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The immune system not only fights infection, but can also fight cancer cells. Recently, doctors have been able to use patients' own immune cells to help treat their cancer. These immune cells can also attack the patient's normal tissues, which is harmful. Dr. Richman is working to learn how normal tissues might be protected while still allowing the immune cells to effectively kill the cancer cells.

Women who are diagnosed with cancer before 40 are often concerned about whether they will be able to have children in the future. Women can freeze their eggs or embryos (called fertility preservation) to help protect their fertility, but these services may not be widely available. Dr. Nichols has looked at how often women in North Carolina have children after cancer treatment and whether the health of their babies is different from women without cancer. She is further examining the use of fertility preservation after diagnosis and its association with birth rates and outcomes. This research will provide information to improve the long-term health of AYAs with cancer.

The EWSR1-FLI1 family of cancer genes causes Ewing sarcoma. However, no drugs currently exist that specifically block the action of EWSR1-FLI1 to cause cancer cells to grow. The McFadden Lab has engineered a "self-destruct button" into the EWSR1-FLI1 gene in Ewing sarcoma cells cultured in the laboratory, and these cells stop growing when the EWSR1-FLI1 gene is turned off. Dr. McFadden is using this laboratory tool to identify proteins that work with EWSR1-FLI1, and identify other genes it controls to cause Ewing sarcoma cells to grow. These studies will help identify new ways to stop the growth of Ewing sarcoma cells.

Rhabdomyosarcoma is a pediatric cancer of the developing skeletal muscle. The mechanisms that drive this tumor are poorly understood. From Dr. McEvoy's preliminary analysis, one possible mechanism is epigenetic deregulation of a group of long noncoding RNAs (lncRNA). This is exciting because lncRNAs play a role in tumorigenesis in other cancer types, including a subset of pediatric tumors. This presents a unique opportunity to develop novel therapeutic approaches for children with rhabdomyosarcoma. Dr. McEvoy's team hypothesizes that lncRNA deregulation is essential for rhabdomyosarcoma development. This study is working to understand the underlying mechanisms that drive this disease and identify potential new therapies. These results will have tremendous impact on patients, especially those with metastatic disease since only 20-40% will survive using current treatments.

Based on progress to date, Dr. Ladle was awarded a new grant in 2020 to fund an additional year of this Scholar grant. As the Aiden's Army Fund St. Baldrick's Scholar, Dr. Ladle is using the body's own immune system to destroy cancer - specifically a class of cancer in children originating from connective tissues called sarcomas. Using fire as an analogy, Dr. Ladle seeks to build an intense flame of a powerful immune response which will specifically kill the cancer cells. To create this fire, one must follow specific steps. The kindling, which must be easily burned, is protein targets on the cancer cells (termed tumor antigens) recognized by the immune system. Next, the spark to ignite the kindling is initial inflammation in the tumor against these tumor antigens. Finally, to feed the fire, fuel or lighter fluid can be added in the form of recently approved immune modulator drugs which, when infused into patients, bind to immune cells residing in the tumor and activates them to kill the tumor cells. Each ordered step is essential in building an effective fire. This project addresses each of these key aspects for generating a successful immune response to treat sarcomas and creating new tumor antigens, adding inflammation to jump start the immune response against these antigens, and combining with new immune modulators allowing the immune cells to be active in destroying sarcomas.

This grant is funded by and named for the Aiden's Army Fund, a St. Baldrick's Hero Fund. When he was 8 years old, Aiden Binkley was diagnosed with Stage IV rhabdomyosarcoma. He had a huge tumor in his pelvis and the cancer had metastasized to his lungs. But this bright, funny and courageous boy believed he got cancer so he could grow up to find a cure for it. Aiden’s story has inspired so many people and his vision to cure cancer is being carried on by Aiden’s Army through the funding of research. They will march until there is a cure!

Brain tumors are the leading cause of cancer-related deaths in children, and ependymomas are the third most common kind. Recent studies have shown that educating the patients own immune system to fight cancers immunotherapy can be safe and effective. Dr. Kohanbash's team has identified three peptides that might activate immune cells to specifically fight one of the more lethal types of ependymoma. Dr. Kohanbash is testing these peptides in the lab. He is also looking at how immunotherapy could help fight all six types of ependymoma that affect kids, and thus is studying relevant characteristics in the largest-ever series of pediatric ependymoma tumors as well as in ependymoma patients already participating in a clinical trial of a vaccine based on another peptide.

A portion of this grant is generously co-supported by the Henry Cermak Fund for Pediatric Cancer Research and the Team Campbell Foundation. The Henry Cermak Fund for Pediatric Cancer Research, a St. Baldrick's Hero Fund was created in memory of a brave boy who had an amazing spirit throughout his battle with a brain tumor. This fund is dedicated to Henry’s wish that “no one gets left out.”

The Team Campbell Foundation, a St. Baldrick's partner, was established in memory of Campbell Hoyt, who courageously battled anaplastic ependymoma, a rare cancer of the brain and spine for five years. Its mission is to improve the lives of families facing a childhood cancer diagnosis through raising awareness, funding research and providing psycho-social enrichment opportunities.

A portion of Dr. Kohanbash's grant was also generously supported by the Henry Cermak Fund for Pediatric Cancer Research.

Based on progress to date, Dr. Green was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-grade gliomas (HGG) are aggressive brain cancers that affect both adults and children. Current treatment options are very limited, and the vast majority of patients die of their tumors within five years of diagnosis. One subtype of high-grade glioma that almost exclusively occurs in children, diffuse intrinsic pontine glioma (DIPG), is the last incurable childhood cancer, with zero percent long-term survivors. As the Luke's Army Pediatric Cancer Research Fund St. Baldrick's Scholar, Dr. Green and his team intend to address these tumors by focusing on a new field of cancer treatment called epigenetics, which literally means "above genetics" and refers to all changes to DNA that do not involve changes to the DNA sequence itself, but instead affect which genes are made into protein. Through prior work, Dr. Green's team has found a gene, BPTF, which controls the expression of many other genes and appears to drive HGG and DIPG growth. Dr. Green aims to determine how exactly BPTF drives growth by interacting with other genes, to measure how BPTF inhibition works with drugs called HDAC inhibitors and whether this strategy could work with current standard treatments, and to measure the effect of a new chemical that inhibits BPTF that could serve as a precursor to medicines targeting BPTF.

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.

Based on progress to date, Dr. Gowda was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Children with high risk B-cell leukemia, especially with loss or dysfunction of IKZF1 gene have very poor outcomes and high relapse rate. Every other child who relapses with high risk leukemia dies from the disease and there has not been much advancement in treatment for this group for the last 30 years. Dr. Gowda and team have found that a cancer promoting protein called casein kinase II (CK2) impairs the important functions of a protein that helps prevent leukemia. Inhibiting the CK2 protein will restore the ability of this protein to function properly and prevent leukemia. Dr. Gowda's team is testing if using a drug that inhibits CK2 protein along with the drugs that already are known to work in leukemia will have stronger anti-leukemia effect and improve the outcome. Using two agents that target same gene or pathway via different mechanisms will ensure effective shutdown of the particular pathway resulting in strong therapeutic effect. This strategy would also help lower the doses of each drug used and reduce their side effects and associated toxicity.

Based on progress to date, Dr. Cimmino was awarded a new grant in 2020 to fund an additional year of this Scholar grant. Vitamin C is essential for maintaining healthy hair, skin, immune system and heart function. In addition to these health benefits, Dr. Cimmino and team propose that vitamin C might be a non-toxic therapeutic for the treatment of patients with pediatric acute myeloid leukemia. Recently, it was discovered that vitamin C enhances the activation of a group of enzymes called TET proteins that are required for normal blood development. A significant fraction of children and young adolescents with acute myeloid leukemia have mutations in TET2, causing impaired TET2 activity and a block in normal blood cell formation. However, only one of the two copies of the TET2 gene is defective in these patients. Dr. Cimmino's team is working to determine if treatment with high-dose vitamin C could enhance the activity of the remaining, non-mutant, TET2 protein, kill leukemia cells and restore normal blood development. Alternative therapies such as treatment with vitamin C might provide a safe and effective strategy to improve outcome for pediatric leukemia patients. Awarded at the New York University School of Medicine, and transferred to University of Miami.

Based on progress to date, Dr. Agarwal was awarded a new grant in 2020 to fund an additional year of this Scholar grant. High-risk neuroblastoma is an aggressive cancer of very young children with less than 50% overall survival. Current therapy includes high-dose chemotherapy and radiation, which has long-term toxic side-effects. Despite these intensive therapies, neuroblastoma commonly relapse. This relapse is the primary cause of death from neuroblastoma due to disease spread, drug-resistance, and toxicity. As the Oliver Wells Fund for Neuroblatoma St. Baldrick's Scholar, Dr. Agarwal is focusing his research on developing effective therapeutic approaches to target those tumor cells which escape initial treatment and regenerate drug-resistant disease. Recently, Dr. Agarwal's team discovered a chemotherapy-resistant, highly tumorigenic sub-population of cells in neuroblastoma tumors. These cells escape initial therapy and may cause aggressive, drug-resistant relapsed disease. Furthermore, they found that specific epigenetic enzymes maintain this cell sub-population by activating key genes. These epigenetic modifiers can be successfully targeted with novel epigenetic inhibitors, currently under pre-clinical trials. These exciting findings suggest a new epigenetic therapeutic approach for high-risk neuroblastoma. This grant supports efforts to uncover the mechanisms controlling neuroblastoma tumorigenicity and relapse, and develop an effective targeted approach for high-risk neuroblastoma.

A portion 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. 

A portion of this grant was also funded by and 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.

Awarded at the Baylor College of Medicine, and transferred to St. John's University.

This grant funds a doctoral student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds two medical students to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds a medical student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds a medical student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds a medical student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds an undergraduate student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.

This grant funds a medical student to complete work in pediatric oncology research for the summer. The experience may encourage them to choose childhood cancer research as a specialty.