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Ewing sarcoma (EwS) is a bone and soft tissue tumor that most commonly affects children and young adults (AYA). Patients with relapsed/refractory (R/R) EwS continue to face dismal outcomes. While chemotherapy backbones are effective in patients with R/R EwS, outcomes are insufficient, and there is a need to identify combination strategies with novel agents to improve outcomes. Dr. Ganapathi and colleagues have found that EwS is critically dependent on the menin protein, which cooperates with the fusion protein EWS::FLI1 to drive growth and metastasis. Lab and model studies show that blocking menin with Revumenib (FDA approved for pediatric leukemias) powerfully suppresses tumor invasion and metastasis. Dr. Ganapathi and lab will conduct an early phase clinical trial of Revumenib combined with chemotherapy. This combination will ultimately improve outcomes for patients with R/R EwS.

This grant is generously supported by JC Strong, a St. Baldrick’s Hero Fund, established in honor of Jackson Kalbhenn to celebrate his survivorship. Jackson was 5 years old when he was diagnosed with Ewing sarcoma. After 14 cycles of aggressive chemotherapy, 69 nights in the hospital, and a resection on his left leg, his treatment was complete. Today, Jackson shows no evidence of disease and enjoys an active life filled with all things sports related. Through JC Strong, his family hopes to make meaningful difference for every child and family facing Ewing sarcoma.

Childhood leukemia happens when certain blood cells grow out of control. Dr. Wang'ondu and colleagues will focus on a protein called IKZF1, a transcription factor that helps young immune cells develop normally. In some children, IKZF1 is altered in a way that disrupts this process and makes leukemia more likely to form. Children whose leukemia has IKZF1 changes often have worse outcomes with current treatments. Dr. Wang'ondu will study how a specific change called IKZF1 N159Y disrupts normal immune cell functions and how Dr. Wang'ondu and team can target it with new types of drugs. Dr. Wang'ondu will work to understand how changes in transcription factors like IKZF1 cause leukemia and will help develop more effective treatments for children affected by these changes.

Dr. Shen will develop a new immune-based approach to treat diffuse midline glioma, a deadly childhood brain tumor with very limited treatment options. Dr. Shen and colleagues' work will focus on helping the body's natural killer immune cells better recognize and destroy tumor cells while sparing healthy brain tissue. Dr. Shen will test whether standard radiation therapy can make tumor cells more visible to these immune cells, thus improving treatment effectiveness and safety. Dr Shen's research will lay the groundwork for a more precise and less toxic therapy for children with this devastating disease.

This grant is funded by and named for Luke's Army Pediatric Cancer Research Fund. This Hero Fund was created in memory of Luke Ungerer who brought smiles and sunshine wherever he went with plenty to share with everyone. He battled a brain tumor with a positive spirit and inspired others with his courage in his short life. This fund intends to carry on Luke’s legacy of positivity with the hope that it will ripple across many lives for many years to come.

Despite advances in pediatric AML treatment, some leukemia cells can temporarily shut down and hide from therapy, later reawakening to cause relapse, the leading reason many cancers return after remission. Dr. Wang and colleagues seek to understand what allows these dormant leukemia cells to evade treatment and how they may eliminate them. Using gene-editing technology, Dr. Wang and team will work with a protein called DDX6, which acts as a key switch that keeps certain genes turned off in active leukemia cells. When DDX6 is lost, cancer cells enter a dormant, treatment-resistant state. This is a promising way to target these dormant cancer cells by blocking a signaling pathway called MEK which makes cells sensitive to chemotherapy. By uncovering how leukemia cells use dormancy to escape therapy and identify a way to target them, Dr. Wang and team's work will lay the foundation for new treatments to prevent relapse and improve long-term survival for pediatric AML patients.

Dr. Graves and colleagues will improve treatment for osteosarcoma, an aggressive bone cancer that frequently becomes resistant to chemotherapy. Cells that become resistant to chemotherapy often undergo adaptations to help them survive and recover. Dr. Graves will investigate how chemotherapy remodels iron usage and mitochondrial function to support survival in osteosarcoma cells. Early data suggest these cells become more dependent on energy production in the mitochondrial to survive. By impacting energy production, Dr. Graves and colleagues will test whether cancer cells become less resistant, grow more slowly, and will be less likely to spread. Dr. Graves will evaluate a new "multi-strike" treatment that delivers chemotherapy first, followed by carefully timed drugs that disrupt cancer cell metabolism before tumors can rebound. By identifying weaknesses in cells that survive chemotherapy and optimizing treatment timing, Dr. Graves and team will prevent tumor regrowth and improve outcomes in osteosarcoma.

Children, adolescents, and young adults (AYA) diagnosed with sarcoma, often face long-term health challenges commonly seen among older adults who have never been diagnosed with cancer. This may be due to a phenomenon known as accelerated aging, where cancer and its treatment cause patients to age faster than they should. Dr. Robinson and colleagues will explore factors that may affect accelerated aging and the impact of accelerated aging on an individual's physical function and quality of life. Dr. Robinson and team will assess age acceleration among sarcoma patients and survivors, ages 12-25, and study its association with their demographics, lifestyle, disease, and treatment. In doing so, Dr. Robinson and colleagues will guide the development of interventions to improve the long-term health and well-being of pediatric and AYA sarcoma survivors.

This grant is named for Madison Richards Mighty Research Marauders, a St. Baldrick's Hero Fund. It was established to celebrate Madison's extraordinary courage and resilience. Madison survived a harrowing ordeal with Ewing sarcoma in her chest cavity. Her extended treatment required more than 140 nights in the hospital and included a month-long coma while she fought West Nile Virus. Through every challenge, she faced each days with remarkable grace and poise. When surgery and treatment were complete, she returned to high school, completed an 18-month mission for her church, and graduated from college. Madison is a true example of making it through the storm and living life to the fullest on the other side.

Neuroblastoma (NB) is a common and deadly solid tumor in children, and despite receiving the most intensive treatments available, 50% of all children with high-risk disease die from this cancer. NB is aggressive in part because it has high levels of chemicals called polyamines (which are made from arginine/ARG). A drug called difluoromethylornithine (DFMO), blocks polyamine production. Dr. Turn and colleagues combine DFMO with decreased ARG to cure 1/3 of models with NB without using chemotherapy in their research. There are however, remaining tumors that have increased macrophages. Although macrophages can eat cancer cells, many of these macrophages are hijacked to help the tumor to grow despite decreased polyamines. Dr. Turn and colleagues will learn how macrophages do this and will find ways to target them to improve this therapeutic strategy in NB.

This grant is 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.

Medulloblastoma is the most common malignant brain tumor in children. While multimodal therapy for medulloblastoma at diagnosis has resulted in improved outcomes, there are very few options at the time of relapse, with overall poor survival. Preclinical data have shown anti-tumor activity with liothyronine (T3, activated thyroid hormone) in medulloblastoma models. T3 has safety data and dosing which are available for children. Dr. Ronsley and colleagues will translate these preclinical findings into a clinical trial, which will treat children and adolescents with relapsed medulloblastoma with T3 in combination with chemotherapy and evaluate both safety and efficacy and the role for monitoring with liquid biopsy.

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.”

Osteosarcoma is a bone cancer that affects children and adolescents. Unfortunately, its treatment has remained the same since the 1980's, and once the cancer spreads to other organs, less than 40% of the patients survive despite treatment. Dr. Aquino Lopez and colleagues will use the immune system to eliminate tumor cells. During an illness with a virus, immune cells called virus specific T cells (VSTs) eliminate infected cells by recognizing "viral signals". Dr. Aquino Lopez will use an artificial virus combination called CAdVEC to modify cancer cells and make them look like they are infected with a virus. Doing so, will trigger immune cells to eliminate the cancer cells.

Neuroblastoma (NB) is a deadly childhood cancer. Children with NB are classified as high-risk (HR) due to genetics or metastatic disease. Metastatic disease occurs when tumor cells (TC) leave the original tumor and travel to distant organs and develop into new tumors by a complex set of steps. Before tumor cells can form metastasis, they often go through a long period of dormancy (rest) to evade therapy. In NB, the bone marrow is the most common site of metastasis and relapse. The MYCN_TT is a unique model of NB, that spontaneously metastasizes to distant sites such as kidney marrow (equivalent to bone marrow). Dr. Anderson and colleagues will utilize the MYCN_TT model in understanding the molecular and cellular mechanisms underlying metastasis and dormancy, which will inform the development of novel therapeutic strategies for HR-NB.

Bone marrow transplantation (BMT) is an effective treatment for patients with blood cancers, utilizing healthy donor cells to fight diseases. However, these donor cells contain specialized immune cells (T cells) that can also attack and damage the patient's healthy organs, known as graft-versus-host disease (GvHD). Because both GvHD and anti-cancer effect (GvL) are mediated by the same T cells, it is hard to separate GvHD from the beneficial GvL. Current treatments use global immune-suppressive drugs to prevent GvHD, but they also reduce the ability of T cells to fight cancer, increasing the risk of cancer recurrence. Dr. Kim and colleagues will investigate if targeting VLDLR-PPARd signaling selectively eliminates GvHD without compromising the GvL in preclinical models of BMT.

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.

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.

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 a devastating childhood bone cancer. Doctors treat these pediatric patients with toxic chemotherapies, radiation, and surgery. Dr. Langdon and colleagues will develop targeted combination therapies to safely and effectively kill Ewing sarcoma cells. Dr. Langdon finds changing where proteins are normally found in cancer cells create potential new targetable vulnerabilities for Ewing sarcomas. Each vulnerability is thought of as a new "Achilles heel" for these cancers. Dr. Langdon and colleagues believe that combining two drugs - one which changes where proteins are normally found and one which targets the new vulnerability - will kill Ewing sarcomas. Dr. Langdon's team will look to why these drugs work so well against Ewing sarcomas and determine if they are truly safe and effective.

Brain tumors are the deadliest type of cancer that afflicts children. The ability of brain tumor cells to spread (metastasize) outside of the original tumor along the leptomeninges, the covering of the brain and spinal cord, is responsible for making many brain tumors so hard to treat. How cancer cells embedded in the leptomeninges survive, thrive, and resist best treatments is poorly understood. A better understanding of leptomeningeal metastasis is required to make new therapies that can meaningfully increase survival for children diagnosed with aggressive brain cancers. Dr. Reinecke and colleagues will create a way that can identify and screen potential therapies in a cell culture dish, thereby streamlining interventions they take to models of pediatric brain tumors. Dr. Reinecke and colleagues believe that establishing this preclinical platform has the potential to identify therapies that have a chance to positively impact the lives of children diagnosed with metastatic brain tumors.

The second year of 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.

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 everywhere in the world get cancer but their chances of surviving differ based on where they live. Disparities in childhood cancer diagnoses and survival have been described by sex and age, but there are gaps in this literature from countries with limited resources. The first goal of Dr. Force's project is to analyze how childhood cancer diagnoses and survival differ by sex, age, and world region, using data from the most comprehensive international collection of hospital cancer registries, and to assess potential underlying drivers of these disparities, which would be beneficial in identifying interventions to improve equity in childhood cancer outcomes. The second goal of Dr. Force's project is to compare childhood cancer data from hospitals and population-based cancer registries, to determine whether hospital data could be used to supplement information on childhood cancer burden where data is currently lacking in global models, better illuminating the disparities that exist globally.

Diffuse midline glioma (DMG), previously known as diffuse intrinsic pontine glioma (DIPG), is a deadly childhood tumor with no effective treatments. Dr. Li's project seeks to understand the genetic and epigenetic dysregulation of DMGs. Through cutting-edge single-cell analyses and advanced AI models, researchers aim to map the tumor's epigenetic landscape, identify key regulatory elements, and predict the function of risk mutations. This knowledge could pave the way for new targeted therapies and improve DMG outcomes.

The final year of this three-year St. Baldrick's Scholar grant is funded by and named in honor of the Hannah Levy Miller Fund. Hannah passed away from Diffuse Intrinsic Pontine Glioma (DIPG) shortly before her 12th birthday. She was an avid reader who loved dancing, playing the cello, and playing soccer. To honor her memory and create a lasting legacy, her family established the Hero Fund to support research for DIPG. This mission reflects exactly what Hannah would have wanted—to help other children and families facing this devastating disease.

The first year St. Baldrick's Scholar award is funded by and named for #Joe Strong 71, a St. Baldrick’s Hero Fund created in memory of Joe Purdue. Joe was a talented football player and cherished friend and son. He was diagnosed with DIPG shortly after graduating from high school, cutting short his plans to attend college. He is remembered for determination as he battled the most lethal form of brain cancer. #Joe Strong 71 carries on Joe's legacy by funding research for DIPG.