. However, in cancer, B cells may behave unpredictably, sometimes helping the body fight against tumors, but in other cases, supporting tumor growth. By examining the specific features of B cell receptors in pediatric high-grade gliomas, they aim to better understand how these immune cells influence tumor development and progression. The goal of this project is to learn more about how the immune system responds to brain tumors in children and how altering these responses could lead to more effective treatments. Ultimately, this research hopes to improve therapies and outcomes for children diagnosed with deadly brain tumors, offering them better chances for recovery and long-term well-being. This work is being completed under the mentorship of Dr. Gary Kohanbash.

This grant funds a student to complete work in pediatric oncology research for the summer. Neuroblastoma, medulloblastoma, rhabdomyosarcoma, and desmoplastic small round cell tumors overexpress human epidermal growth factor receptor II (HER2) and cluster of differentiation (CD24) on their surface. Radioimmunotherapy targets those antigens using antibodies. Radioisotopes bound to those antibodies kill the cells. Radioimmunotherapy causes toxicity to normal tissues. Two step pre-targeted radioimmunotherapy reduces this by allowing excess antibody to clear from normal tissues before radiation is delivered. This is further optimized by the self-assembling and disassembling (SADA) antibody platform. Antibodies aggregate within the tumor and disperse outside of the tumor. Therefore, antibodies bound to the tumor remain in the body longer while excess antibodies are cleared faster. This study will compare two-step pretargeted radioimmunotherapy with the SADA platform to one-step radioimmunotherapy against tumors with HER2/CD24 via laboratory testing and a model study. This work is being completed under the mentorship of Dr. Nai-Kong Cheung.

This grant funds a student to complete work in pediatric oncology research for the summer. Currently, developing a cancer drug in the lab and bringing it to the clinic takes years or even decades to accomplish. Although many initial drugs are promising candidates, they ultimately fail because they have no effect, off-target effects, or are simply too toxic. One exciting avenue for cancer treatment involves modifying a patient's immune cells by adding chimeric antigen receptors (CARs) to their surface. Peptide-centric CARs (PC-CARs) can recognize protein fragments called peptides on the surface of cancer cells, which originate from cancer proteins within. Dr. Yarmarkovich's laboratory has developed PC-CARs against one of the most common childhood cancers called neuroblastoma and is currently pursuing clinical trials. To rapidly develop new PC-CARs, they have used new advancements in artificial intelligence to redesign their PC-CARs to recognize another upregulated neuroblastoma peptide called CHRNA3. Eventually, their research can be expanded to treating all cancers alike. This work is being completed under the mentorship of Dr. Mark Yarmarkovich.