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Ewing sarcoma is a deadly cancer that affects children and young adults. Dr. Gao and team know that these cancer cells depend on a special protein, ETV6, to stay alive. Certain natural fats inside the cell can weaken this protein and slow the cancer's growth. Dr. Gao and colleagues will learn exactly how these fats affect the cancer cells and will search for medicines that safely increase these fats to damage the tumor. Dr. Gao's research will open the door to new and gentler treatment options for children with Ewing sarcoma.

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.

Rhabdomyosarcoma (RMS) is an aggressive childhood cancer that resembles developing muscle but cannot mature. The most dangerous form is driven by the PAX3::FOXO1 gene fusion, which forces uncontrolled growth. Better treatments are urgently needed because current therapy cures fewer than one in three children whose cancer returns. Dr. Khan and colleagues research targets FGFR4, a protein expressed at very high levels in RMS but not in normal tissues. A highly specific antibody against FGFR4 enables precise delivery of drugs into cancers. Dr. Khan and team will use this antibody in two ways: (1) to create an antibody-drug conjugate that delivers a drug blocking EP300, a key partner PAX3::FOXO1 uses to activate cancer genes: and (2) to build an antibody-PROTAC shuttle that brings a protein-destroying drug into FGFR4-positive cells to eliminate EP300. By disabling EP300 only in cancer cells, Dr. Khan and team will halt RMS growth while sparing healthy tissue and pave the way for safer, more effective treatments.

Year 1 of this grant is made possible in part, through funding from The Maddox Potter Foundation and is named in memory of Maddox, who is the inspiration for the Foundation. “Mad Dog” was a beautiful son, devoted big brother, diligent student and accomplished junior golfer. He fought alveolar rhabdomyosarcoma bravely for a year, and his final request was that someone, somewhere, would learn something from him. The Foundation is committed to honoring Maddox’s request by supporting families facing childhood cancer, funding critical research, and connecting the game he loved to hope, purpose, and philanthropy.

MLL (Mixed Lineage Leukemia)-rearranged leukemias are one of the most prevalent childhoods leukemias and carries poor to intermediate prognosis. Current therapy is still inadequate for MLL-rearranged (MLLr) leukemias, highlighting the need to develop better therapeutics. A protein complex that helps to rearrange the packing of genomic DNA within the cell, the BAF chromatin remodeling complex, is vital for MLLr leukemia cell survival. In addition, loss of BAF complex has also been shown to boost anti-tumor immunity in previous published studies. Dr. Zhu and colleagues will target BAF complex leads to dual effects of eradicating leukemia cells, boosting anti-tumor immunity which synergizes to curb leukemia. Dr. Zhu and colleagues' research will identify a novel dual acting therapy for MLLr leukemia.

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.

Vincristine is a common and effective chemotherapy for children with leukemia, sarcomas, and brain tumors. Unfortunately, many children who receive it develop nerve damage that cause pain, tingling, and long-term disability. There are currently no treatments that prevent this side effect. To understand why this nerve damage occurs, Dr. Gomez Deza and colleagues use cell models (derived sensory neurons that closely mimic how children's nerves respond to vincristine). Using a large genetic screen, Dr. Gomez Deza and team will use a molecular regulator that functions as a key switch helping drive nerve injury. Blocking this pathway protects nerve cells in the lab, reduces pain-related behaviors in models, and does not interfere with vincristine's ability to fight cancer. Dr. Gomez Deza and colleagues will uncover how this pathway controls the harmful stress responses triggered by chemotherapy and will test whether inhibitors already in clinical trials for other uses could serve as protective treatments for children receiving vincristine.

Pediatric craniopharyngioma (CP) is a rare brain tumor (5-10% of cases). CP is located near vital areas such as the eyes and the hormone control center, and recurs after surgery in 50% of cases. Standard treatments (surgery and radiation) lead to serious issues and reduced quality of life for most patients. No drugs are available for treating CP. Dr. Lee and colleagues will target the Wnt signaling pathway, a system that cells use to communicate and control when they grow or divide in CP tumors when Wnt signaling is overactive. Dr Lee and team will test promising drugs for CP: FDA-approved drug pyrvinium and a newer, unapproved drug SSTC3. These drugs block Wnt signaling and are effective in other cancers. Dr. Lee and team will use CP tumor cells from patients and CP models, and measure how the drugs block Wnt signaling in cells determining if the drugs shrink tumors. Dr. Lee and colleagues will pioneer targeted therapies and reduce the need for harsh CP treatments.

Dr. Boelens and colleagues will study how the immune system recovers after a bone marrow transplant in children with leukemia. These transplants can cure cancer, but some children relapse or develop a serious complication called chronic graft-versus-host disease, where the donor immune system attacks the child's tissues. Dr. Boelens and team will focus on the thymus, an organ that trains new immune cells but is easily damaged by chemotherapy and radiation. Dr. Boelens and colleagues will measure how well the thymus works and how immune cells grow during the first year after transplant. Dr. Boelens will confirm their findings by working with many hospitals through a large pediatric transplant network. By finding early warning signs of poor recovery, Dr. Boelens and colleagues will help doctors identify children at high risk and guide more personalized care so that more children survive transplant and grow up healthy, with fewer long-term complications.

This year, neuroblastoma will account for approximately 15% of cancer-related deaths in children. Despite recent advances in treating high-risk neuroblastoma, nearly half of patients will relapse with no curative treatments. Thus, safe and effective treatments are urgently needed. Using recently developed tools to find new therapy targets at a large scale, Dr. Mabe and colleagues understand that neuroblastoma tumors require polycomb repressive complex 1 (PRC1). Dr. Mabe and colleagues will identify that a new class of drugs that blocks PRC1 activity can reduce neuroblastoma tumor growth. To determine the exact mechanism by which these drugs reduce neuroblastoma growth, Dr. Mabe's lab will apply sequencing technologies to assess how PRC1 changes the neuroblastoma tumors to become less like a cancer cell and more like nerve cells.

Neuroblastoma is one of the most common childhood cancers. Babies under 18 months often have tumors that can shrink on their own, while older children face more aggressive disease. This difference may be due to how the immune system works early in life. Infant T cells, the white blood cells that kill cancer, are more responsive and resist "exhaustion," a problem that limits current immunotherapies. Dr. Rowe and colleagues will study what makes infant T cells unique and use this knowledge to design improved, less toxic T-cell therapies for children with neuroblastoma and other hard-to-treat cancers. By discovering how early-life immunity naturally fights cancer, Dr. Rowe and colleagues will create safer treatments that cure more children and help survivors live long, healthy lives.

Many children with bone tumors cannot be cured. This is true in particular if the tumor has come back. Dr. Gottschalk and colleagues will use the immune system to fight cancer. In their approach, they will take immune cells from patients and train them in the laboratory to fight cancer. Dr. Gottschalk and team's training methods willl consist of inserting a gene into immune cells that are a called T-cells. Once the cells have completed training in the laboratory, they are given back to patients. A second gene is inserted into T cells so that the T cells can grow better in patients after infusion. Dr. Gottschalk and colleagues will figure out the best structure of the second gene and will perform laboratory studies to determine which T cell works best, resulting in selecting the best T cell product for a future clinical study for children with bone tumors.

Rhabdomyosarcoma (RMS) is a childhood cancer that affects muscle cells which attach to bones, affecting how a child moves, smiles, walks, and performs daily tasks. RMS can be very difficult to cure when it spreads. Children with metastatic or relapsed RMS have poor survival, and current treatments often cause serious long-term side effects including second cancer. New, safer therapies are urgently needed. Dr. Chen and colleagues will work with a gene called BRD1, which helps RMS cells move and invade other tissues. BRD1 controls another gene, MYOG, which is abnormally active in this cancer. When either gene is reduced, RMS cells become much less able to spread. Dr. Chen and colleagues will uncover how BRD1 and MYOG work together to drive metastasis and will identify the genes they regulate to serve as new drug targets. By revealing a new biological pathway that supports tumor spread, Dr. Chen and team will lay the foundation for more effective, less toxic treatments for children with high-risk RMS.

Dr. Ruff and colleagues will develop a targeted medical therapy for adamantinomatous craniopharyngioma, a brain tumor that affects children and causes lifelong vision loss, hormone problems, and cognitive impairment in the majority of survivors. Currently, the only treatments are surgery and radiation, which often cause irreversible damage to the developing brain, and despite this the tumor frequently comes back requiring repeated interventions. Combining two existing FDA-approved medications to shrink these tumors dramatically, Dr. Ruff and colleagues will study how likely these medications are to work in patients with this tumor. By analyzing tumor samples for the medication target and patient responses, Dr. Ruff and team will design a clinical trial that could transform care for children with this disease.

Leukemia is a leading cause of death in children. Exciting new treatment strategies direct immune cells against B acute lymphoblastic leukemia (B-ALL) cells, however they are not effective for many patients, particularly those with relapsed disease. Why these therapies work in some children but not others is not understood. There is evidence that leukemia cells protect themselves by creating an environment around themselves (the immune microenvironment) that inhibits immune cells. Data indicates that B-ALL cells may impair a process called emergency myelopoiesis (EM), inhibiting normal immune cell function, if the leukemia cells express a protein called Siglec15. Dr. Porter and colleagues will determine if B-ALL impairs EM, whether that is dependent on Siglec15 and if EM can be restored to improve the immune response to leukemia cells. Dr. Porter and team will demonstrate that impaired EM plays an important role in the cell's development.

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.

Acute myeloid leukemia (AML) is a high-risk blood cancer in children. Even after intensive chemotherapy and bone marrow transplant, small numbers of leukemic cells, called measurable residual disease (MRD), can persist and later cause relapse. Current hospital tests examine large mixtures of cells and can miss these dangerous clones, causing clinicians to underestimate a child's relapse risk. Dr. Furlan and team will apply advanced single-cell genomic technologies to profile tens of thousands of cells from each child, pinpoint rare residual leukemia cells, and define the programs that allow them to survive treatment. Using these insights, Dr. Furlan and colleagues will develop practical, more sensitive MRD assays that children's hospitals could implement to better identify which patients are truly in deep remission and which remain at high risk, guiding earlier intervention and improving long-term cure rates for children with AML.

This grant is generously supported by Super Soph's Pediatric Cancer Research Fund, a St. Baldrick's Hero Fund. Sophie Rossi was diagnosed with AML at 3 months of age. Throughout her courageous battle, she was always smiling, always joyful. This fund was created to honor her spunky, sweet spirit by funding research to find cures for AML and all childhood cancers.

Germ cell tumors can occur in children, adolescents, and young adults. Remarkably, these cancers begin in the fetus, before birth. About 10,000 new germ cell cancers are diagnosed annually in the US, and the incidence is increasing. An individual's genetic make-up, early development, and environmental exposures all affect the risk for germ cell cancer, but the underlying mechanisms are poorly understood, due in part to a lack of research models. Dr. Weiss and colleagues will use a model that accurately recapitulates germ cell cancers and will test how risk factors, including both genetic and developmental factors, promote germ cell cancers to study environmental factors like forever chemicals and pesticides that have been linked to increased germ cell cancer incidence. By testing risk factors and understanding how they act, Dr. Weiss and colleagues will identify new approaches to detect and prevent these cancers in pediatric and young adult populations.

The most difficult cases of Ewing sarcoma to treat are when it comes back after completing initial treatment. Dr. Brian Ladle and colleagues at Johns Hopkins University and the National Cancer Institute believe the immune system is capable of mounting a powerful immune response against Ewing sarcoma as an effective treatment. Dr. Ladle and team will identify the best immune targets in Ewing sarcoma and discover ways to activate the immune system against the most promising targets. These targets will be tested in their lab using models.

To make a significant impact for kids fighting Ewing sarcoma, five funding partners have banded together with St. Baldrick’s to support this grant – Advancing Cures for Ewing Sarcoma (ACES) award supported by the Sam Day Foundation, The Faris Foundation, Rutledge Cancer Foundation, The Shohet Family Fund for Ewing Sarcoma Research (a St. Baldrick’s Foundation Hero Fund), and Alan B. Slifka Foundation.

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).

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!

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.

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.

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.